StudyFinder
Search Results
345 Study Matches
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Alexandra Sestito - alexandra.sestito@cchmc.org
ALL
PHASE1
NCT05821959
Criteria for Inclusion:
• Participants may be of any age, based on Cohort Criteria
• At least two mutations in the otoferlin gene
• Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
• Preserved distortion product otoacoustic emissions (DPOAEs)
• Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial Criteria for Exclusion:
• Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
• Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
• Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial
• Participants may be of any age, based on Cohort Criteria
• At least two mutations in the otoferlin gene
• Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR
• Preserved distortion product otoacoustic emissions (DPOAEs)
• Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial Criteria for Exclusion:
• Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy
• Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF
• Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial
COMBINATION_PRODUCT: AAVAnc80-hOTOF via Akouos Delivery Device, COMBINATION_PRODUCT: AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
Sensorineural Hearing Loss, Bilateral
Otoferlin, Auditory neuropathy (AN), Deafness, autosomal recessive 9 (DFNB9), Hearing loss, Hearing loss, bilateral, Hearing loss, sensorineural
Precise Infliximab Exposure and Pharmacodynamic Control (REMODEL-CD)
Kimberly Jackson - kimberly.jackson@cchmc.org
ALL
6 years to 22 years old
PHASE2
NCT05660746
Inclusion Criteria:
• Written informed consent from the patient (≥18 years old) or from parent/legal guardian if patient is \<18 years old
• Written informed assent from patient when age appropriate
• Diagnosis of Crohn's disease within the last 90 days (luminal-only or luminal with a perianal fistula or abscess treated with antibiotics for at least 7 days)
• ≥6 years to ≤22 years of age, anti-TNF naïve and starting infliximab
• Clinical activity and luminal inflammation, defined by both (1) and (2) * (1) PCDAI≥10 (\<18 years old) or CDAI ≥150 (≥18 years old) in last 60 days before the decision to start infliximab * (2) SES-CD\>6, or SES-CD\>3 for isolated ileal disease (or a report of large intestinal ulcerations)\* within the last 60 days or a fecal calprotectin \>250 μg/g within last 75 days prior to screening
• C-reactive protein \>1.0 mg/dL in last 30 days and/or fecal calprotectin \>250 μg/g within last 75 days prior to screening
• Negative TB (tuberculosis) interferon-gamma release test and a negative urine pregnancy test for female patients (if menstruation has started)
Exclusion Criteria:
• Diagnosis of ulcerative colitis or inflammatory bowel disease-unspecified
• Prior use of anti-TNF therapy (infliximab, adalimumab, certolizumab pegol, or golimumab)
• Internal (abdominal/pelvic) penetrating fistula(e) in last 180 days
• Intra-abdominal abscess/phlegmon/inflammatory mass in the last 180 days
• Active perianal abscess (receiving oral antibiotics for \<7 days)
• Intestinal stricture (luminal narrowing with pre-stenotic dilation \>3 cm) and surgery planned in the next 90 days
• Have tested positive for Clostridium difficile toxin (stool assay) or other intestinal pathogens within 14 days of screening unless a repeat examination is negative and there are no signs of ongoing infection with that pathogen.
• Current hospitalization for complications of severe Crohn's disease
• Planned use of methotrexate or 6-mercaptopurine (azathioprine) during the induction (first 3 doses of infliximab) phase
• Current ileostomy, colostomy, ileoanal pouch, and/or previous extensive small bowel resection (\>35 cm) or any CD surgery planned within the next 90 days
• History of autoimmune hepatitis, primary sclerosing cholangitis, thyroiditis, or juvenile idiopathic arthritis
• Treatment with another investigational drug in the last four weeks
• History of malignancy (including lymphoma or leukemia)
• Currently receiving treatment for histoplasmosis
• History of TB, human immunodeficiency virus (HIV), an immunodeficiency syndrome, a central nervous system demyelinating disease, history of heart failure or receiving intravenous antibiotics in last 14 days for any infection
• Currently pregnant, breast feeding or plans to become pregnant in the next 1 year
• Inability or failure to provide informed assent/consent
• Any developmental disabilities that would impede providing assent/consent
DEVICE: RoadMAB, DRUG: Infliximab
Crohn Disease
Crohn's
Mirdametinib in Histiocytic Disorders
Monica Trapp - Monica.Trapp@cchmc.org
ALL
2 years and over
PHASE2
NCT06153173
Inclusion Criteria:
• Subjects must be ≥ 2 years of age AND have a diagnosis of a histiocytic disorder that requires systemic therapy * If patient has had a diagnostic biopsy, biopsy must be reviewed and confirmed by CCHMC pathologist as feasible * If patient has had a biopsy but has not had molecular testing done, must have tissue available for mutational analysis * If patient has isolated pituitary/CNS disease or situations where biopsy is not feasible, positive ddPCR blood test for mutation associated with histiocytic neoplasm with clinical features of histiocytosis is sufficient
• Must have measurable disease on PET scan or brain MRI
• Subjects must demonstrate adequate organ function as defined: * Renal: maximum serum creatinine 2x the upper limit of normal (ULN) OR a creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 * Liver: ALT ≤ 3x ULN AND normal INR (≤ 1.5) * Hematologic: Hematology: Albumin ≥ 2.8 g/dL; Absolute neutrophil count ≥ 1.5 x 109/L; Platelets ≥ 100 x 109/L; Hemoglobin ≥ 9.0 g/dL * Patients with organ function abnormalities outside of these thresholds deemed to be the result of histiocytic disease will be considered eligible
Exclusion Criteria:
• Prior therapy with stipulations as described: * Myelosuppressive Chemotherapy: Must not have received any cytotoxic chemotherapy which impacts the growth and development of cells in the bone marrow within 14 days of enrollment onto this study (i.e. cytarabine, cladribine, clofarabine, mercaptopurine, methotrexate, vinblastine) * MEK Inhibitors: Must not have received a MEK inhibitor within 30 days (or 5 half-lives, whichever is longer) of enrollment, NOR have had disease progression on MEK inhibitor * Steroids: Due to the increased risk of an ocular event, the use of systemic oral, inhaled, or ocular glucocorticoid therapy is prohibited within 14 days prior to first dose of mirdametinib. Throughout the treatment period, short term glucocorticoid treatment (30 days or less) is permitted. Any patients requiring long-term steroid use (more than 30 consecutive days) are not eligible. The exception to this rule is subjects with endocrine deficiencies who require physiologic steroids * Radiation: Must not have received radiation within 14 days of study enrollment or have received radiation to the orbit at any time
• Risk factors for retinal vein occlusion (RVO) are listed. Exclusion should be considered by clinical discretion if they have any of the following risk factors for RVO at screening: * Intraocular pressure (IOP) \> 21 mmHg; if IOP is unable to be obtained (eg age, cooperation, tolerability), ophthalmologist's exam findings and overall assessment will be utilized. If in the ophthalmologist's assessment there are no signs of raised IOP, the subject will be considered eligible for this parameter * Glaucoma or any significant abnormality (≥ grade 2) on ophthalmologic exam that is uncontrolled with intervention * Serum cholesterol \> 300 mg/dL * Serum triglycerides \> 300 mg/dL * Hyperglycemia (either fasting blood glucose \> 125 mg/dL OR random blood glucose \> 200 mg/dL) * Uncontrolled hypertension (participants ≤ 12 years of age with a blood pressure ≥ 95th percentile for age + 12 mmHg; participants ≥ 13 years of age with a blood pressure ≥ 140/90 mm Hg) unresolved on repeat measurement
• LVEF \< 55% at screening OR history of clinically significant cardiac disease, unless deemed to be the direct result of disease
• Subjects who are pregnant or breastfeeding, or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study
DRUG: Mirdametinib
Langerhans Cell Histiocytosis (LCH), Juvenile Xanthogranuloma (JXG), Rosai-Dorfman Disease (RDD), Histiocytic Disorders
Protocol CAUSE-03 / CHEETAH (CHEETAH)
Molly Brausch-Bradner - Molly.Brausch-bradner@cchmc.org
ALL
6 years to 17 years old
NCT06136091
Inclusion Criteria:
• Participant and/or parent guardian must be able to understand and provide informed consent and assent
• Have a primary place of residence in one of the pre-selected recruitment census tracts as outlined in the Protocol CAUSE-03 Manual of Operations (MOP) a. Participants who do not live in the pre-selected census tracts but live within the Office of Management and Budget (OMB) defined Metropolitan Statistical Area and have publicly funded health insurance will qualify for inclusion
• Either:
• Have had a diagnosis of asthma made \> 1 year prior to recruitment; participants who received an asthma diagnosis by a clinician \<= 1 year prior to recruitment must report that their respiratory symptoms were present for more than 1 year prior to recruitment (asthma group), or
• No report of ever being diagnosed with asthma (non-asthma group)
• Either:
• Require at least Step 2 therapy at the Screening/Enrollment Visit (asthma group), or
• Have not used any asthma medications in the prior year (non-asthma group)
• Are able to perform acceptable and repeatable spirometry per American Thoracic Society (ATS) criteria prior to enrollment
• Have documentation of current medical insurance with prescription coverage at the Screening/Enrollment Visit
• Participant and/or parent guardian has a smartphone compatible with the study electronic Patient Reported Outcomes (ePRO) system, Medidata Patient Cloud, and is willing to download one application for study use
Exclusion Criteria:
• Parent or guardian is not able or willing to give written informed consent or comply with study protocol
• Have concurrent medical problems that would require systemic corticosteroids or other immunomodulators during the study
• Are currently receiving immunotherapy
• Are currently receiving treatment with a biologic therapy or have received a biologic therapy within 3 months prior to enrollment
• Are currently requiring greater than fluticasone 500 mcg bid plus Long-Acting Beta Agonists (LABA) one puff twice daily or its equivalent plus Long Acting Muscarinic Antagonists (LAMA) and/or individuals using oral corticosteroids daily or every other day for more than 14 days at the time of the Screening/Enrollment Visit
• Are currently pregnant or lactating, or plan to become pregnant during the time of study participation. Females of child-bearing potential (post-menarche) must be abstinent or use a medically acceptable birth control method throughout the study (i.e., oral subcutaneous, mechanical, or surgical contraception)
• Have a known, pre-existing clinically important lung condition other than asthma.
• Have a current malignancy or previous history of cancer in remission for less than 12 months prior to enrollment
• Have a known immunodeficiency disease
• Use of investigational drugs within 4 weeks of enrollment
• Have past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the site investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study
• If in the asthma group, will not allow the study clinician, an asthma specialist, to manage their disease for the duration of the study or who are not willing to change their asthma medications to follow Protocol CAUSE-03 CHEETAH
• If in the non-asthma group, having bronchodilator reversibility (improvement in Forced expiratory volume in 1 second (FEV1) with albuterol \> = 10%) at the Screening/Enrollment visit
• Have had a life-threatening asthma exacerbation in the last 2 years requiring intubation, mechanical ventilation or resulting in a hypoxic seizure. Potential participants may be reassessed as outlined in the Protocol CAUSE-03 Manual of Procedures (MOP)
Asthma
Asthma, Observational study, Children
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (FLY)
Jenel Facey - jenel.facey@cchmc.org
ALL
2 years to 17 years old
PHASE2
NCT06328608
Inclusion Criteria:
* Participants with the provision of informed consent from their legal guardians
* Boys and girls aged 2 to 7 years (Cohort A), 8 to 12 years (Cohort B), or 13 to \<18 years (Cohort C).
