StudyFinder
Search Results
345 Study Matches
Optimal Ventilation for Cardiac Arrest (OPTI-VENT)
Daniel Loeb - Daniel.Loeb@cchmc.org
ALL
37 weeks to 18 years old
NA
NCT07114510
Inclusion Criteria:
* Invasive airway in place at the start of CPR or airway placed within the first 5 minutes
* Received at least 1 minute of CPR.
Exclusion Criteria:
* Lack of commitment to aggressive ICU therapies (e.g., CPR performed as part of end-of-life care.
* Brain death determination prior to the CPR event.
* Out-of-hospital cardiac arrest was the reason for initial admission to the hospital (known poor outcomes).
* Supported by Veno-Arterial Extra Corporeal Membrane Oxygenation at the start of CPR OTHER: OPTI-VENT Bundle, OTHER: Transition, OTHER: None - control
Cardiac Arrest (CA)
Pediatric
Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
513-636-2799 - cancer@cchmc.org
ALL
12 years to 39 years old
PHASE2
NCT04616560
Inclusion Criteria:
* Phase 1 (Part A): Patients must be at least 2 years and less than 12 years of age at the time of study enrollment
* Phase 2: Wilms tumor patients (Part B1): All Wilms tumor patients enrolled must be less than 18 years of age at enrollment
* Until the completion of the Phase 1 dose confirmation, patients must be at least 12 years of age and less than 18 years of age at the time of study enrollment
* Following dose confirmation of DS-8201a in children at least 2 to less than 12 years old in the Phase 1 component, Wilms tumor patients at least 2 to less than 18 years of age will be allowed on the Phase 2 component
* Phase 2: DSRCT patients (Part B2): Until the completion of the Phase 1 component, patients enrolling on the Phase 2 component of the study must be from at least 12 to 39 years of age at the time of study enrollment
* Following dose confirmation of DS-8201a in children at least 2 to less than 12 years old in the Phase 1 component, DSRCT patients at least 2 to 39 years of age will be allowed on the Phase 2 component
* Patients must have had histologic verification of Wilms tumor or desmoplastic small round cell tumor at original diagnosis or relapse
* Solid tumors: Patients must have measurable disease according to Response Evaluation Criteria in Solid Tumors (RECIST) 1.1. Patients with clinically inactive brain metastases may be included in the study. Patients with treated brain metastases that are no longer symptomatic and who require no treatment with corticosteroids or anticonvulsants may be included in the study if they have recovered from the acute toxic effect of radiotherapy. Lastly, patient must have unresectable lesions or lesions with no intention to surgically remove the lesions in the 6 months following enrollment
* Wilms tumor: WT patients must have either refractory disease or a very high risk relapse, defined as ANY of the following:
* Relapse after initial treatment with 4 or more chemotherapy agents (e.g. Regimens vincristine, dactinomycin, doxorubicin, cyclophosphamide, etoposide and radiation \[M\], vincristine, dactinomycin and doxorubicin, vincristine, and irinotecan \[MVI\], doxorubicin, vincristine, cyclophosphamide, carboplatin, etoposide and radiation \[UH-1\], doxorubicin, vincristine, cyclophosphamide, carboplatin, etoposide, vincristine, and irinotecan \[UH-2\], vincristine, irinotecan, cyclophosphamide, carboplatin, etoposide and doxorubicin \[UH-3\], and etoposide, carboplatin, cyclophosphamide, and doxorubicin \[HR-1\])
* Relapse with high risk histology (anaplasia, blastemal predominant)
* Multiple relapses
* Desmoplastic small round cell tumor: DSRCT patients with relapsed or refractory disease are eligible
* Patient's current disease state must be one for which they have received at least standard initial therapy, defined as systemic therapy combined with either radiation or surgery for local control of the primary tumor at diagnosis. Prior therapy after relapse is not required
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0 or 1. Use Karnofsky for patients older than 16 years of age and Lansky for patients 16 years of age and younger
* Patients must have 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: For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned Research Coordinator prior to enrollment
* \>= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
* Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil count \[ANC\] counts): \>= 7 days after the last dose of agent
* Antibodies: \>= 4 weeks (28 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
* 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: \>= 30 days after the completion the infusion 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: \>= 4 weeks (28 days) including palliative radiation therapy to the chest. \>= 14 days after palliative local XRT to areas other than the chest or for whole brain radiotherapy
* Radiopharmaceutical therapy (e.g., radiolabeled antibody, samarium): \>= 42 days after systemically administered radiopharmaceutical therapy
* Patients must not have received prior HER2 therapies including antibody drug conjugates (e.g. TDM-1 or DS-8201a), HER2 directed cellular therapies, HER2 receptor therapy (e.g. trastuzumab, pertuzumab, margetuximab, zanidatamab, zenocutuzumab) or small molecule antagonists of HER2 (e.g lapatinib, tucatinib, or neratinib). Prior exposure to antibody drug conjugates which do not target HER2 as well as prior treatment with topoisomerase 1 inhibitors (e.g. irinotecan, topotecan) are permitted
* Patients must be at least 14 days from the date of last surgery
* Peripheral absolute neutrophil count (ANC) \>= 1000/uL, (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) (for patients with solid tumors without known bone marrow involvement)
* Hemoglobin \>= 8.0 g/dL at baseline (Red Blood Cell transfusion is not allowed within 1 week prior to enrollment) (for patients with solid tumors without known bone marrow involvement)
* For patients less than or equal to 17 years old, "Bedside" Schwartz formula (2009)
* Estimated glomerular filtration rate (GFR) (eGFR) ≥ 70 mL/min/1.73 m\^2 (\> 70mL/min/1.73 m\^2 for patients \> 17 years old)
* For patients older than 17 years of age the Cockroft-Gault equation should be utilized to calculate creatinine clearance ≥ 70 ml/min:
* OR for any age group:
* A 24 hour urine creatinine clearance ≥ 70 mL/min/1.73 m\^2 (\> 70mL/min for patients ≥ 17 years old) OR
* A directly measured GFR ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
* Bilirubin (sum of conjugated + unconjugated or total) =\< 1.5 x upper limit of normal (ULN) for age. For patients with documented Gilbert's syndrome (unconjugated hyperbilirubinemia) bilirubin must be \< 3 x ULN for age (patients with solid tumors)
* Aspartate aminotransferase (AST) =\< 3 x ULN
* Serum albumin \>= 2.5 g/dL (patients with solid tumors)
* International normalized ratio (INR)/prothrombin time (PT) =\< 1.5 x ULN. Exception for patients receiving coumarin-derivative anticoagulants or other similar anticoagulant therapy, who must have INR/PT within the therapeutic range as deemed appropriate by the investigator
* Shortening fraction of \>= 27% by echocardiogram, or ejection fraction of \>= 50% by either an echocardiogram (ECHO) or multigated acquisition (MUGA) scan within 28 days before Step 1 enrollment
* Corrected QT interval (QTc) prolongation to \< 480 ms based on average triplicate 12-lead electrocardiogram (ECG)
* Pulse oximetry \> 93% on room air
* Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled as evidenced 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 chemotherapy, surgery, and/or radiation must be =\< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months of enrollment are eligible