* Confirmed diagnosis of Fabry disease
* Presence of at least one of the following characteristic features of Fabry disease: neuropathic pain, cornea verticillata, and/or clustered angiokeratoma.
* History of Fabry pain: Fabry crises OR chronic pain.
* Clinical condition that, in the investigator's opinion, requires ERT treatment.
Exclusion Criteria:
All Subjects:
* Estimated glomerular filtration rate (eGFR) at screening \< 80 mL/min/1.73 m2.
* History of type I hypersensitivity reactions (anaphylactic or anaphylactoid life-threatening reaction) to other ERT treatment for Fabry disease or any component of the study drug.
* Initiation of treatment with an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin II receptor blocker (ARB) or a dose change in ongoing treatment in the four weeks before screening.
* Urine protein to creatinine ratio (UPCR) \> 0.5 g/g (0.5 mg/mg or 500 mg/g) if not treated with an ACE inhibitor or ARB.
* Currently taking another investigational drug for any condition.
* History of acute kidney injury in the 12 months before screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g., ischaemia, toxic injury); or extrarenal pathology (e.g., prerenal azotaemia, acute postrenal obstructive nephropathy).
* History of renal dialysis or kidney transplantation.
* History of or current malignancy requiring treatment.
* Severe cardiomyopathy or significant unstable cardiac disease within six months before screening.
* A positive test for Severe Acute Respiratory Syndrome-Coronavirus 2 (SARS-CoV-2) within three months before screening.
* Presence of any medical, emotional, behavioural, or psychological condition that, in the Investigator's judgement, could interfere with the subject's compliance with the requirements of the study.
Additional Exclusion Criteria for Subjects Enrolled in Stage I:
* Female
* Non-classic form of Fabry disease
* Receipt of treatment for Fabry disease within six months before screening
* Positive for anti-PRX-102 antibodies at screening
Additional Exclusion Criteria for Subjects in Stage II (i.e., non-treatment naïve males or females):
* Unwilling to discontinue current ERT treatment for Fabry disease before baseline.
* Females: Pregnant or lactating, or of childbearing potential with a fertile male partner and unwilling to use a highly reliable method of contraception from the informed consent signature until 30 days after the last infusion. DRUG: PRX-102 1 mg/kg every two weeks
Fabry Disease
Comparison of Uncomplicated Candidemia Therapy Duration in Children (COUNT)
Caitlin Caitlin Brammer - Caitlin.Brammer@cchmc.org
ALL
4 month(s) and over
NA
NCT05763251
Inclusion Criteria:
• Age \> 120 days at the time of the first negative blood culture at any participating site;
• Candidemia with at least one positive blood culture for any Candida spp;
• Receiving/received an echinocandin (caspofungin, micafungin, anidulafungin, or rezafungin) as primary antifungal therapy for candidemia for at least 2 days from day of first negative culture with continuation of uninterrupted systemic antifungal therapy at the time of enrollment);
• Sustained clearance of Candida spp. defined as negative blood culture(s) obtained after onset of candidemia and before day of randomization;
• Partial or complete clinical response, as defined by published guidelines (Table 5), on or before day of randomization;
• Between the onset of qualifying candidemia and randomization, no suspicion of disseminated candidiasis by patient's clinical team or, if deemed clinically necessary, documented negative radiological imaging such as an abdominal ultrasound or abdominal CT scan.
Exclusion Criteria:
• Already receiving antifungal therapy for a previously diagnosed systemic invasive fungal disease;
• Neutropenic (absolute neutrophil count \< 500 cells/µl) at the time of enrollment or anticipated to be neutropenic in the week following randomization;
• Have an underlying condition that requires them to be on antifungal prophylaxis when not receiving directed therapy for an invasive fungal disease;
• Previous enrollment in this trial;
• Females of childbearing age with a current pregnancy diagnosis or without a negative pregnancy test for their current admission;
• A documented DNR order;
• Have an implantable cardiac device (e.g., ventricular assist device, pacemaker)
OTHER: therapy duration
Invasive Candidiasis
CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma
- cancer@cchmc.org
ALL
12 months to 21 years old
PHASE1
NCT04870944
Inclusion Criteria:
* Parts A and B: Patients must be \>= 12 months and =\< 21 years of age at the time of study enrollment
* Patients must have had histologic verification of malignancy at original diagnosis or relapse, except in patients with diffuse intrinsic brain stem tumors, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers, including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG)
* Part A: Patients with relapsed or refractory solid tumors or lymphoma, including patients with CNS tumors or known CNS metastases (including untreated or progressive) are eligible
* Part B: Patients with progressive or recurrent DIPG (diagnosed by biopsy or imaging characteristics) and other H3 K27-altered DMG previously treated with radiation therapy
* Part A: Patients must have either measurable or evaluable disease
* Part B: Patients must have measurable disease
* Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
* Patients must have a performance status corresponding to Easter Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age. Patients must have a Karnofsky or Lansky score \>= 50%
* Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately
* Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive
* Solid tumor patients: \>= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
* Anti-cancer agents not known to be myelosuppressive (eg, not associated with reduced platelet or absolute neutrophil count \[ANC\] counts): \>= 7 days after the last dose of agent
* Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
* Corticosteroids: If used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid. Patients with CNS tumors receiving corticosteroids must have been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment
* Hematopoietic growth factors: \>= 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
* Interleukins, interferons and cytokines (other than hematopoietic growth factors): \>= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
* Stem cell Infusions (with or without total body irradiation \[TBI\]):
* Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: \>= 84 days after infusion and no evidence of graft versus host disease (GVHD)
* Autologous stem cell infusion including boost infusion: \>= 30 days
* Cellular therapy: \>= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer \[NK\] cells, dendritic cells, etc.)
* Radiation therapy \[XRT\]/external beam irradiation including protons: \>= 14 days after local XRT; \>= 150 days after TBI, craniospinal XRT or if radiation to \>= 50% of the pelvis; \>= 42 days if other substantial bone marrow (BM) radiation
* Radiopharmaceutical therapy (e.g., radiolabeled antibody, I-131 metaiodobenzylguanidine \[131I MIBG\]): \>= 42 days after systemically administered radiopharmaceutical therapy
* Patients must not have received prior exposure to CBL0137
* For patients with solid tumors without known bone marrow involvement:
* Peripheral absolute neutrophil count (ANC) \>= 1000/uL (performed within 7 days prior to enrollment unless otherwise indicated)
* Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled must be evaluable for hematologic toxicity
* For patients with solid tumors without known bone marrow involvement:
* Platelet count \>= 100,000/uL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) (performed within 7 days prior to enrollment unless otherwise indicated)
* Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled must be evaluable for hematologic toxicity
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a creatinine based on age/sex as follows (performed within 7 days prior to enrollment unless otherwise indicated):
* Age: Maximum serum creatinine (mg/dL)
* 1 to \< 2 years: 0.6 (male); 0.6 (female)
* 2 to \< 6 years: 0.8 (male); 0.8 (female)
* 6 to \< 10 years: 1 (male); 1 (female)
* 10 to \< 13 years: 1.2 (male); 1.2 (female)
* 13 to \< 16 years: 1.5 (male); 1.4 (female)
* \>= 16 years: 1.7 (male); 1.4 (female)
* Patients with solid tumors:
* Bilirubin (sum of conjugated + unconjugated or total) =\< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment unless otherwise indicated)
* Patients with solid tumors:
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L (performed within 7 days prior to enrollment unless otherwise indicated)
* Shortening fraction of \>= 27% by echocardiogram (performed within 7 days prior to enrollment unless otherwise indicated) or
* Ejection fraction of \>= 50% by gated radionuclide study (performed within 7 days prior to enrollment unless otherwise indicated)
* Corrected QT (QTC) \< 480 msec (performed within 7 days prior to enrollment unless otherwise indicated)
* Patients with seizure disorder may be enrolled if seizures well controlled without the use of enzyme-inducing anti-convulsant agents. Well controlled is defined by no increase in seizure frequency in the prior 7 days
* Nervous system disorders (Common Terminology Criteria for Adverse Events \[CTCAE\] version \[v\]5) resulting from prior therapy must be =\< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
* Patients have consented to receive a central venous catheter prior to the administration of CBL0137. A central line is required for CBL0137 administration
Exclusion Criteria:
* Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, OR because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study. Abstinence is an acceptable method of birth control
* Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, \>= 14 days must have elapsed since last dose of corticosteroid
* Patients who are currently receiving another investigational drug are not eligible
* Patients who are currently receiving other anti-cancer agents are not eligible (except leukemia patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy)
* Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
* Patients who are receiving drugs that are strong inducers or inhibitors of CYP3A4, CYP2B6 (e.g., carbamazepine) and CYP1A2 (e.g., ciprofloxacin, enoxacin, fluvoxamine, smoking) are not eligible. These agents are to be avoided for 7 days prior to the start of CBL0137 and for the duration of the protocol therapy. Sensitive substrates of CYP2D6 (e.g., atomoxetine, desipramine, dextromethorphan, eliglustat, nebivolol, nortriptyline, perphenazine, tolterodine, R-venlafaxine) should also be avoided for the duration protocol therapy
* Patients who are receiving drugs associated with a known risk of Torsades de Pointes (TdP) are not eligible. Drugs associated with known risk of Torsades de Pointes (TdP) are to be avoided for 7 days prior to the start of CBL0137 and for duration of the protocol therapy
* Patients with known peripheral vascular disease are excluded
* Patients with a history of pro-thrombotic disorder are not eligible
* Patients who have an uncontrolled infection are not eligible
* Patients who have received a prior solid organ transplantation are not eligible
* Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspirate, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Echocardiography Test, DRUG: FACT Complex-targeting Curaxin CBL0137
Diffuse Midline Glioma, H3 K27-Altered, Metastatic Malignant Neoplasm in the Central Nervous System, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Diffuse Midline Glioma, H3 K27-Altered, Recurrent Lymphoma, Recurrent Malignant Solid Neoplasm, Recurrent Primary Malignant Central Nervous System Neoplasm, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Primary Malignant Central Nervous System Neoplasm
A Study of BLB-201 RSV Vaccine in Infants and Children
Jamie Kidd - jamie.kidd@cchmc.org
ALL
6 months to 5 years old
PHASE1
NCT05655182
Inclusion criteria for sero+ children 18 to 59 months of age enrolled in Groups 1 and 2:
Healthy children at least 18 months but less than 60 months of age whose legally-acceptable representative (LAR) understands and signs the trial informed consent and agrees to vaccine administration following a detailed explanation of the trial.
Determined by medical history, targeted physical exam, and clinical judgement of the investigator to be in a good state of health. Screening laboratory values slightly outside lab normal ranges may be acceptable if the site investigator determines that they are not clinically significant. Permitted concomitant medications include nutritional supplements, medications for gastroesophageal reflux, eye drops, and topical medications, including topical steroids, topical antibiotics, and topical antifungal agents.
Sero+ for RSV as defined by serum RSV antibody titer assay
Participant is expected to be available for the duration of the trial.
The LAR confirms that the subject has received routine immunizations appropriate for age based on the current Advisory Committee on Immunization Practices (ACIP) Recommended Immunization Schedule for Children and Adolescents Aged 18 Years or Younger.
Growing normally for age as demonstrated on a World Health Organization (WHO) growth chart, AND has a current height and weight above the 3rd percentile for age.