* All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines
Exclusion Criteria:
* Pregnant, planning to become pregnant, or breast-feeding women will not be entered on this study. 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 and upon completion of the study and for at least 7 months for females and 4 months for males after the last dose of study drug. Abstinence is an acceptable method of birth control
* Methods considered as highly effective methods of contraception include:
* Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
* Oral
* Intravaginal
* Transdermal
* Progestogen-only hormonal contraception associated with inhibition of ovulation:
* Oral
* Injectable
* Implantable
* Intrauterine device (IUD)
* Intrauterine hormone-releasing system (IUS)
* Bilateral tubal occlusion
* Vasectomized partner
* Complete sexual abstinence defined as refraining from heterosexual intercourse. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception
* Non-child-bearing potential defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea (in questionable cases, a blood sample with simultaneous follicle-stimulating hormone \[FSH\] \> 40 mIU/mL and estradiol \< 40 pg/mL \[\< 147 pmol/L\] is confirmatory). Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the contraception methods outlined for women of child-bearing potential if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal status prior to study enrollment. For most forms of HRT, at least 2-4 weeks will elapse between the cessation of therapy and the blood draw; this interval depends on the type and dosage of HRT. Following confirmation of their post-menopausal status, they can resume use of HRT during the study without use of a contraceptive method
* Male patients must not freeze or donate sperm starting at enrollment and throughout the study period, and at least 4 months after the final study drug administration. Preservation of sperm should be considered prior to enrolment in this study
* Female patients must not donate, or retrieve for their own use, ova from the time of enrollment and throughout the study treatment period, and for at least 7 months after the final study drug administration
* 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
* 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 chloroquine or hydroxychloroquine within 14 days are not eligible for this trial
* Patients who received a live, attenuated vaccine (messenger ribonucleic acid \[mRNA\] and replication deficient adenoviral vaccines are not considered attenuated live vaccines) within 30 days prior to enrollment are not eligible for this trial
* Note: Participants, if enrolled, should not receive live vaccines during the study and up to 90 days after the last dose of study intervention. It is recommended that patients receive a yearly influenza killed vaccination and additional killed vaccinations based on local or national recommendations. Consider vaccination against viral pathogens that cause pneumonias according to local or national guidelines
* Patients who have received a prior solid organ transplantation are not eligible
* Patients with a medical history of myocardial infarction within 180 days before enrollment, symptomatic congestive heart failure (CHF) (New York Heart Association Class II to IV) or troponin levels consistent with myocardial infarction as defined according to the manufacturer 28 days prior to enrollment are not eligible
* Additionally, patients with a history of any of the following congenital heart disease are not eligible:
* Single ventricle heart defects (hypoplastic left heart syndrome, unbalanced atrioventricular septal defects, double inlet left ventricle, tricuspid atresia, the presence of superior cavopulmonary anastomosis or Fontan palliation);
* Unpalliated defects with significant hemodynamic alterations or palliated lesions with residual hemodynamic alterations (ductal-dependent or shunt-dependent physiology, large unrestrictive ventricular septal defect, transposition of the great arteries, greater than moderate atrioventricular valve insufficiency, moderate or greater aortic valve stenosis, moderate or greater aortic valve insufficiency, large atrial septal defects with significant right ventricular volume overload, large patent ductus arteriosus)
* Patients who have a pleural effusion, ascites or pericardial effusion that requires drainage, peritoneal shunt, or cell-free and concentrated ascites reinfusion therapy (CART) are not eligible. (Drainage and concentrated ascites reinfusion therapy are not allowed within 2 weeks prior to enrollment)
* Patients who have spinal cord compression or clinically active central nervous system metastases, defined as untreated and symptomatic, or requiring therapy with corticosteroids or anticonvulsants to control associated symptoms are not eligible
* Patients with a known history of severe hypersensitivity to DS-8201a, any excipient contained in the DS-8201a drug formulation, or HER2-targeted monoclonal antibodies (trastuzumab, pertuzumab, margetuximab) are not eligible
* Patients who have an uncontrolled infection or non-healing surgical site are not eligible
* Patients with a known history of substance abuse or any other clinically significant medical conditions (i.e. psychological conditions) that may, in the opinion of the investigator, interfere with the patient's participation in the clinical study or evaluation of the clinical study results are not eligible
* Patients who have pulmonary compromise, ex hypoxia, resulting from intercurrent pulmonary illnesses including, but not limited to, any underlying pulmonary disorder (i.e. pulmonary emboli within three months of the study enrollment, severe asthma, severe chronic obstructive pulmonary disease (COPD), restrictive lung disease, pleural effusion etc.), or prior pneumonectomy are not eligible
* Patients who have a history of (non-infectious) ILD (interstitial lung disease)/pneumonitis that required steroids, has current ILD/pneumonitis, or for whom suspected ILD/pneumonitis cannot be ruled out by imaging at screening are not eligible. Patients who have history of genetic disorders of the lung (i.e. cystic fibrosis 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
* Patients with known hepatitis B or C with detectable viral load are not eligible
* Patients with any autoimmune, connective tissue or inflammatory disorders (e.g., rheumatoid arthritis, Sjogren's, sarcoidosis etc.) where there is documented, or a suspicion of pulmonary involvement at the time of enrollment or genetic diseases involving the lung are not eligible
* Patients with an active primary immunodeficiency are not eligible PROCEDURE: Biospecimen Collection, PROCEDURE: Computed Tomography, PROCEDURE: Echocardiography Test, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Multigated Acquisition Scan, BIOLOGICAL: Trastuzumab Deruxtecan
Desmoplastic Small Round Cell Tumor, Osteosarcoma, Recurrent Desmoplastic Small Round Cell Tumor, Recurrent Kidney Wilms Tumor, Recurrent Osteosarcoma, Refractory Desmoplastic Small Round Cell Tumor, Refractory Wilms Tumor, Wilms Tumor
TIDES 2.0: Prevalence and Longitudinal Course of Depression, Anxiety, and Behavior Problems in Children With Cystic Fibrosis Under 12 Years of Age (TIDES 2)
Stephanie Filigno, PhD - stephanie.filigno@cchmc.org
ALL
18 months to 11 years old
NCT07048574
Inclusion Criteria:
• Child with a diagnosis of Cystic fibrosis (CF) actively followed by the CF care team at a participating site
• Child is age 18 months thru 11 years
• English and/or Spanish speaking
• Parent/legal guardian willing and able to give informed consent, and for minor participants ages 7 thru 11 years able to give assent.