Inclusion criteria for sero+ or sero- infants and children 8 to 24 months of age enrolled in Groups 3 through 6:
Healthy children at least 8 months but less than 25 months of age whose LAR understands and signs the trial informed consent and agrees to vaccine administration following a detailed explanation of the trial.
Determined by medical history, targeted physical exam, and clinical judgement of the investigator to be in a good state of health. Screening laboratory values slightly outside lab normal ranges may be acceptable if the site investigator determines that they are not clinically significant. Permitted concomitant medications include nutritional supplements, medications for gastroesophageal reflux, eye drops, and topical medications, including topical steroids, topical antibiotics, and topical antifungal agents.
Sero- OR sero+ for RSV antibody, defined by serum RSV antibody titer assay not more than 30 days prior to vaccination.
Participant is expected to be available for the duration of the trial.
The LAR confirms that subject has received routine immunizations appropriate for age based on the current Advisory Committee on Immunization Practices (ACIP) Recommended Immunization Schedule for Children and Adolescents Aged 18 Years or Younger.
Growing normally for age as demonstrated on a World Health Organization (WHO) growth chart, AND
If \<1 year of age: has a current height and weight above the 5th percentile for age.
If ≥1 year of age: has a current height and weight above the 3rd percentile for age.
Subject Exclusion Criteria
\<8 months of age and \>60 months of age at the time of planned vaccine inoculation.
Born at less than 34 weeks gestation for subjects ≥ 1 year of age at enrollment
Born at less than 37 weeks gestation, and at the date of inoculation less than 1 year of age.
Maternal history of a positive HIV test before or during pregnancy.
Maternal history of illicit drug abuse or alcohol abuse.
Evidence of chronic disease except for chronic diseases that are mild, stable and not immune compromising or require recent change (\< 60 days) in management (e.g., mild stable eczema, mild allergic rhinitis)
Clinically significant pulmonary, cardiovascular, hepatic or renal functional abnormality as determined by medical history or physical exam. Abnormal pulse oximetry testing during screening for undetected critical congenital heart disease or concern for such by medical history or physical exam.
Acute or chronic medical condition or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgement, make the participant inappropriate for the study.
History of severe infection (e.g., requiring hospitalization).
Known or suspected impairment of immunological functions, bone marrow/solid organ transplant recipients.
Receiving immunosuppressive therapy including systemic corticosteroids.
Major congenital malformations, including congenital cleft palate or cytogenetic abnormalities.
Suspected or documented developmental disorder, delay, or other developmental problem.
Cardiac abnormality requiring treatment. Participants with clinically insignificant cardiac abnormalities (e.g., clinically insignificant patent foramen ovale) requiring no treatment may be enrolled.
Lung disease or reactive airway disease.
History of wheezing episode/s or receipt of bronchodilator therapy
Previous receipt of supplemental oxygen therapy in a home setting.
History of severe RSV infection or severe respiratory virus infection (e.g., requiring hospitalization).
Previous immunization with an investigational RSV vaccine.
Previous or planned administration of any anti-RSV antibody product within 6 months of receipt of study vaccine.
Previous receipt of immunoglobulin or any other antibody products within the past 6 months.
Previous receipt of any blood products within the past 6 months.
Previous anaphylactic reaction.
Previous serious vaccine-associated adverse reaction or one that was Grade 3 or above.
Known hypersensitivity to any study vaccine product component.
Household contact with any of the following groups of individuals for the period up to 28 days after vaccination (including after each dose for cohorts receiving two doses of vaccine):
Member of a household that contains an infant who is less than 6 months of age at the date of inoculation through the 28th day after inoculation. In groups assigned to two doses of vaccine, to include date of inoculation through the 28th day after the second inoculation.
Pregnant woman.
Persons with hospitalization for asthma or other chronic respiratory disease in the past 5 years.
Member of a household that, at the date of inoculation through the 28th day after inoculation (including second dose if scheduled), contains an immunocompromised individual including but not limited to:
A person who is HIV-infected.
A person who has cancer and has received chemotherapy within the 12 months prior to enrollment.
A person with a solid organ or bone marrow transplant.
A person currently receiving immunosuppressive agents.
Attends a daycare facility that does not separate children by age and contains an infant
\<6 months of age at the date of inoculation through the 28th day after inoculation.
Neurological and neurodevelopmental conditions (e.g., cerebral palsy, epilepsy, stroke, seizures).
History of postinfectious or postvaccine neurological sequelae.
Autoimmune, inflammatory, vascular, or rheumatic disease.
Household contact of another child enrolled into the trial.
Inadequate venous access for repeated phlebotomy.
Subject's LAR/s who, in the opinion of the site investigator, are not suitable participants for the study, for any reason not previously delineated, including subjects with any condition that would in the opinion of the site investigator place the subject at unacceptable risk of injury or render the subject unable to meet the requirements of the protocol.
Subjects testing positive for infection with RSV, Influenza, or SARS-CoV-2 in the 3 months prior to enrollment.
Planned receipt of any of the following prior to planned trial vaccine receipt (Day 1 and Day 57 for group receiving 2 doses of vaccine):
Inactivated influenza vaccine within 14 days prior, or
Any other inactivated vaccine or live-attenuated rotavirus vaccine within the 14 days prior, or
Any live vaccine, other than rotavirus vaccine, within the 28 days prior, or
Another investigational vaccine or investigational drug within 28 days prior.
Salicylate (aspirin) or salicylate-containing products within 28 days prior.
Planned receipt of any of the following after planned trial vaccine receipt (Day 1 and Day 57 for groups receiving 2 doses of vaccine):
Inactivated vaccine or live-attenuated rotavirus vaccine within the 14 days after, or
Any live vaccine other than rotavirus in the 28 days after, or
Another investigational vaccine or investigational drug in the 56 days after.
Planned receipt of any of the following medications within 7 days of trial enrollment and 7 days after trial vaccine (Day 1 and also Day 57 for groups receiving 2 doses of vaccine):
Systemic antibacterial, antiviral, antifungal, anti-parasitic, or antituberculous agents, whether for treatment or prophylaxis, or systemic or nasal steroid therapy for acute illness.
Any other intranasal medications, or
Other prescription medications except permitted concomitant medications. Permitted concomitant medications (prescription or non-prescription) include nutritional supplements, medications for gastroesophageal reflux, eye drops, and topical medications, including (but not limited to) cutaneous (topical) steroids, topical antibiotics, and topical antifungal agents.
History of bleeding disorder or significant problem with bleeding
American Indian or Alaska Native Infants/Children (high risk for severe RSV infection) AND eligible to receive nirsevimab
Temporary exclusion criteria for sero+ children, sero- children, and infants:
The following are temporary or self-limiting conditions, and once resolved, the subject may be enrolled, if otherwise eligible. If the period of temporary exclusion is greater than 30 days, sero- children will need to be rescreened for levels of RSV neutralizing antibody.
Any of the following events at the time of enrollment:
Fever (temperature of ≥100.4°F per site standard based on age; e.g., oral for older children, rectal for infants, axillary screening), or
Upper respiratory signs or symptoms (rhinorrhea, cough, or pharyngitis) or
Nasal congestion significant enough to interfere with successful vaccination.
Otitis media.
Contact with a person diagnosed with RSV, Influenza, coronavirus disease-2 (COVID- 19) or other viral respiratory illnesses within the preceding 10 days.
BIOLOGICAL: PIV5-vectored RSV Vaccine (BLB-201) Low Dose, BIOLOGICAL: PIV5-vectored RSV Vaccine (BLB-201) High Dose, DRUG: Placebo
Respiratory Syncytial Virus Infections
Human respiratory syncytial virus (RSV), Lower respiratory tract infection (LRTI)
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Alexandra Duell - alexandra.duell@cchmc.org
ALL
12 years and over
PHASE1
NCT07089121
Inclusion Criteria:
* At least age 12
* definitive diagnosis of childhood-onset systemic lupus erythematous, juvenile Myasthenie gravis, juvenile dermatomyositis and AAV
* Signs and symptoms of moderate disease
* History of systemic treatment
* Parent/Guardian/Patient must be able to give written informed consent
Exclusion Criteria:
* Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient;
* Abnormal PT/INR or PTT increased \> 1.5-fold or patient is on anticoagulation therapy (except in cases of elevated PTT with documented lupus anticoagulant; or in patients who have been on stable doses of anticoagulation therapy for more than 6 months of VTE diagnosis; or in patients on stable doses of anticoagulation therapy for at least 8 weeks of atrial fibrillation diagnosis; these conditions will not be exclusionary unless, in the investigator's opinion, they make participation in the study unsafe);
* ANC \< 1000 cells/microliter ;
* Hemoglobin \< 8.0 g/dL ;
* Platelets \< 50,000/mm3 (NOTE: platelet transfusions are permissible);
* ALT and/or AST with GGT ≥ 3× upper limit of normal
* Creatine Clearance less than 30mL/min /1.73 m2;
* History of primary immunodeficiency, organ, or allogeneic bone marrow transplant;
* Patients must be seronegative for hepatitis B surface antigen;
* Patients must be seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patients must be tested for the presence of viremia by RT-PCR and must be HCV RNA negative;
* History of positive HIV or positive HIV at screening;
* Active tuberculosis or positive QuantiFERON test at screening;
* Any other laboratory abnormality that, in the opinion of the investigator, may jeopardize the subject's ability to participate in the study; 23. Any active significant cardiac or pulmonary disease not related to the primary indication as determined by principal investigator and medical monitor Note: Patients with asthma and COPD controlled with inhaled medications are allowed; 24. Any arterial or venous thromboembolic events in the past 3 months; 25. History of malignancy that required treatment in the past 3 years except for successfully-treated squamous cell and/or basal cell carcinoma of the skin and/or breast or colon cancer that is surgically removed and did not require adjuvant chemotherapy or radiotherapy; 26. Treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer); 27. Receipt of a live vaccination within 4 weeks prior to baseline (Day 1) or intent to receive live vaccination during the study (Note: mRNA-based vaccines such as those against SARS-CoV-2 are not considered live; likewise, the Janssen Covid-19 vaccine is not live); 28. History of significant recurrent infections or any active infection that may interfere with the patient's participation in the opinion of the investigator; 29. Any known psychiatric illness that may interfere with the patient's participation in the study in the opinion of the investigator. DRUG: Descartes-08
Childhood-onset Systemic Lupus Erythematous, ANCA-Associated Vasculitis (AAV), Juvenile Myasthenia Gravis, Juvenile Dermatomyositis
Quality of Pediatric Resuscitation in a Multicenter Collaborative (pediRES-Q)
Maya Maya Dewan - Maya.Dewan@cchmc.org
ALL
0 years to 18 years old
NCT02708134
Inclusion Criteria:
* Patient received chest compressions for at least 1 minute
* Patient between gestational age ≥37 weeks and 18 years of age
Exclusion Criteria:
* Patient on veno-arterial extracorporeal membrane oxygenation (ECMO) therapy at beginning of CPR event Cardiac Arrest, Cardiopulmonary Arrest
Chest compressions, Cardiopulmonary Resuscitation, Pediatric
Biospecimen Collection to Identify Gene Mutations for High Risk Pancreatic Cancer in Pediatric Patients, INSPPIRE 2 Study
Maisam Abu-El-Haija - Maisam.Haija@cchmc.org
ALL
Up to 17 years old
NCT06651580
Inclusion Criteria:
* All subjects/parents must sign an informed consent and/or assent indicating that they are aware of the investigational nature of this study
* Subjects/parents must have signed an authorization for the release of their or their child's protected health information
* All children must be under 18 years of age at the time of enrollment
* All children providing samples should fit the ARP or CP inclusion criteria defined below:
* Acute pancreatitis (AP): AP is defined as requiring 2 of the following:
* Abdominal pain compatible with AP
* Serum amylase and/or lipase values \>= 3 times upper limits of normal
* Imaging findings of AP, such as gland enlargement, acute inflammatory changes, and fluid collections
* ARP is defined as: At least 2 episodes of acute pancreatitis with complete resolution of pain and a \>= 1 month pain-free interval between episodes
* Chronic Pancreatitis:
* Children with at least:
* One irreversible structural change in the pancreas with or without abdominal pain +/- exocrine pancreatic insufficiency +/- diabetes
* Irreversible structural changes:
* Ductal calculi, dilated side branches, parenchymal calcifications found in any imaging (abdominal ultrasound \[abd US\], magnetic resonance imaging/magnetic resonance cholangiopancreatography \[MRI/MRCP\], computerized tomography \[CT\], endoscopic retrograde cholangiopancreatography \[ERCP\], endoscopic US \[EUS\])
* Ductal obstruction or stricture/dilatation/irregularities that are persistent (for \>= 2 months) on any imaging
* Parenchymal atrophy, irregular contour, accentuated lobular architecture, cavities alone are not diagnostic findings for CP
* Surgical or pancreatic biopsy specimen demonstrating histopathologic features compatible with CP (acinar atrophy, fibrosis, protein plugs, infiltration with lymphocytes, plasma cells, macrophages)
Exclusion Criteria:
* Subjects must not have any significant medical illnesses that in the investigator's opinion cannot be adequately controlled with appropriate therapy or would compromise the subject's ability to tolerate study interventions PROCEDURE: Biospecimen Collection, OTHER: Quality-of-Life Assessment, OTHER: Questionnaire Administration
Chronic Pancreatitis, Exocrine Pancreas Carcinoma, Recurrent Acute Pancreatitis
Engaging Mental Health Services for Preschoolers at Risk (NTRI-MH)
Andrea Davila Bocaranda - andrea.davilabocaranda@cchmc.org
ALL
18 years and over
NA
NCT07054554
Inclusion Criteria:
* Family Navigators: Having experience working as family navigator and/or community health worker; Latinx, Black or White; \> 18 years old.
* Caregivers: current caregiver of a HStart preschooler at-risk for MH disorders (ASQ-SE above threshold score)
* Teachers: Current Head Start (HStart) teacher from one of the 2 HStart site;\> 18 years old; Any race/ethnicity.
* PCPs: Currently providing primary care for HStart preschool age children at risk for MH disorders; \> 18 years old; Any race/ethnicity.
Exclusion Criteria:
* Unable to read/speak either English or Spanish. OTHER: NTRI-MH
Mental Health Disorder, Family Navigation, Minoritized Population, Preschool Age Children
Family Navigation, mental health disorder, Preschool Age Children
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)
Jamie Wilhelm - jamie.wilhelm@cchmc.org
ALL
3 month(s) and over
PHASE2
NCT02143830
Inclusion Criteria:
* Patients must have a diagnosis of Fanconi anemia
* Patients must have one of the following hematologic diagnoses:
• Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features:
• Platelet count \<20 x 109/L or platelet transfusion dependence\*
• ANC \<1000 x 109/L
• Hgb \<8 gm/dl or red cell transfusion dependence\*
• Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification
• Acute Myelogenous Leukemia (untreated, in remission or with refractory or relapsed disease) * Donors will be either human leukocyte antigen (HLA) compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor). * Patients and donors may be of either gender or any ethnic background. * Patients must have a Karnofsky adult, or Lansky pediatric performance scale status \> 70%. * Patients must have adequate physical function measured by:
• Cardiac: asymptomatic or if symptomatic then 1) left ventricular ejection fraction (LVEF) at rest must be \> 50% and must improve with exercise or 2) Shortening Fraction \> 29%
• Hepatic: \< 5 x upper limit of normal (ULN) alanine transaminase (ALT) and \< 2.0 mg/dl total serum bilirubin.
• Renal: serum creatinine \<1.5 mg/dl or if serum creatinine is outside the normal range, then CrCl \> 50 ml/min/1.73 m2
• Pulmonary: asymptomatic or if symptomatic, DLCO \> 50% of predicted * Each patient must be willing to participate as a research subject and must sign an informed consent form. * Female patients and donors must not be pregnant or breastfeeding at the time of signing consent. Women must be willing to undergo a pregnancy test prior to transplant and avoid becoming pregnant while on study.
Exclusion Criteria:
* Active CNS leukemia
* Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.
* Active uncontrolled viral, bacterial or fungal infection
* Patient seropositive for HIV-I/II; HTLV -I/II DRUG: Busulfan, DRUG: Cyclophosphamide, DRUG: Fludarabine, DRUG: rabbit ATG, DRUG: G-CSF, BIOLOGICAL: Peripheral blood stem cell
Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
marrow aplasia, cytopenia, myelodysplasia, AML, bone marrow transplant, cytoreductive regimen, T-cell reduction
A Study to Evaluate the Efficacy and Safety of Cannabidiol Oral Solution (CBD-OS [GWP42003-P, JZP926]) for the Treatment of Focal-Onset Seizures
Katie Hall - Katie.Hall@cchmc.org
ALL
12 years to 75 years old
PHASE1
NCT07233239
Participants are eligible to be included in the main study only if all of the following criteria apply:
• Participant has a documented diagnosis of focal epilepsy according to the ILAE Classification of Epilepsy, 2017, characterized by focal seizure types with typical interictal/ictal EEG findings (eg, history of an EEG with focal sharp waves or slowing). Participants with a documented diagnosis of focal epilepsy and a normal EEG are eligible for inclusion.
• Participant is currently treated with at least 1, but no more than 4, antiseizure medications on a stable regimen.
• Participant is aged 12 to 75 years old, inclusive, at Screening. Participants are excluded from the study if any of the following criteria apply:
• Has a concurrent, confirmed diagnosis of non-epileptic seizures or events that can confound the assessment of the efficacy measures, in the opinion of the investigator.
• Has clinically significant unstable medical condition(s), other than epilepsy.
• History of suicidal behavior, current suicidal risk as determined from history, or presence of active suicidal ideation as indicated by a positive response to Item 4 or Item 5 on the C-SSRS or is considered at risk of suicide or self-harm based on the clinical judgement of the investigator following interview with the participant and/or caregiver.
• Has known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention, such as sesame oil.
• Is currently treated with Epidiolex or received treatment with Epidiolex within 28 days prior to Screening (Visit 1).
• Is currently using or has used recreational or medicinal cannabis, cannabinoid/CBD based medications, products, or supplements (botanical or synthetic) within 28 days prior to Screening (Visit 1) and/or is unwilling to abstain for the duration of the study.
• Presence of only nonmotor focal aware seizures or primary generalized epilepsies.
• Participant has a documented diagnosis of focal epilepsy according to the ILAE Classification of Epilepsy, 2017, characterized by focal seizure types with typical interictal/ictal EEG findings (eg, history of an EEG with focal sharp waves or slowing). Participants with a documented diagnosis of focal epilepsy and a normal EEG are eligible for inclusion.
• Participant is currently treated with at least 1, but no more than 4, antiseizure medications on a stable regimen.
• Participant is aged 12 to 75 years old, inclusive, at Screening. Participants are excluded from the study if any of the following criteria apply:
• Has a concurrent, confirmed diagnosis of non-epileptic seizures or events that can confound the assessment of the efficacy measures, in the opinion of the investigator.
• Has clinically significant unstable medical condition(s), other than epilepsy.
• History of suicidal behavior, current suicidal risk as determined from history, or presence of active suicidal ideation as indicated by a positive response to Item 4 or Item 5 on the C-SSRS or is considered at risk of suicide or self-harm based on the clinical judgement of the investigator following interview with the participant and/or caregiver.
• Has known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention, such as sesame oil.
• Is currently treated with Epidiolex or received treatment with Epidiolex within 28 days prior to Screening (Visit 1).
• Is currently using or has used recreational or medicinal cannabis, cannabinoid/CBD based medications, products, or supplements (botanical or synthetic) within 28 days prior to Screening (Visit 1) and/or is unwilling to abstain for the duration of the study.
• Presence of only nonmotor focal aware seizures or primary generalized epilepsies.
DRUG: CBD-OS
Focal Seizures
Focal Seizures
Third Party Viral Specific T-cells (VSTs)
Jamie Wilhelm - Jamie.Wilhelm@cchmc.org
ALL
0 month(s) and over
PHASE2
NCT02532452
Inclusion Criteria:
* Immunocompromised patient with evidence of viral infection or reactivation
* Age \>1 day
* Recipients who have had a stem cell transplant must be at least 21 days after stem cell infusion
* Clinical status must allow tapering of steroids to \< 0.5mg/kg prednisone or other steroid equivalent
* Must be able to receive CTL infusion in Cincinnati
* Informed consent obtained by PI or sub-investigator either in person or by phone
Exclusion Criteria:
* Active acute GVHD grades II-IV
* Uncontrolled bacterial or fungal infection
* Uncontrolled relapse of malignancy requiring treatment with chemotherapy
* Infusion of ATG or alemtuzumab within 2 weeks of VST infusion
* Biopsy confirmed acute rejection of solid organ transplant OR empiric treatment of suspected but not confirmed acute rejection of solid organ transplant within the last 30 days BIOLOGICAL: Viral Specific VST Infusion
Viral Infection, Viral Reactivation, Infection in an Immunocompromised Host
Epstein-Barr Virus (EBV), Adenovirus (ADV), Cytomegalovirus (CMV), T-Cells, Donor, BK virus (BKV)
Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
- cancer@cchmc.org
ALL
Up to 21 years old
PHASE3
NCT05304585
Inclusion Criteria:
* All patients must be enrolled on APEC14B1 (NCT02402244) and consented to the Molecular Characterization Initiative (Part A) prior to enrollment and treatment on ARST2032 (this trial).
* Patients must be =\< 21 years at the time of enrollment.
* Patients must have newly diagnosed embryonal rhabdomyosarcoma (ERMS), spindle cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma (ARMS) (institutional FOXO1 fusion results are acceptable). RMS types included under ERMS include those classified in the 1995 International Classification of Rhabdomyosarcoma (ICR) as ERMS (classic, spindle cell, and botryoid variants), which are reclassified in the 2020 World Health Organization (WHO) classification as ERMS (classic, dense and botryoid variants) and spindle cell/sclerosing RMS (encompassing the historical spindle cell ERMS variant and the newly recognized sclerosing RMS variant). Enrollment in APEC14B1 is required for all patients.
* All patients will be evaluated for stage and clinical group. Note that clinical group designation assigned at the time of enrollment on study remains unchanged regardless of any second-look operation that may be performed.
* Patients will be eligible for the very low-risk stratum (Regimen VA) if they have Stage 1, CG I disease.
* Patients will be eligible for the low-risk stratum (Regimen VAC/VA) if they have Stage 1, CG II disease, Stage 2, CG I or II disease, or Stage 1, CG III (orbit only) disease.
* Paratesticular Tumors: Staging ipsilateral retroperitoneal lymph node sampling (SIRLNS) is required for all patients \>= 10 years of age with paratesticular tumors who do not have gross nodal involvement on imaging.
* Extremity Tumors: Regional lymph node sampling is required for histologic evaluation in patients with extremity tumors.