Exclusion Criteria:
* Unable or unwilling to participate in study procedures, or at Site PI discretion. Cystic Fibrosis (CF)
Cystic Fibrosis, Children, Mental health screening, Depression, Anxiety, CFTR modulators, Neuropsychiatric adverse events
A Tailored Medication Adherence-Promotion Intervention for Adolescents and Young Adults With Cancer
- cancer@cchmc.org
ALL
15 years to 24 years old
NA
NCT07223463
Inclusion Criteria:
* Patient is 15.00 to 24.99 years of age
* Patient is diagnosed with cancer
* Patient is prescribed an oral anticancer/antitumor agent or prophylaxis
Exclusion Criteria:
* Patient is not fluent in English
* Patient evidences significant cognitive deficits
* Patient's medical status or treatment precludes participation
* Patient enrolled on a medical trial requiring medication storage in a trial-provided container
* Patient demonstrates greater than or equal to 95% adherence during run-in period
* Patient declines to use or has difficulty using electronic adherence monitoring device during run-in BEHAVIORAL: Tailored Program, BEHAVIORAL: Feedback Program (Uniform Standard of Care)
Cancer, Medication Adherence
adolescents and young adults, cancer, adherence
Feto-Endoscopic Tracheal Occlusion (FETO) for Severe Congenital Diaphragmatic Hernia (FETO)
Sandi Bechtol - sandra.bechtol@cchmc.org
FEMALE
18 years to 50 years old
NA
NCT02986087
Inclusion Criteria:
* Isolated CDH with liver up
* Severe pulmonary hypoplasia with ultrasound O/E LHR \<25% at the time of surgery
* Gestational age at FETO procedure 27 weeks 0 days to 29 weeks 6 days
* Moderate pulmonary hypoplasia with ultrasound O/E LHR \<30% and liver-up at the time of surgery
* Gestational age at FETO procedure 30 weeks 0 days to 31 weeks 6 days in this moderate category
* Maternal age greater than or equal to 18 years
* Gestational age at enrollment prior to 29 weeks 6 days, or 31 weeks 6 days in moderate category
* Normal karyotype or FISH
* Normal fetal echocardiogram
* Singleton pregnancy
* Willing to remain in the greater Cincinnati area for remainder of pregnancy
* Family considered and decline option of termination of the pregnancy at less than 24 weeks 0 days
* Family meets psychosocial criteria
Exclusion Criteria:
* Patient \< 18 years old
* Multi-fetal pregnancy
* Rubber latex allergy
* Preterm labor, cervix shortened (\<15 mm) or uterine anomaly strongly predisposing to preterm labor, placenta previa
* Bilateral CDH, isolated left sided CDH with an O/E \> 30%
* Additional fetal anomaly by ultrasound, MRI, or echocardiogram
* Chromosomal abnormalities
* Maternal contraindications to fetoscopic surgery or severe maternal condition in pregnancy
* Incompetent cervix with or without a cerclage
* Placental abnormalities known at time of enrollment
* Maternal HIV, Hepatits B, Hepatitis C
* Maternal uterine anomaly
* No safe or technically feasible fetoscopic approach to balloon placement
* Participation in another intervention study that influences maternal and fetal morbidity and mortality or participation in this trial in a previous pregnancy DEVICE: Fetal Tracheal Occlusion
Congenital Diaphragmatic Hernia, Pulmonary Hypoplasia, Pulmonary, Hypertension
A Study to Assess Growth in Children With Idiopathic Short Stature
Cierra Farrell - cierra.farrell@cchmc.org
ALL
2 years to 16 years old
NCT06309979
Inclusion Criteria:
• Participants must be \> 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the informed consent.
• A height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth chart (https://www.cdc.gov/growthcharts/zscore.htm).
• Participants who have either never received hGH, or who are currently receiving hGH treatment.
• Historic stimulation test result with serum or plasma GH level greater than 10 μg/L.
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.
Exclusion Criteria:
• Diagnosis of systemic disease or condition that may cause short stature, eg renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. Children with such diagnoses can be considered for inclusion if their condition is well controlled, at the discretion of the Medical Monitor.
• Known presence of one or more pituitary hormone deficiencies
• Bone age advanced over chronological age by more than 3 years.
• For hGH naïve participants, historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (between -1.00 SDs and +2.00 SDs).
• For participants currently on hGH treatment, historic results before GH treatment of stimulation test with serum or plasma GH level greater than 10 μg/L or serum IGF-1 test between -1.00 SDs and +2.00 SDs.
• Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .
Idiopathic Short Stature
Clinical, Imaging, and Endoscopic Outcomes of Children Newly Diagnosed With Crohn's Disease (CAMEO)
Katie Lake - kathleen.lake@cchmc.org
ALL
6 years to 17 years old
PHASE4
NCT05781152
Phase 1 Inclusion Criteria
• Age ≥ 6 years and \< 18 years at enrollment
• Suspected diagnosis of CD
• Stool culture if performed that is negative for routine enteric pathogens (Salmonella, Shigella, Campylobacter, E. coli 0157:H7) and Clostridium difficile toxin in patients presenting with diarrhea. If history of C. difficile then a minimum of 6 weeks duration from treatment start and negative repeat stool for C. difficile toxin.
• Parent/guardian consent and patient assent
• Ability to remain in follow-up for up to 6 months of initial observation followed by a minimum of 52 weeks after possible start of anti-TNF therapy Phase 1 Exclusion Criteria
• Diagnosis of CD following abdominal resectional surgery/appendectomy at initial presentation
• Investigator judgment that patient has high likelihood (\>50%) of needing bowel resection within 3 months of diagnosis (i.e., presentation with perforation, bowel obstruction from stricture)
• Use of any oral CS for non-gastrointestinal indication within the four weeks prior to diagnostic assessment and biosampling (e.g., asthma)
• Use of any investigational drug within the past four weeks prior to diagnostic assessment and sampling
• Pregnancy
• Patients with poorly controlled medical conditions (e.g. diabetes, congestive heart failure)
• Previous treatment with immunomodulators within one year of enrollment or anti-TNF therapy within two years of enrollment for other medical conditions (e.g., juvenile idiopathic arthritis)
• Previous treatment with non-anti TNF biologics or small molecules for non-IBD indications in the past 6 months, with the exception of dupilumab (Dupixent) for asthma, eczema, or eosinophilic esophagitis
• Inability to have MRE because of claustrophobia or other reasons Phase 2 Inclusion Criteria
• Met all eligibility criteria for Phase 1 and participated in Phase 1
• Diagnosed with macroscopic CD involving the terminal ileum and/or colon by endoscopic evaluation and/or MRE
• MRE imaging within 6 weeks of ileocolonoscopy and no more than 4 weeks after starting initial therapy (TT). A limited 'research protocol' MRE is acceptable in participants who have undergone a clinical CTE during their initial diagnostic evaluation; see Manual of Procedures for details.
• Received at least one of the following as initial therapy upon diagnosis:
• Corticosteroids
• Immunomodulator
• Aminosalicylic acids (5-ASA)
• Defined nutritional therapy
• Anti-TNF (adalimumab or infliximab)
• Commenced adalimumab or infliximab anti-TNF therapy guided by ROADMAB™ CDST as first therapy or within 180 days of diagnosis (TD), with or without concomitant immunomodulator 6 a. Had ileal and rectal biopsies, OR b. Ileal biopsies are not obtained secondary to inflammatory or structural changes at the ileocecal valve or distal ileum that prevent ileal intubation. To be acceptable for Phase 2, the following additional criteria must be met: b1. Gross inflammation or obvious narrowing at the IC valve or distal ileum as documented by the video colonoscopy, AND b2. MRE documentation of TI inflammation with or without narrowing, OR c. Ileal biopsies are not obtained secondary to inflammatory or structural changes due to colonic CD. 7\. Parent/guardian consent and patient assent 8. Ability to remain in follow-up for a minimum of 52 weeks after start of anti-TNF therapy Phase 2 Exclusion Criteria
• Diagnosis of CD using video capsule endoscopy only with normal ileocolonoscopy and normal MRE
• Orofacial CD only
• Esophageal, gastric, duodenal, and/or jejunal CD only
• Severe complex fistulizing perianal disease +/- abscess, or perianal disease requiring surgical intervention or likely to require on-going surgical intervention possibly including diversion. The placement of a seton is not exclusionary. Incision and drainage of a perirectal abscess is also not exclusionary.
• Perianal CD only with no evidence of luminal disease
• Internal fistulizing disease at diagnosis
• Initial IBD treatment with non-anti-TNF biologic or small molecule therapy
• Received any anti-TNF agent other than adalimumab or infliximab
• Investigator judgment that patient unlikely to return for clinical, endoscopic or MRE follow-up
• Inability to have MRE because of claustrophobia or other reasons
• Video of baseline endoscopy not available for central reading, unless otherwise approved by the Clinical Coordinating Center (Adequate photo documentation required)
• Underwent bowel resection within 3 months of diagnosis (TD)
• Age ≥ 6 years and \< 18 years at enrollment
• Suspected diagnosis of CD
• Stool culture if performed that is negative for routine enteric pathogens (Salmonella, Shigella, Campylobacter, E. coli 0157:H7) and Clostridium difficile toxin in patients presenting with diarrhea. If history of C. difficile then a minimum of 6 weeks duration from treatment start and negative repeat stool for C. difficile toxin.
• Parent/guardian consent and patient assent
• Ability to remain in follow-up for up to 6 months of initial observation followed by a minimum of 52 weeks after possible start of anti-TNF therapy Phase 1 Exclusion Criteria
• Diagnosis of CD following abdominal resectional surgery/appendectomy at initial presentation
• Investigator judgment that patient has high likelihood (\>50%) of needing bowel resection within 3 months of diagnosis (i.e., presentation with perforation, bowel obstruction from stricture)
• Use of any oral CS for non-gastrointestinal indication within the four weeks prior to diagnostic assessment and biosampling (e.g., asthma)
• Use of any investigational drug within the past four weeks prior to diagnostic assessment and sampling
• Pregnancy
• Patients with poorly controlled medical conditions (e.g. diabetes, congestive heart failure)
• Previous treatment with immunomodulators within one year of enrollment or anti-TNF therapy within two years of enrollment for other medical conditions (e.g., juvenile idiopathic arthritis)
• Previous treatment with non-anti TNF biologics or small molecules for non-IBD indications in the past 6 months, with the exception of dupilumab (Dupixent) for asthma, eczema, or eosinophilic esophagitis
• Inability to have MRE because of claustrophobia or other reasons Phase 2 Inclusion Criteria
• Met all eligibility criteria for Phase 1 and participated in Phase 1
• Diagnosed with macroscopic CD involving the terminal ileum and/or colon by endoscopic evaluation and/or MRE
• MRE imaging within 6 weeks of ileocolonoscopy and no more than 4 weeks after starting initial therapy (TT). A limited 'research protocol' MRE is acceptable in participants who have undergone a clinical CTE during their initial diagnostic evaluation; see Manual of Procedures for details.