* Clinically or radiographically enlarged nodes must be sampled for histologic evaluation.
* Patients must have a Lansky (for patients =\< 16 years of age) or Karnofsky (for patients \> 16 years of age) performance status score of \>= 50. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing performance score.
* Peripheral absolute neutrophil count (ANC) \>= 750/uL (within 7 days prior to enrollment).
* Platelet count \>= 75,000/uL (transfusion independent) (within 7 days prior to enrollment).
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine (within 7 days prior to enrollment) based on age/gender as follows:
* Age: 1 month to \< 6 months; Maximum serum creatinine (mg/dL): 0.4 (male) : 0.4 (female)
* Age: 6 months to \< 1 year; Maximum serum creatinine (mg/dL): 0.5 (male) : 0.5 (female)
* Age: 1 to \< 2 years; Maximum serum creatinine (mg/dL): 0.6 (male) : 0.6 (female)
* Age: 2 to \< 6 years; Maximum serum creatinine (mg/dL): 0.8 (male) : 0.8 (female)
* Age: 6 to \< 10 years; Maximum serum creatinine (mg/dL): 1 (male) : 1 (female)
* Age: 10 to \< 13 years; Maximum serum creatinine (mg/dL): 1.2 (male) : 1.2 (female)
* Age: 13 to \< 16 years; Maximum serum creatinine (mg/dL): 1.5 (male) : 1.4 (female)
* Age \>= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male) : 1.4 (female)
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment), and
* If there is evidence of biliary obstruction by the tumor, then the total bilirubin must be \< 3 x ULN for age.
* Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L.
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L
* If there is evidence of biliary obstruction by the tumor, then the total bilirubin must be \< 3 x ULN for age
* Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
* All patients and/or their parents or legal guardians must sign a written informed consent.
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Exclusion Criteria:
* Patients who have received prior chemotherapy and/or radiation therapy for cancer prior to enrollment. Surgical resection alone of previous cancer(s) is permitted.
* Patients who have received chemotherapy or radiation for non-malignant conditions (e.g., autoimmune diseases) are eligible. Patients must discontinue chemotherapy for non-malignant conditions prior to starting protocol therapy.
* Vincristine is sensitive substrate of the CYP450 3A4 isozyme. Patients must not have received drugs that are moderate to strong CYP3A4 inhibitors and inducers within 7 days prior to study enrollment.
* Patients unable to undergo radiation therapy, if necessary, as specified in the protocol.
* Evidence of uncontrolled infection.
* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
* Lactating females who plan to breastfeed their infants.
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation. PROCEDURE: Biopsy Procedure, PROCEDURE: Bone Scan, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, BIOLOGICAL: Dactinomycin, PROCEDURE: Magnetic Resonance Elastography, PROCEDURE: Positron Emission Tomography, RADIATION: Radiation Therapy, DRUG: Vincristine
Embryonal Rhabdomyosarcoma, Fusion-Negative Alveolar Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma
Clinical Course Of Disease In Participants With FA-CM
Jaynee.Bartsch@cchmc.org
ALL
6 years and over
NCT06865482
Inclusion Criteria:
* Male or female, ages ≥6 years at the time of signing the informed consent (and assent, if applicable).
* Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on both alleles or compound heterozygous), with onset of FA occurring at ≤25 years of age
* Confirmed left ventricular hypertrophy (LVH)
* Left ventricular ejection fraction ≥30%
Exclusion Criteria:
* Presence of other form(s) of CM contributing to heart failure (HF), clinically significant cardiac anatomic abnormality or congenital cardiac malformation, clinically significant coronary artery, uncorrected, hemodynamically significant primary structural valvular disease not due to CM
* Currently receiving intermittent or continuous intravenous (IV) inotrope infusion, presence of a ventricular assist device, or history of prior heart transplantation
* Contraindication to cMRI, participants \<12 years of age who cannot complete the cMRI without sedation will instead undergo ECHOs and are exempt from this criterion.
* Prior organ transplantation
* Initiation of cardiac resynchronization therapy (CRT) within 6 months prior to screening.
* History of prior gene transfer or cell therapy.
* Poorly controlled diabetes (hemoglobin A1c ≥8%)
* Active hematologic or solid organ malignancy Friedreich Ataxia, Cardiomyopathy
Friedreich Ataxia, FA-CM, Cardiomyopathy, FA, Cardiac Disease
A Study of Eptinezumab in Pediatric Participants With Episodic Migraine (PROSPECT-1)
Morgan Thurza - morgan.thurza@cchmc.org
ALL
6 years to 17 years old
PHASE3
NCT05897320
Inclusion Criteria:
* Diagnosis of migraine (with or without aura) according to the International Classification of Headache Disorders, 3rd edition (ICHD-3; in the opinion of the investigator) with history of migraine headaches of at least 6 months prior to the Screening Visit.
* During the 28-day screening period, the participant (and their parent/caregiver, when applicable) must adequately complete the headache eDiary (≥23 of the 28 days) following the day of the Screening Visit.
* During the 28-day screening period, the participant must have ≤14 headache days, of which at least 4 are migraine days as documented in the eDiary.
Exclusion Criteria:
* History or diagnosis of chronic tension-type headache, hypnic headache, cluster headache, hemicrania continua, new daily persistent headache, or unusual migraine subtypes (previously referred to as complicated migraine), such as hemiplegic migraine (sporadic and familial), migraine with brainstem aura, recurrent painful ophthalmic neuropathy, or migraine with neurological accompaniments that are not typical of migraine aura (diplopia, altered consciousness, or long duration, e.g., \>60 min).
* History of moderate or severe head trauma or other neurological disorder or systemic medical disease that is, in the investigator's opinion, likely to affect the functions of the central nervous system.
* Current psychiatric condition that is uncontrolled and/or untreated for a minimum of 6 months prior to the Screening Visit. Participants with a lifetime history of psychosis and/or mania are excluded.
* Any other disorder for which the treatment takes priority over treatment of migraine or is likely to interfere with study treatment or impair treatment compliance.
Other inclusion and exclusion criteria may apply. DRUG: Eptinezumab, DRUG: Placebo
Episodic Migraine
Targeted Investigation of Microbiome 2 Treat Atopic Dermatitis (TIME-2)
Elsie Parmar - elsie.parmar@cchmc.org
ALL
6 years and over
PHASE1
NCT06504160
Inclusion Criteria:
Each individual must meet all of the following criteria at Screening to be eligible for enrollment as a study participant:
• Participant and/or parent/legal guardian must be able to understand and provide informed consent and assent (if applicable).
• Male or female participant 6 years of age or older.
• Meet ADRN Standard Diagnostic Criteria for active AD. Each individual must meet all of the following criteria at Baseline to be eligible for enrollment as a study participant:
• Have at least 7 cm2 of lesional skin within the upper extremities, lower extremities, and/or trunk. Lesions on the face, neck, hands, feet, and intertriginous areas do not count toward the required area, as samples may not be taken from these areas. The required area may be one contiguous area or may be comprised of multiple areas with a compliant total area.
• Have at least 3% body surface area of AD involvement as indicated by derived total area of involvement score during SCORAD assessment.
• Have an IGA score of two or greater.
• Each potential participant who can become pregnant must meet either of the following criteria prior to randomization to be eligible for enrollment as a study participant.
• Willing to remain abstinent from intercourse that may result in a pregnancy.
• Willing to use an FDA-approved method of contraception for the duration of study participation. Acceptable methods include the following: * Permanent sterilization of partner * Long-acting reversible contraceptives (e.g., intrauterine devices or systems, implantable rods, contraceptive injections) when used as directed for at least 7 days prior to Baseline. * Short-acting hormonal contraceptives (e.g., oral contraceptive pills, patch, vaginal ring) when used as directed for at least 30 days prior to Baseline * Barrier methods (e.g., condoms; diaphragm, sponge, or cervical cap with spermicide)
• Have obtained negative pregnancy test results during both the Screening and Baseline Visits.
Exclusion Criteria:
Individuals who meet any of the following criteria at Screening or Baseline are not eligible for enrollment as study participants:
• Inability or unwillingness to give written informed consent or comply with study protocol.
• Has self-reported as pregnant or lactating during the Screening or Baseline Visit, or is pregnant as indicated by a positive pregnancy test result obtained at the Screening or Baseline Visit.
• Sensitivity to or difficulty tolerating Dove® fragrance-free bar soap, Cetaphil® lotion, alcohol-based cleaners, clobetasol and fluocinonide ointments, triamcinolone ointment, hydrocortisone ointment, glycerol, hydroxyethylcellulose or soy products.
• Known recalcitrance to topical steroids, including class 1 steroids, within 6 months of the Screening Visit.
• History of serious life-threatening reaction to tape or adhesives.
• Known allergy to all antibiotics to which S. hominis A9 is sensitive. These include ampicillin-sulbactam, cefazolin, cefoxitin, clindamycin, daptomycin, doxycycline, levofloxacin, linezolid, minocycline, moxifloxacin, mupirocin, nitrofurantoin, oxacillin, rifampin, trimethoprim-sulfamethoxazole, and vancomycin.
• Has a major defect in the epidermal barrier such as open wounds or genodermatoses (e.g., Netherton's syndrome).
• Is immunocompromised (e.g., Human Immunodeficiency Virus (HIV)/Acquired Immunodeficiency Syndrome (AIDS), Wiskott-Aldrich Syndrome) or has an immune system disorder (e.g., autoimmune disease).
• Has current malignant disease (except non-melanoma skin cancer in an area not affected by treatment).
• Has a history of psychiatric disease or history of alcohol or drug abuse that, in the opinion of the study investigator, would interfere with the ability to comply with the study protocol.
• Ongoing participation in another investigational trial or use of investigational drugs within 8 weeks, or five half-lives (if known), whichever is longer, of the Screening Visit.
• Treatment with non-steroid systemic immunosuppressant within 6 months of the Screening Visit.
• Treatment with any biologic, including dupilumab, within 16 weeks of the Screening Visit.
• Treatment with oral or injectable therapy for AD (excluding oral steroids) within five half-lives (if known) or 16 weeks before the Screening Visit, whichever is longer.
• Treatment with oral retinoids (e.g., isotretinoin) within 60 days of the Screening Visit.
• Treatment with allergen immunotherapy within 30 days of Screening Visit.
• Has close contacts (e.g., spouse, children, or members in the same household) who have severe barrier defects or are immunocompromised.
• May, in the opinion of the investigator, have difficulty tolerating the medication washout requirements for topical AD treatments, prescription moisturizers, antibiotics, oral steroid therapies, and phototherapy ahead of Baseline.
• Past or current medical conditions or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study. Individuals who meet any of the following criteria at Baseline are not eligible for enrollment as study participants:
• Have more than 30% body surface area of AD involvement, as indicated by the derived total area of involvement score during SCORAD assessment.
• Active bacterial, viral, or fungal skin infections, except for onychomycosis and tinea pedis.
• Any noticeable breaks or cracks in the skin on the target areas of investigational product application, including severely excoriated skin or skin with open or weeping wounds suggestive of an active infection. a. At the investigator's discretion, participants with minor breaks, cracks, or excoriations on body areas not eligible for sampling during the trial may be enrolled, provided there is no evidence of infection and investigational product is not applied to these areas until healed.
• Use of topical AD treatments - including steroids and calcineurin inhibitors - on the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), or trunk within seven days of the Baseline Visit.
• Treatment with prescription moisturizers classified as medical device (e.g., Atopiclair®, MimyX®, Epiceram®, etc.) on the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), or trunk within seven days of the Baseline Visit.
• Use of any systemic microbialª within fourteen days of the Baseline Visit, or use of any topical antimicrobial on the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), or trunk within fourteen days of the Baseline Visit. a. Antimicrobials include antibiotics, antifungals, antiparasitics, and antivirals. Certain systemic antivirals that do not exhibit systemic anti-inflammatory effects may be permitted with prior approval from a protocol co-chair).
• Within the upper extremities (exclusive of the hands), lower extremities (exclusive of the feet), and trunk, use of topical products - prescription or over-the-counter, all formulations - not specified per protocol, within seven days of the Baseline Visit.
• Use of systemic corticosteroid therapies for any indication within 28 days of the Baseline Visit.
• Use of systemic corticosteroid therapies for treatment of an asthma exacerbation within 3 months of the Baseline Visit.
• Require a dose greater than 880 mcg/day of fluticasone propionate or equivalent inhaled corticosteroid to maintain asthma control, at the time of the Baseline Visit.
• Any phototherapy for skin disease (such as narrow band ultraviolet B \[NBUVB\], ultraviolet B \[UVB\], ultraviolet A1 \[UVA1\], psoralen + UVA \[PUVA\]) or regular use (more than 2 visits per week) of a tanning bed within 28 days of the Baseline Visit.
DRUG: ShA9 Topical Gel, DRUG: Hydrocortisone Ointment, DRUG: Clobetasol Ointment, DRUG: Fluocinonide Ointment, DRUG: Placebo (Vehicle) Topical Gel, DRUG: Triamcinolone Ointment
Atopic Dermatitis
atopic dermatitis, eczema, ADRN, TIME-2, microbiome, Staphylococcus hominis
Relapsed and Refractory Hepatoblastoma: A Prospective Registry and Liver Tumor Research Consortium Initiative
- rrHBLRegistry@cchmc.org
ALL
NCT05556642
Inclusion Criteria:
* All patients of any age with a suspected diagnosis (per treating oncologist/surgeon) or confirmed diagnosis of a rrHBL and all patients with Hepatocellular Malignant Neoplasm- Not Otherwise Specified (HCN-NOS) who are \<6 years of age at the time of initial diagnosis
* To allow for tumor modelling to be performed with fresh tissue from these cases, patients with suspected rrHBL are eligible to enroll on study
* Unless the patient is deceased, all patients and/or one parent or legal guardian must provide written informed consent as well as HIPAA/release of information consent
Exclusion Criteria:
* None OTHER: Biospecimen collection
Relapsed Hepatoblastoma, Refractory Hepatoblastoma
A Study of Fludarabine Dosing in Children and Young Adults With B-cell Acute Lymphoblastic Leukemia
cancer@cchmc.org
ALL
1 year and over
PHASE3
NCT07223021
Inclusion Criteria:
* Patients with B-ALL and eligible to receive commercial tisagenlecleucel.
* Patient's weight \> 9 kg at time of lymphodepleting chemotherapy
* Adequate organ function at time of LD is required and is defined:
* Hepatic: Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia
* Hepatic: AST and ALT \< 5x the upper limit of normal for age, unless thought to be leukemic disease-related
* Renal: Calculated glomerular filtration rate (GFR) ≥ 70 ml/min/1.73m\^2. (based on Schwartz formula GFR (mL/min/1.73 m²) = (36.2 × Height in cm) / Creatinine in mg/dL
* Cardiac: LVEF ≥ 50% by multi-gated acquisition scan (MUGA), resting echocardiogram, or cardiac magnetic resonance imaging (MRI) within 6 weeks of screening
* Pulmonary: Oxygen saturation as recorded by pulse oximetry of ≥ 90% on room air
* Adequate performance status:
* Age ≥ 16 years: ECOG ≤ 1 or Karnofsky \> 60% at treatment
* Age \< 16 years: Lansky ≥ 60% at treatment
* Willing to participate as research subject and provide written informed consent from parents/legal representative, patient, and age-appropriate assent as appropriate before any study specific screening procedures are conducted, according to local, regional or national law and legislation.
Exclusion Criteria:
* Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to the study drugs, or drugs chemically related to study treatment or excipients that contraindicate their participation, including fludarabine, cyclophosphamide and tisagenlecleucel.
* Patients with tisagenlecleucel that is deemed out of specification (OOS) will be excluded from this protocol
* Clinically significant active and uncontrolled infection confirmed by clinical evidence, imaging, or positive laboratory tests (e.g., blood cultures, PCR for DNA/RNA etc.)
* Patient/parent/guardian unable to give informed consent or unable to comply with the treatment protocol.
* Pregnant or lactating women DRUG: Fludarabine, DRUG: Cyclophosphamide, DRUG: Fludarabine, BIOLOGICAL: CAR-T
B-cell Acute Lymphoblastic Leukemia
Fludarabine
Study Evaluating the Safety and Efficacy of Neffy or Intramuscular Adrenalin in Patients With Allergic Reactions After Oral Food Challenge or Allergen Immunotherapy
- yasmin.hassoun@cchmc.org
ALL
4 years and over
PHASE4
NCT06834165
Inclusion Criteria:
* Is a patient 4 years old or greater, inclusive, who are scheduled to undergo OFC, AIT, or other relevant allergy challenge.
* Has body weight 15 kg or greater at the time of allergy challenge.
* Is willing and able to provide written informed consent prior to participating in the study. In the case of minors (\<18 years old), assent can be obtained from his/her legal representative, and as much possible from the patient himself/herself.
* Patient experiences an allergic reaction that, in the opinion of the Investigator, requires treatment with epinephrine via neffy or IM Adrenalin.
Exclusion Criteria:
\- Has any clinically significant medical condition that precludes treatment with epinephrine as assessed by the Investigator. DRUG: Neffy, DRUG: Adrenaline
Allergic Reactions
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis (STEP)
Abigail Abigail Torres - abigail.torres@cchmc.org
ALL
6 years to 19 years old
NCT05022459
Inclusion Criteria:
• Participant (if 18 years of age or older) or parent/LAR is willing and able to provide written informed consent; minor participant is willing and able to provide assent, if applicable based on site and local regulations
• Males and females between 6 to ≤ 19 years of age at time of enrollment with moderate to severe Hemophilia A (FVIII activity ≤ 5%) without inhibitors are eligible for participation in this study
• Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations
• Participants must be engaging in or registered to start participating in one or more sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating \>/= 2).
• Participant must be willing to keep activity, bleed, and treatment logs for the duration of the study
Exclusion Criteria:
• Participant/parent/LAR unwilling to provide informed consent/assent
• Unwilling to log or document bleeds and treatment information as per study guidelines
• Participants with any other bleeding disorders will be excluded
• Patients who are pregnant, planning to become pregnant, or breastfeeding should not be enrolled in the study
• Participants on concomittent FVIII replacement and emicizumab for sports participation
DRUG: Emicizumab, DRUG: FVIII
Hemophilia A
NRSTS2021, A Risk Adapted Study Evaluating Maintenance Pazopanib, Limited Margin, Dose-Escalated Radiation Therapy and Selinexor in Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS)
cancer@cchmc.org
ALL
Up to 30 years old
PHASE1
NCT06239272
Inclusion Criteria:
Inclusion Criteria - All Patients
* Patients must be 1-30 years at the time of the biopsy that established the diagnosis of NRSTS.
* Surgical Resection: Patients who had an upfront resection prior to enrollment will be eligible if they are able to begin therapy within 28 days of resection assuming other eligibility criteria are met. Delayed resection is preferred for all patients with intermediate and high-risk disease.
* Lansky performance status score ≥ 60 for patients ≤ 16 years of age. Karnofsky performance status score ≥ 60 for patients \>16 years of age. Note patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
Diagnosis
• Patients with CIC-DUX 4 rearranged sarcomas will be enrolled on the high-risk stratum only, regardless of presence of metastasis, size, or resection status.
Patient has low-risk disease if the patient has a:
* Low-grade tumor of any size where R0 or R1 surgical margins are anticipated or achieved.
* High-grade tumors that are \< 5 cm where R0 or R1 resection margins are anticipated or achieved.
Patient must have adequate organ function in the organs that will be within the radiotherapy field.
Adequate renal function defined as:
* Creatinine clearance or radioisotope GFR \> 70 mL/min/1.73 m2, or
* A normal serum creatinine based on age/gender as follows
Age Maximum Serum Creatinine (mg/dL) Male Female 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4 \> 16 years 1.5 1.4
Adequate liver function defined as:
* Total bilirubin \< 1.5 x upper limit of normal (ULN) for age
* SGOT (AST) or SGPT (ALT) \< 2.5 x ULN for age
Adequate cardiac function defined as:
* Ejection fraction of \> 55% by echocardiogram or cardiac MRI
* QTc \< 480 msec
Adequate pulmonary function defined as:
* No evidence of dyspnea at rest, no exercise intolerance, and a resting pulse oximetry reading \> 94% on room air if there is clinical indication for determination.
Inclusion Criteria - Intermediate and High Risk Participants
Patient has intermediate-risk if the patient has a:
* Low-grade non-metastatic initially unresectable disease at study enrollment where delayed resection is planned.
* High-grade \< 5 cm non-metastatic initially unresectable disease at study enrollment where delayed resection is planned. Of note, patients enrolled on the low-risk arm who were unable to achieve gross total resection where delayed re-resection is planned are eligible for this arm.
* High-grade tumor \> 5 cm that is potentially resectable.
Patient high-risk if the patient has:
* Metastatic disease at presentation
* Unresectable disease at study enrollment where delayed resection is not anticipated. Of note, patients enrolled on the low-risk arm who were unable to achieve gross total resection where delayed re-resection is not-planned are eligible for this arm.
* CIC-DUX4 rearranged sarcoma
Organ Function
Adequate bone marrow function defined as:
* Absolute neutrophil count \> 1000/µL
* Platelet count \> 100,000/µL
* Hemoglobin \> 8 g/dL for patients \< 16 years of age
* Hemoglobin \> 9 g/dL for patients \> 16 years of age
Note: No transfusions are permitted 7 days prior to laboratory studies to determine eligibility.
Adequate renal function defined as:
* Creatinine clearance or radioisotope GFR \> 70 mL/min/1.73 m2, or
* A normal serum creatinine based on age/gender as follows
Age Maximum Serum Creatinine (mg/dL) Male Female 2 to \< 6 years 0.8 0.8 6 to \< 10 years 1 1 10 to \< 13 years 1.2 1.2 13 to \< 16 years 1.5 1.4 \> 16 years 1.5 1.4
Adequate liver function defined as:
* Total bilirubin \< 1.5 x upper limit of normal (ULN) for age
* SGOT (AST) or SGPT (ALT) \< 2.5 x ULN for age
Adequate cardiac function defined as:
* Ejection fraction of \> 55% by echocardiogram or cardiac MRI
* QTc \< 480 msec
Adequate pulmonary function defined as:
* No evidence of dyspnea at rest, no exercise intolerance, and a resting pulse oximetry reading \> 94% on room air if there is clinical indication for determination.
Anticoagulation
Patients on low molecular weight heparin, warfarin (with a stable INR), or direct oral anticoagulants (DOAC) who have been on a stable dose of are eligible. Patients being treated for a pulmonary embolism or deep venous thrombosis (DVT) must have been treated for a minimum of 6 weeks prior to starting therapy treatment.
Life Expectancy
Patient must have a life expectancy of at least 3 months with appropriate therapy.