• Received at least one of the following as initial therapy upon diagnosis:
• Corticosteroids
• Immunomodulator
• Aminosalicylic acids (5-ASA)
• Defined nutritional therapy
• Anti-TNF (adalimumab or infliximab)
• Commenced adalimumab or infliximab anti-TNF therapy guided by ROADMAB™ CDST as first therapy or within 180 days of diagnosis (TD), with or without concomitant immunomodulator 6 a. Had ileal and rectal biopsies, OR b. Ileal biopsies are not obtained secondary to inflammatory or structural changes at the ileocecal valve or distal ileum that prevent ileal intubation. To be acceptable for Phase 2, the following additional criteria must be met: b1. Gross inflammation or obvious narrowing at the IC valve or distal ileum as documented by the video colonoscopy, AND b2. MRE documentation of TI inflammation with or without narrowing, OR c. Ileal biopsies are not obtained secondary to inflammatory or structural changes due to colonic CD. 7\. Parent/guardian consent and patient assent 8. Ability to remain in follow-up for a minimum of 52 weeks after start of anti-TNF therapy Phase 2 Exclusion Criteria
• Diagnosis of CD using video capsule endoscopy only with normal ileocolonoscopy and normal MRE
• Orofacial CD only
• Esophageal, gastric, duodenal, and/or jejunal CD only
• Severe complex fistulizing perianal disease +/- abscess, or perianal disease requiring surgical intervention or likely to require on-going surgical intervention possibly including diversion. The placement of a seton is not exclusionary. Incision and drainage of a perirectal abscess is also not exclusionary.
• Perianal CD only with no evidence of luminal disease
• Internal fistulizing disease at diagnosis
• Initial IBD treatment with non-anti-TNF biologic or small molecule therapy
• Received any anti-TNF agent other than adalimumab or infliximab
• Investigator judgment that patient unlikely to return for clinical, endoscopic or MRE follow-up
• Inability to have MRE because of claustrophobia or other reasons
• Video of baseline endoscopy not available for central reading, unless otherwise approved by the Clinical Coordinating Center (Adequate photo documentation required)
• Underwent bowel resection within 3 months of diagnosis (TD)
DRUG: Anti-TNF therapy
Crohn Disease
crohn disease, children, pediatric, anti-TNF therapy, therapeutic drug monitoring, intestinal microbiome, inflammatory bowel disease, gene expression, genomic DNA
International Diffuse Intrinsic Pontine Glioma (DIPG)/Diffuse Midline Glioma (DMG) Registry and Repository
Lori Lori Backus - Lori.Backus@cchmc.org
ALL
NCT03101813
Inclusion Criteria:
* All patients of any age (living or deceased) with a diagnosis (either current or past) of a DIPG/DMG tumor
* 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. Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Diffuse Midline Glioma
Study of Oral Food Challenge Biomarkers (SAFER) (SAFER)
Glenda Knox - glenda.knox@cchmc.org
ALL
12 months to 17 years old
NCT07279467
Inclusion Criteria:
• Parent or guardian must be able to understand and provide written informed consent and participant must be able to understand and provide assent (if applicable)
• Are 12 months - 17 years of age during Screening.
• Are sensitized to peanut, as demonstrated by one of the following: a. A participant-reported reaction to peanut (at any time) deemed by the investigator to be consistent with an IgE-mediated reaction and one of the following within the past 12 months i. Positive Skin Prick Test (SPT) to peanut (wheal diameter that is ≥ 3mm larger than saline control) ii. Positive peanut-specific IgE (sIgE; ≥ 0.10 kUA/L) determined by ImmunoCap b. An Ara h 2 sIgE ≥ 1.0 kUA/L, measured within the past 12 months
• Are currently avoiding peanut
Exclusion Criteria:
• Are pregnant
• Are currently receiving treatment or have received treatment within the prior 2 years for peanut allergy
• Have a history of life-threatening anaphylaxis to peanut, defined as neurological compromise or requiring intubation
• Have received treatment with dupilumab/Dupixent® within the prior 2 years, OR treatment with omalizumab/Xolair®, other biologics, or systemic immunomodulatory agents within the prior year
• Inability to comply with the required aspects of the study protocol
• Have past or current medical conditions or findings from the physical examination, not already listed, that, in the judgment of the site investigator, may pose additional risks related to participation in the study, interfere with the participant's ability to comply with study requirements, or impact the quality or interpretation of the study data
• Have been treated with oral steroid or beta blockers within 14 days of the Oral Food Challenge (OFC)
• Are unable to discontinue medications, as specified in the Protocol CoFAR-15 MOP, for the minimum wash-out periods prior to Skin Prick Test (SPT) or OFC
• Have poorly controlled atopic dermatitis (AD) at Screening, per the PI's discretion
• Have poorly controlled or severe asthma/wheezing at Screening, as defined as experiencing or including any of the following:
• Meeting the Global Initiative for Asthma (GINA) criteria for uncontrolled asthma
• History of two or more systemic corticosteroid courses within six months of Screening or one course of systemic corticosteroids within three months of Screening to treat asthma/wheezing;
• Prior intubation/mechanical ventilation for asthma/wheezing;
• One hospitalization or ED visit for asthma/wheezing within six months of Screening;
• Forced expiratory volume in one second (FEV1) \<80% of predicted or FEV1/forced vital capacity (FVC) \<75% of predicted, with or without controller medications (only for participants who are aged seven years or older and are able to perform spirometry);
• Inhaled corticosteroid (ICS) dosing of \>500 mcg daily fluticasone (or equivalent ICS based on the CoFAR Inhaled Corticosteroid Equivalency Tables MOP)
• Refuse blood sample collection during Screening
• The SPT performed during Screening is negative to peanut allergen without dilution (wheal diameter that is \< 3mm larger than saline control).