Exclusion Criteria:
* Patients with known primary CNS sarcoma or CNS metastases are not eligible. Note: Brain imaging is not an eligibility requirement. Tumors with intracranial extension will be allowed.
* Patients with the following histologic diagnosis are not eligible: intermediate locally aggressive tumors as defined by WHO, malignant rhabdoid tumor, alveolar soft part sarcoma, infantile fibrosarcoma, unresectable/metastatic dermatofibrosarcoma protuberans, inflammatory myofibroblastic tumor, desmoid fibromatosis, rhabdomyosarcoma, desmoplastic small round cell tumor, BCOR-CCNB3 fusion positive sarcoma.
* Bleeding diathesis: Patients with evidence of active bleeding or bleeding diathesis will be excluded (Note: Patients aged \> 17 years with excess of 2.5 mL of hemoptysis are not eligible).
* Uncontrolled hypertension: Patients with uncontrolled hypertension (CTCAE v5 Grade ≥ 2) are ineligible. Hypertension must be well controlled on stable doses of medication for at least two weeks.
Prior Therapy
* Patients must have had no prior systemic therapy for the treatment of the NRSTS
* Patients must have had no prior anthracycline or ifosfamide chemotherapy
* Patients must have had no prior use of pazopanib or similar multi-targeted TKI.
Patients must have had no prior radiotherapy to tumor-involved sites.
Note: Patients previously treated for a non-NRSTS cancer are eligible provided they meet the prior therapy requirements. Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier are excluded.
* CYP3A4 Substrates WITH Narrow Therapeutic Indices: Patients chronically receiving medications known to be metabolized by CYP3A4 and with narrow therapeutic indices within 7 days prior to study enrollment, including but not limited to pimozide, aripiprazole, triazolam, ergotamine and halofantrine are not eligible. Note: the use of fentanyl is permitted.
* CYP3A4 Inhibitors: Patients chronically receiving drugs that are known potent CYP3A4 inhibitors within 7 days prior to study enrollment, including but not limited to itraconazole, clarithromycin, erythromycin, many NNRTIs, diltiazem, verapamil, and grapefruit juice are not eligible.
* CYP3A4 Inducers: Patients chronically receiving drugs that are known potent CYP3A4 inducers within 14 days prior to study enrollment, including but not limited to carbamazepine, phenobarbital, phenytoin, rifampin, and St. John's wort are not eligible (with the exception of glucocorticoids).
* Certain medications that are associated with a risk for QTc prolongation and/or Torsade's de Pointes, although not prohibited, should be avoided or replaced with medications that do not carry these risks, if possible.
* Subjects with any condition that may impair the ability to absorb oral medications/investigational product including:
* prior surgical procedures affecting absorption including, but not limited to major resection of stomach or small bowel
* active peptic ulcer disease
* malabsorption syndrome
• 4.12 Thyroid Replacement Therapy: Patients who require thyroid replacement therapy are not eligible if they have not been receiving a stable replacement dose for at least 4 weeks prior to study enrollment.
• 4.13 Subjects with any condition that may increase the risk of gastrointestinal bleeding or gastrointestinal perforation, including: * active peptic ulcer disease * known intraluminal metastatic lesions * inflammatory bowel disease (e.g., ulcerative colitis, Crohn's disease) or other gastrointestinal conditions which increase the risk of perforation * history of abdominal fistula, gastrointestinal perforation or intra- abdominal abscess within 28 days prior to beginning study treatment.
• 4.14 Pulmonary embolism or DVT. Patients must not have experienced: * An untreated pulmonary embolism or DVT in last 6 months or * treated pulmonary embolism or DVT which has been treated with therapeutic anticoagulation for less than 6 weeks * arterial thrombosis in last 12 months History of serious or non-healing wound, ulcer, or bone fracture. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. HIV-positive subjects on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with pazopanib. In addition, these subjects are at increased risk of lethal infections when treated with marrow-suppressive therapy. Patients who are receiving any other investigational agent(s). Pregnancy and Breast Feeding * Pregnancy and Breast Feeding Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events as seen in animal/human studies. * Lactating females are not eligible unless they have agreed not to breastfeed their infants during treatment and for a period of 1 month following completion of treatment. * Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained. * Unwillingness to use an effective contraceptive method for the duration of their study participation and for at least 1 month after treatment is completed if sexually active with reproductive potential.
PROCEDURE: Surgical resection, RADIATION: Proton beam radiation therapy, DRUG: Pazopanib, DRUG: Ifosfamide, DRUG: Doxorubicin, DRUG: Selinexor
Adipocytic Neoplasm, Liposarcoma, Atypical Fibroxanthoma, Angiomatoid Fibrous Histiocytoma, Fibrosarcoma NOS, Myxofibrosarcoma, Angiosarcoma, Osteosarcoma, Extraskeletal, Dedifferentiated Liposarcoma, Myxoid Liposarcoma, Pleomorphic Liposarcoma, Myxoid Pleomorphic Liposarcoma, Low Grade Fibromyxoid Sarcoma, Sclerosing Epithelioid Fibrosarcoma, Malignant Tenosynovial Giant Cell Tumor of Soft Tissue, Epithelioid Hemangioendothelioma, Glomus Tumor, Inflammatory Leiomyosarcoma, Leiomyosarcoma, Ossifying Fibromyxoid Tumor, Malignant, Myoepithelioma, Synovial Sarcoma, Epithelioid Sarcoma, Perineurioma, Malignant, Clear Cell Sarcoma, Extraskeletal Myxoid Chondrosarcoma, Granular Cell Tumor, Malignant, Melanotic Malignant Nerve Sheath Tumor, Malignant Peripheral Nerve Sheath Tumor, Perivascular Epithelioid Tumor, Malignant, Intimal Sarcoma, Myoepithelial Carcinoma, Undifferentiated Sarcoma, Pleomorphic Sarcoma, Undifferentiated, Round Cell Sarcoma, Undifferentiated, NTRK-Rearranged Spindle Cell Neoplasm, Phosphaturic Mesenchymal Tumor, Malignant, Round Cell Sarcoma, Well Differentiated Liposarcoma, Giant Cell Tumor of Soft Parts NOS, Low Grade Myofibroblastic Sarcoma, Dermatofibrosarcoma Protuberans, Fibrosarcomatous
Proton therapy
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome (ARGUS)
Samantha Ballard - samantha.ballard@cchmc.org
ALL
2 years and over
PHASE3
NCT04462770
Key
• Male and female participants 2 years and older at time of consent.
• Participant or parent/legally authorized representative (LAR) willing and able to provide written informed consent and assent (if applicable) prior to initiation of any study related procedures.
• Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the following criteria: * Onset of seizures prior to 18 months of age, * Normal development at onset, * History of at least one type of countable motor seizure (CMS), * Brain MRI without cortical malformation (not including mild atrophy associated with the natural progression of DS), * Genetic mutation of the SCN1A gene must be documented. Key
• Known sensitivity, allergy, or previous exposure to clemizole HCl.
• Exposure to any investigational drug or device \<90 days prior to screening or plans to participate in another drug or device trial at any time during the study.
• Seizures secondary to illicit drug (this includes concomitant use of tetrahydrocannabinol \[THC\] and nonprescription cannabidiol preparations) or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease.
• Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
• Concurrent use of fenfluramine.
• Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
Inclusion Criteria:
• Male and female participants 2 years and older at time of consent.
• Participant or parent/legally authorized representative (LAR) willing and able to provide written informed consent and assent (if applicable) prior to initiation of any study related procedures.
• Clinical diagnosis of DS. Participants must have seizures which are not completely controlled by AEDs with the following criteria: * Onset of seizures prior to 18 months of age, * Normal development at onset, * History of at least one type of countable motor seizure (CMS), * Brain MRI without cortical malformation (not including mild atrophy associated with the natural progression of DS), * Genetic mutation of the SCN1A gene must be documented. Key
Exclusion Criteria:
• Known sensitivity, allergy, or previous exposure to clemizole HCl.
• Exposure to any investigational drug or device \<90 days prior to screening or plans to participate in another drug or device trial at any time during the study.
• Seizures secondary to illicit drug (this includes concomitant use of tetrahydrocannabinol \[THC\] and nonprescription cannabidiol preparations) or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease.
• Concurrent use of lorcaserin. Note: Prior use of lorcaserin is permitted if at least 30 days have passed since the last dose.
• Concurrent use of fenfluramine.
• Epilepsy surgery planned during the study or epilepsy surgery within 6 months prior to Screening.
DRUG: Clemizole HCl, DRUG: Placebo
Dravet Syndrome
Clemizole hydrochloride, Convulsive seizure, Pediatric epilepsy, Dravet syndrome
The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study
- cancer@cchmc.org
ALL
Up to 22 years old
PHASE1
NCT04726241
Inclusion Criteria:
* Patients must be less than 22 years of age at the time of study enrollment
* Patient must have one of the following at the time of study enrollment:
* Patient has known or suspected relapsed/refractory (including primary refractory) AML as defined in protocol
* This includes isolated myeloid sarcoma
* Patient has known or suspected relapsed/refractory (including primary refractory) myeloid leukemia of Down syndrome (ML-DS)
* Patient has known or suspected relapsed ALL as defined in protocol that meets one of the following criteria:
* Second or greater B-ALL medullary relapse, excluding KMT2Ar
* Any first or greater B-ALL medullary relapse involving KMT2Ar
* Any first or greater T-ALL medullary relapse with or without KMT2Ar
* Patient has known or suspected relapsed/refractory (including primary refractory) mixed phenotype acute leukemia (MPAL) as defined in protocol
* Patient has known or suspected de novo or relapsed/refractory (including primary refractory) treatment-related AML (t-AML)
* Patient has known or suspected de novo or relapsed/refractory (including primary refractory) myelodysplastic syndrome (MDS) or treatment-related myelodysplastic syndrome (t-MDS)
* Note: Relapsed/refractory disease includes stable disease, progressive disease, and disease relapse.
* Patient has known or suspected de novo or relapsed/refractory (including primary refractory) juvenile myelomonocytic leukemia (JMML)
* Note: Relapsed/refractory disease includes stable disease, progressive disease, and disease relapse.
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met PROCEDURE: Biospecimen Collection
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Juvenile Myelomonocytic Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome Post Cytotoxic Therapy, Myeloid Leukemia Associated With Down Syndrome
Improving ADHD Teen Driving - Virtual Reality
- ADHDdriving@cchmc.org
ALL
16 years to 19 years old
NA
NCT06960980
Inclusion Criteria:
• Aged 16-19.
• Teens will meet DSM ADHD criteria for ADHD-Predominantly Inattentive Presentation or ADHD-Combined Presentation based on the K-SADS interview.
• Possess a valid driver's license and regularly spend at least 3 hours per week engaged in unsupervised driving.
• IQ ≥80 as measured by the Kauffman Brief Intelligence Scale - Second Edition (KBIT-2).
• Parent willing to participate.
Exclusion Criteria:
• On ADHD medication that cannot be washed out on assessment days.
• Drug or alcohol dependence based on self-report on the Simple Screening Instrument for Alcohol and Other Drugs survey.
• On psychotropic or neuroleptic medications.
• At-risk for motion sickness in the driving simulator or in virtual reality.
• History of moderate to severe head trauma, neurological disorder, or any other organic disorder that could possibly affect brain function.
• Cannot see the secondary task stimuli without the use of glasses (contacts acceptable).