DRUG: Oral Food Challenge (OFC): Peanut Protein
Allergy
Food Allergy, Peanut Allergy, Oral Food Challenge, CoFAR-15, SAFER, Biomarkers
Registry of Asthma Characterization and Recruitment 3 (RACR3) (RACR3)
Kathryn Geil - kathryn.geil@cchmc.org
ALL
NCT05272241
Inclusion Criteria:
• Participant is either:
• At least 18 years old, willing and able to provide informed consent at the time of enrollment
• Under the age of 18, accompanied by a legal guardian who is willing and able to provide informed consent at the time of enrollment
• Participant has a primary place of residence within the Office of Management and Budget (OMB)-defined Metropolitan Statistical Area (MSA)
Exclusion Criteria:
• Participant does not speak English or Spanish and/or guardian does not speak English or Spanish
• Participant does not have access to a phone, either personal or public, with regularity that could be used for scheduling and safety follow-up
• Past or current medical problems or findings from physical examination or laboratory testing, 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 affect the quality or interpretation of the data obtained from the study Participants who are pregnant or lactating will not be excluded or discontinued from the study, but will not undergo any procedures that are prohibited during pregnancy per the Childhood Asthma in Urban Settings 02 (CAUSE-02) Registry for Asthma Characterization and Recruitment 3 (RACR3) Manual of Procedures (MOP)(e.g., allergen skin testing, spirometry) during the pregnancy. Potential participants may be reassessed as outlined in the Protocol CAUSE-02 MOP.
Asthma
Asthma, Allergy, CAUSE, RACR
A Study Testing the Combination of Dasatinib or Imatinib to Chemotherapy Treatment With Blinatumomab for Children, Adolescents, and Young Adults With Philadelphia Chromosome Positive (Ph+) or ABL-Class Philadelphia Chromosome-Like (Ph-Like) B-cell Acute Lymphoblastic Leukemia (B-ALL)
- cancer@cchmc.org
ALL
366 days to 46 years old
PHASE2
NCT06124157
Inclusion Criteria:
* Patients must be \> 365 days and \< 18 years (for AIEOP-BFM), \> 365 days and \< 22 years (for Children's Oncology Group \[COG\]) and \> 365 days and \< 46 years (for ALLTogether sites) at the time of enrollment
* Newly-diagnosed Ph+ or ABL-class Ph-like B-ALL. Leukemic blasts must express CD19. ABL-class fusions are defined as rearrangements involving the following genes predicted to be sensitive to imatinib and/or dasatinib: ABL1, ABL2, CSF1R, and PDGFRB
* Evidence of BCR::ABL1 should be documented by a clinically-validated assay prior to study entry on day 15 from the first dose of vinCRIStine during Induction therapy. ABL-class Ph-like B-ALL gene rearrangements should be documented by a clinically-validated assay and enrolled on study by day 1 of Blinatumomab Block 1. Accepted methods of detection include fluorescence in situ hybridization (FISH) using break-apart of colocalization signal probes, singleplex or multiplex reverse-transcription polymerase chain reaction (RT-PCR), whole-transcriptome or panel-based ribonucleic acid (RNA) sequencing (e.g., Hematologic Cancer Fusion Analysis, TruSight RNA Pan-Cancer Panel or equivalent). Confirmation of 5' fusion partner genes is not required for study enrollment
* Patients with Ph+ B-ALL must have previously started Induction therapy, which includes vinCRIStine, a corticosteroid, pegaspargase or calaspargase pegol, with or without anthracycline, and/or other standard cytotoxic chemotherapy
* Patients with Ph+ B-ALL have not received more than 14 days of systemic Induction therapy beginning with the first Induction dose of vinCRIStine
* Patients with ABL-class Ph-like B-ALL must have previously completed 4 or 5 weeks of multiagent Induction chemotherapy (Induction 1A)
* Patients may have started either imatinib or dasatinib prior to study entry but should have received no more than 14 days of TKI for Ph+ B-ALL or no more than 35 days of TKI for ABL-class Ph-like B-ALL
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of ≤ 2 or Karnofsky and Lansky performance scores ≥ 50%. Use Karnofsky for patients \> 16 years of age and Lansky for patients ≤ 16 years of age
* For pediatric patients (age 1-17 years): a glomerular filtration rate (GFR) ≥ 50 mL/min/1.73 m\^2, as determined by one of the following methods (must be performed within 7 days prior to enrollment unless otherwise indicated):
* Estimated GFR (eGFR) ≥ 50 mL/min/1.73 m2
* Measured GFR ≥ 50 mL/min/1.73 m\^2 (any age). If measured GFR is used, it must be performed using direct measurement with a nuclear blood sampling method or small molecule clearance method (iothalamate or other molecule per institutional standard
* For adult patients (age 18 years or older): Creatinine clearance ≥ 30 mL/min, as estimated by the Cockcroft and Gault formula. The creatinine value used in the calculation must have been obtained within 28 days prior to registration. Estimated creatinine clearance is based on body weight
* Direct bilirubin \< 2.0 mg/dL (34.2 micromoles/L) (must be performed within 7 days prior to enrollment unless otherwise indicated)
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 10 x upper limit of normal (ULN) (must be performed within 7 days prior to enrollment unless otherwise indicated)
* \* Shortening fraction of ≥ 27% by echocardiogram (must be obtained within 21 days prior to enrollment and start of protocol therapy \[repeat if necessary\]) OR
* Left Ventricular Ejection fraction of ≥ 50% by radionuclide angiogram or echocardiogram (must be obtained within 21 days prior to enrollment and start of protocol therapy \[repeat if necessary\]) AND
* Corrected QT Interval, QTc \< 480mSec (must be obtained within 21 days prior to enrollment and start of protocol therapy \[repeat if necessary\])
* Note: Repeat echocardiogram and electrocardiogram are not required if they were performed at or after initial ALL diagnosis before study enrollment
Exclusion Criteria:
* Known history of chronic myeloid leukemia (CML)
* ABL-class Ph-like B-ALL who are CNS2 or CNS3 at end of Induction phase
* ALL developing after a previous cancer treated with cytotoxic chemotherapy