BEHAVIORAL: FOCAL+, BEHAVIORAL: VR-FOCAL+
Attention Deficit Hyperactivity Disorder (ADHD)
Visual attention, Automobile Driving
A Study of Codrituzumab in Children and Young Adults With Solid Tumors and Have Not Responded to Treatment or Have Come Back After Treatment
- cancer@cchmc.org
ALL
1 year to 21 years old
PHASE1
NCT04928677
Inclusion Criteria:
* Consent/Assent: All patients and/or their parents or legally authorized representatives must sign written informed consent; assent, when appropriate, will be obtained according to institutional guidelines.
* Age: Patients must be aged 12 months up to but not including 22 years at the time of study registration. A patient who is 21 at the time of enrollment but turns 22 thereafter will still be considered eligible for the purposes of this study.
Diagnosis:
* Patients must have a diagnosis of a primary extra-cranial solid tumor that is recurrent or refractory to standard therapy.
* For the purposes of this study, the following definitions will be used:
* Refractory is defined as any tumor which progresses despite maximal standard therapies
* Recurrent (relapsed) is defined as a completion of planned therapy after which point the tumor recurs within 5 years of treatment. Additionally, any tumor which recurs twice is considered relapsed.
* Tumor GPC3 Expression: Patients must have demonstrated positive GPC3 expression via immunohistochemistry (IHC) on any prior tumor sample. Confirmation of GPC3 expression may include a diagnostic or relapsed sample, at a primary or metastatic site. There is no limit to how long ago this sample was collected. This GPC3 expression via IHC will be centrally confirmed by Ventana as in prior studies of codrituzumab and described in the treatment. Patients may choose to enroll on the prescreening portion, which allows for assessment of GPC3 expression only, prior to enrollment on the full clinical trial.
* Tumor GPC3 Expression: Patients must have demonstrated a minimum of 1+ GPC3 expression via immunohistochemistry (IHC) on any prior tumor sample. Confirmation of GPC3 expression may include a diagnostic or relapsed sample, at a primary or metastatic site. There is no limit to how long ago this sample was collected. This GPC3 expression via IHC will be centrally confirmed by Ventana as in prior studies of codrituzumab and described in the treatment plan section. Patients may choose to enroll on the prescreening portion, which allows for assessment of GPC3 expression only, prior to enrollment on the full clinical trial.
* Disease Status: Patients must have measurable disease based on RECIST 1.1.
* Performance Level: Patients must have Karnofsky Performance Score (for patients \> 16 years of age) or Lansky Performance Score (for patients ≤ 16 years of age) ≥ 50% assessed within 2 weeks of study enrollment.
* Neurological Deficits: Patients with neurologic deficits must have been stable and off of steroids for a minimum of 1 week prior to study entry; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
* Pregnancy/Contraception: Patients must not be pregnant or breast-feeding; females, excluding pre-menstrual, must have a negative serum or urine pregnancy test within 7 days prior to enrollment; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method, which includes abstinence, for 90 days after the last dose of study drug.
* Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study; recovery is defined as all AEs, attributable to prior therapy, having improved to grade 2 or better or as outlined below. For agents that have known adverse events occurring beyond 28 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair.
* Systemic Anticancer Therapy (e.g. Chemotherapy):
* Not Myelosuppressive: \> 7 days must have elapsed from their last dose of anticancer therapy not known to be myelosuppressive (e.g. not associated with reduced platelet or ANC counts).
* Myelosuppressive: \> 14 days must have elapsed from their last dose of known myelosuppressive anticancer therapy.
* Antibodies: \> 21 days must have elapsed from the infusion of last anticancer antibody.
* Cellular Therapies: \> 42 days must have elapsed from the completion of any type of cellular therapy, including modified T cells, NK cells, dendritic cells, etc.
* Radiation: Patients who have had radiation must have had their last fraction of:
* Local irradiation to the primary tumors or other limited sites (cumulative dose \< 40Gy) \> 14 days prior to registration.
* Local irradiation to the primary tumors or other sites (cumulative dose ≥ 40Gy), therapeutic 131I-MIBG or other radiopharmaceutical, and other substantial bone marrow radiation, \> 42 days prior to registration.
* Craniospinal irradiation, radiation to \> 50% of pelvis, or total body irradiation \> 120 days prior to registration.
* Stem Cell Infusions: With or without TBI
* 84 days must have elapsed from an allogeneic bone marrow or stem cell transplant, or any stem cell infusion including DLI or boost infusion and patients must also not have any evidence of acute or chronic GvHD.
* 42 days must have elapsed from an autologous stem cell infusion including boost infusion.
* Supportive Therapies: Patients must be off all growth factor(s) that support platelet, red blood cell, or white blood cell count, number or function for at least 7 days prior to registration (e.g. filgrastim, sargramostim, erythropoietin, romiplostim, eltrombopag, etc.) as well as off Pegylated G-CSF for at least 14 days prior to registration.
* Organ Function: Patients must have documented within 14 days of registration and within 7 days of starting treatment the following:
* Hgb \> 8 gm/dL (may be transfusion-supported)
* Platelet count \> 50,000/mm\^3 (transfusion independent)
* Absolute neutrophil count (ANC) \> 1000/mm\^3
* INR ≤ 2.5
* Total Bilirubin (sum of conjugated + unconjugated) ≤ 3 times institutional upper limit of normal (ULN) for age
* Aspartate aminotransferase (AST) ≤ 5 times institutional ULN for age
* Alanine Aminotransferase (ALT) ≤ 5 times institutional ULN for age
* Serum albumin ≥ 2 g/dL
* GFR ≥ 50 mL/min/1.73 m\^2 as measured using urine creatinine clearance, serum cystatin c, radioisotope GFR, or serum creatinine as measured by the Schwartz equation (Refer to Schwartz, et al. J Am Soc Neph, 2009)
Exclusion Criteria:
* Patients receiving current anti-cancer therapy or investigational agents are not eligible for study entry.
* Patients who do not have tumor tissue available for GPC3 testing are not eligible for study entry.
* Patients who have received any prior GPC3-directed immunotherapy are not eligible for study entry.
* Patients with uncontrolled seizures are not eligible for study entry.
* Subjects with a condition requiring systemic treatment with either corticosteroids (\>0.15 mg/kg daily prednisone equivalents) or other immunosuppressive medications, if used to modify immune adverse events related to prior therapy, \> 14 days must have elapsed since last dose of corticosteroid or immunosuppressive agent. Inhaled or topical steroids, and adrenal replacement doses ≤ 0.15 mg/kg daily prednisone equivalents are permitted in the absence of active autoimmune disease.
* Patients with documented CNS tumor, CNS metastasis, CNS ischemia and/or infarction, whether symptomatic or discovered incidentally without clinical symptoms, will be excluded from study participation.
* Patients with a baseline QTc \> 480. (as measured using Bazett formula; Refer to Bazett, Heart, 1920).
* Patients with an inability to return for follow-up visits, obtain follow-up studies required to assess toxicity to therapy, or comply with the safety monitoring requirements.
* Patients who have an uncontrolled infection are not eligible.
* Patients who have received a prior solid organ transplantation are not eligible. DRUG: Codrituzumab
Primary Extra-cranial Solid Tumor, Recurrent or Refractory Glypican 3 (GPC3)
Codrituzumab, Hepatoblastoma, 20-489
Animal Assisted Therapy After Pediatric Brain Injury: Mediators and Moderators of Treatment Response.
Megan Narad - megan.narad@cchmc.org
ALL
4 years to 24 years old
NA
NCT05250180
Inclusion Criteria:
* Diagnosis: Participants must be admitted to the inpatient rehabilitation unit for treatment of an acquired brain injury (TBI, brain tumor, infection, etc).
* Consent: The family must provide informed consent by parents or legal guardians.
* Assent: The child/adolescent must provide a signature indicating assent to participate in the study.
* Age at the time of screening: 4-21 years old
* Sex: includes both males and females
* Responsiveness: Rancho score greater than 2 at the time of enrollment (as noted in the medical chart).
Exclusion Criteria:
* Allergies: Patient has a significant allergy to dogs
* Fear: Patient has a significant fear of dogs
* Disease: Participant is on contact precautions or has a communicable disease that may pose a risk to the dog or dog handler or has a compromised immune system where interacting with the dog and/or handler would be of significant risk to the patient.
* Medical History: History of developmental delay prior to ABI.
* Behavioral History: Participant or family has a history of animal abuse or cruelty.
* Responsiveness: Rancho score of 2 or less OTHER: Animal Assisted Therapy, OTHER: Control
Brain Injuries
Pediatric brain injury, Animal assisted therapy, inpatient rehabilitation
Prenatally-initiated Psychological Intervention for Mothers of Infants With Congenital Heart Disease (CHD)
Stephanie Lynch, MPH - stephanie.lynch@carelon.com
ALL
18 years and over
NA
NCT07582861
Both mothers and infants are study participants.
• Pregnant individual carrying a fetus diagnosed with CHD or biological mother of an infant diagnosed with CHD in the neonatal period.
• Fetus or newborn with CHD anticipated to require cardiac surgery or transcatheter intervention in the first 30 days of life.
• CHD diagnosis received between 16.0 and 30.0 weeks gestation.
• Singleton pregnancy.
• Pregnant mother is planning to continue the pregnancy and pursue surgical or transcatheter intervention for the infant. 5\. Pregnant mother is able to participate and complete study assessments in English or Spanish.
• Mother, fetal, or infant medical condition determined by a treating clinician to be contraindicative to study participation.
• Mother with a severe, untreated psychiatric condition, substance use disorder, or other circumstances that, in the opinion of the investigator, would interfere with engagement with study tasks or safe participation in the trial.
• Mother with a moderate to severe intellectual disability or is otherwise unable to provide informed consent.
• Postnatal diagnosis of CHD.
• Fetus with a comorbid condition with a predictable and major impact on neurodevelopment (e.g., Trisomy 21, DiGeorge syndrome).
• Pregnant individual is aged \<18 years.
• Pregnant individual is carrying a surrogate pregnancy.
• Consented mother completes \<70% of survey questions at first (baseline) assessment.
Inclusion Criteria:
• Pregnant individual carrying a fetus diagnosed with CHD or biological mother of an infant diagnosed with CHD in the neonatal period.
• Fetus or newborn with CHD anticipated to require cardiac surgery or transcatheter intervention in the first 30 days of life.
• CHD diagnosis received between 16.0 and 30.0 weeks gestation.
• Singleton pregnancy.
• Pregnant mother is planning to continue the pregnancy and pursue surgical or transcatheter intervention for the infant. 5\. Pregnant mother is able to participate and complete study assessments in English or Spanish.
Exclusion Criteria:
• Mother, fetal, or infant medical condition determined by a treating clinician to be contraindicative to study participation.
• Mother with a severe, untreated psychiatric condition, substance use disorder, or other circumstances that, in the opinion of the investigator, would interfere with engagement with study tasks or safe participation in the trial.
• Mother with a moderate to severe intellectual disability or is otherwise unable to provide informed consent.
• Postnatal diagnosis of CHD.
• Fetus with a comorbid condition with a predictable and major impact on neurodevelopment (e.g., Trisomy 21, DiGeorge syndrome).
• Pregnant individual is aged \<18 years.
• Pregnant individual is carrying a surrogate pregnancy.
• Consented mother completes \<70% of survey questions at first (baseline) assessment.
OTHER: HeartGPS
Congenital Heart Disease (CHD)
HeartGPS, CHD, intervention, Parent mental health, Neurodevelopment