* Active, uncontrolled infection or active systemic illness that requires ongoing vasopressor support or mechanical ventilation
* Down syndrome (trisomy 21)
* Pregnancy and breast feeding
* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A negative pregnancy test is required for female patients of childbearing potential within 7 days prior to enrollment
* Lactating females who plan to breastfeed their infants
* Sexually active male and female patients of reproductive potential who have not agreed to use an effective contraception method for the duration of treatment according to protocol
* NOTE: Patients who could become pregnant or could father a child must use effective contraception during protocol treatment and for 30 days after the last dose of dasatinib or 14 days after the last dose of imatinib dose or per institutional standard of care for multiagent chemotherapy, whichever is longer
* Prior treatment with TKIs before study entry with the exception of imatinib or dasatinib
* Patients with congenital long QT syndrome, history of ventricular arrhythmias, or heart block
* Patients with known Charcot-Marie-Tooth disease
* Patients with significant central nervous system pathology that would preclude treatment with blinatumomab, including history of severe neurologic disorder or autoimmune disease with central nervous system (CNS) involvement
* Note: Patients with a history of seizures that are well controlled on stable doses of anti-epileptic drugs are eligible. Patients with a history of cerebrovascular ischemia/hemorrhage with residual deficits are not eligible. Patients with a history of cerebrovascular ischemia/hemorrhage remain eligible provided all neurologic deficits have resolved
* HIV-infected patients are eligible if on effective anti-retroviral therapy that does not interact with planned study agents and with undetectable viral load within 6 months of treatment
* 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, BIOLOGICAL: Blinatumomab, PROCEDURE: Bone Marrow Biopsy, DRUG: Calaspargase Pegol, DRUG: Cyclophosphamide, DRUG: Cytarabine, DRUG: Dasatinib, DRUG: Daunorubicin, DRUG: Doxorubicin, PROCEDURE: Echocardiography Test, DRUG: Imatinib, DRUG: Leucovorin, DRUG: Mercaptopurine, DRUG: Methotrexate, PROCEDURE: Multigated Acquisition Scan, DRUG: Pegaspargase, DRUG: Prednisolone, DRUG: Prednisone, RADIATION: Radiation Therapy, DRUG: Thioguanine, DRUG: Vincristine
B Acute Lymphoblastic Leukemia
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children (BEGIN)
Kelly Thornton - kelly.thornton@cchmc.org
ALL
Up to 10 years old
NCT04509050
Inclusion Criteria:
* Part A:
* Less than 10 years of age at the first study visit.
* Documentation of a CF diagnosis.
Part B:
* Participated in Part A OR less than 7 years of age at the first study visit.
* Documentation of a CF diagnosis.
* CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor).
* Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Exclusion Criteria:
* Part A and Part B:
* Use of an investigational drug within 28 days prior to and including the first study visit.
* Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit.
* Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit. DRUG: Ivacaftor or elexacaftor/tezacaftor/ivacaftor
Cystic Fibrosis
Cystic Fibrosis, CF, CFTR Modulator, triple combination therapy, elexacaftor, tezacaftor, ivacaftor
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT (DMD-001 SUMMIT)
Cuixia Tian, MD - Cuixia.Tian@cchmc.org
MALE
2 years and over
NCT06564974
Inclusion Criteria:
• Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
• Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
• Male patients at least 2 years old.
• Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
• Patient has a current, active prescription for, or is on, AGAMREE®.
Exclusion Criteria:
1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments. DRUG: Vamorolone
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy, Neuromuscular, AGAMREE®, Vamorolone, DMD, Muscular Dystrophy
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy
Jessica Getz - Jessica.getz@cchmc.org
ALL
Up to 5 years old
NCT06503367
Inclusion Criteria:
* Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable).
* Subject must be aged birth to less than 5.0 years of age at time of consent.
* A confirmed diagnosis of LAMA2-RD confirmed via:
a: Two pathogenic variants in the LAMA2 gene (via a CLIA-approved laboratory) or: b. muscle biopsy with absence of merosin (laminin-211) and at least one pathogenic variant in the LAMA2 gene
* Absence of another confirmed genetic disease.
* Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study.
* Willingness to comply with the study protocol, including but not limited to, all study procedures and visits.
Exclusion Criteria:
* Acute medical illness or hospitalization within 30 days prior to informed consent.
* Participation in a previous trial of any investigational agent for LAMA2-RD within 1 month prior to informed consent, or use of any other investigational therapy (including off-label use of Losartan) within 30 days prior to informed consent, or participation in other clinical studies, within 30 days (or 3 half-lives, whichever is longer) prior to informed consent, which in the opinion of the PI, may potentially confound results from this study.
* Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD. LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Early Phase 1, Observational, Natural History, Neuromuscular, Functional Assessments
RASopathy Biorepository
Lindsey Aschbacher-Smith - lindsey.aschbacher-smith@cchmc.org
ALL
NCT04395495
Inclusion Criteria:
* Patients with a suspected or known diagnosis of any of the group of disorders known as RASopathies (e.g., Neurofibromatosis, Costello Syndrome, Noonan Syndrome). Diagnosis may be made clinically and/or confirmed through genetic testing.
* Unaffected relatives of patients with a suspected or known diagnosis of any of the group of disorders known as RASopathies.
Exclusion Criteria:
* Individuals who do not have a suspected or definite diagnosis of a RASopathy.
* Individuals who do not have a relative with a suspected or definite diagnosis of a RASopathy.
* Patients who do not have the ability/capacity to undergo the informed consent process OR whose parent/legal guardian is unable to undergo the informed consent process. RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines, Noonan Neurofibromatosis Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Legius Syndrome, Smith-Kingsmore Syndrome, MTOR Gene Mutation, GATOR-1 Gene Mutation, SYNGAP1-Related Intellectual Disability, DLG4, MAPK1 Gene Mutation
A Clinical Study of Letermovir (MK-8228) in Children and Adolescents Who Receive a Kidney Transplant (KT) (MK-8228-077)
Caitlin Brammer - caitlin.brammer@cchmc.org
ALL
Up to 17 years old
PHASE1
NCT07199465
Inclusion Criteria:
The main inclusion criteria include but are not limited to the following:
* Is a recipient of a primary or secondary allograft kidney
* Is at least 4 weeks posttransplant and not more than 52 weeks posttransplant at the time of enrollment (Day 1) and is being managed per local standard of care
* Has stable kidney function posttransplant
* Has undetectable CMV deoxyribonucleic acid (DNA) from a plasma or whole blood sample collected within 14 days prior to enrollment
* Must be able to take (as assessed by the investigator) letermovir tablets or oral pellets by mouth, or via gastrostomy or nasogastric tube (oral pellets only)
* Does not have a condition that may interfere with the absorption of oral medication (e.g., vomiting, diarrhea, or a malabsorptive condition) from the day of enrollment (Day 1) until the intensive pharmacokinetics (IPK) sampling is completed
* Weighs ≥2.5 and \<40 kg at enrollment (Day 1)
Exclusion Criteria:
The main exclusion criteria include but are not limited to the following:
* Has CMV disease or suspected CMV disease between screening and enrollment
* Is on dialysis or plasmapheresis at the time of enrollment
* Has evidence of CMV viremia at any time from screening until the time of enrollment
* Has Child-Pugh B or C hepatic insufficiency within 14 days before enrollment
* Is a multi-organ transplant recipient (e.g., kidney-pancreas)
* Has any uncontrolled infection on the day of enrollment
* Requires mechanical ventilation, or is hemodynamically unstable, at the time of enrollment
* Has received or is receiving protocol-specified prohibited medications DRUG: Letermovir
Cytomegalovirus Prophylaxis
A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia
Lauren Pommert, MD - lauren.pommert@cchmc.org
ALL
29 days to 21 years old
PHASE2
NCT05955261
Patients with preexisting conditions that may affect chemotherapy tolerance (e.g., bone marrow failure syndrome, cardiac disease, pulmonary disease, and history of significant hepatic impairment) should be discussed with study PI in detail to enable review of the medical history prior to enrollment onto AML23 study.
Inclusion Criteria:
* Diagnosis of AML fulfilling the criteria of the WHO classification of myeloid neoplasms or \< 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality or myeloid sarcoma or primary myelodysplastic syndrome (MDS) with ≥ 10% blasts or a complete blood count with the presence of at least 1,000 blasts/μL (e.g., a WBC count ≥ 10,000/μL with ≥ 10% blasts or a WBC count ≥ 5,000/μL with ≥ 20% blasts
* Age \> 28 days and \< 22 years
* No prior therapy for this malignancy except for one dose of intrathecal therapy and hydroxyurea or low-dose cytarabine (≤ 200 mg/m\^2 per day for ≤ 7 days)
* Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior to enrollment
* Male and female participants of reproductive potential must agree to use an effective contraceptive method during the study and for 6 months after study treatment
* Written informed consent from the patient and/or parent/legal guardian
* Direct bilirubin ≤ 1.5 x institutional upper limit of normal
Exclusion Criteria:
* Patients with treatment-related AML, Down syndrome, acute promyelocytic leukemia, chronic myeloid leukemia in blast crisis, juvenile myelomonocytic leukemia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or other bone marrow failure syndromes are not eligible
* Uncontrolled systemic fungal, bacterial, or viral infection or significant concurrent disease that would compromise patient safety or compliance, study participation, follow up, or interpretation of study results
* Prior exposure to any dose of anthracycline or anthracenedione
* Patients may not receive strong or moderate CYP3A inducers, such as rifampin, within 3 days of enrollment
* Patients may not receive moderate or strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, voriconazole, posaconazole) within 3 days of enrollment. DRUG: Venetoclax, DRUG: Azacitidine, DRUG: Cytarabine, DRUG: Gemtuzumab Ozogamicin, DRUG: Daunorubicin Hydrochloride, DRUG: Fludarabine Phosphate, DRUG: Idarubicin Hydrochloride, DRUG: Mitoxantrone Hydrochloride, DRUG: Etoposide, DRUG: Gilteritinib
Acute Myeloid Leukemia
Starlight Cardiovascular Lifeline Ductus Arteriosus Stent IDE Study
Amy Pajk - amy.pajk@cchmc.org
ALL
1 minute to 6 months old
NA
NCT07114718
Inclusion Criteria:
• Parental or legal authorized representative provide consent for study enrollment
• Infants \< 6 months of age
• Diagnosis of congenital heart defect with ductal-dependent pulmonary circulation requiring infusion of prostaglandins
• Requires ductus arteriosus stent diameters of 3.5mm or 4mm and stent lengths between 16mm and 28mm
Exclusion Criteria:
• Active blood stream infection
• Active or history of endocarditis
• Body weight \<2.5kg
• Gestational age \<32 weeks at birth
• Complete heart block
• Total Anomalous Pulmonary Venous Return
• Anatomic variation judged to be inappropriate for ductal stenting per the treating interventionalist
• Presence of an aortopulmonary collateral that is expected to require unifocalization
• Non-confluent pulmonary arteries (i.e. isolated pulmonary artery of ductal origin) or bilateral patent ductus arteriosus (PDA)
• Pulmonary atresia with intact ventricular septum with RV-dependent coronary circulation
• Currently participating in an investigational drug study or another device study that would confound the study results
• Patient who is on extracorporeal membrane oxygenation (ECMO), ventricular assist device (VAD), or dialysis prior to ductal stenting procedure
• Major co-morbidities which, in the opinion of the investigator, would negatively alter expected 1-year survival (e.g., intracranial hemorrhage, renal failure, etc.)
• Patient who is undergoing another transcatheter procedure (e.g., atrial septostomy, aortic arch intervention, or right ventricular outflow tract intervention) during or within 24 hours prior to the ductus arteriosus stenting procedure
• Patient who, at the time of enrollment, is deemed not to be a candidate for eventual stage II palliation of single ventricle heart disease, complete surgical repair, nor transcatheter treatment with resultant biventricular circulation
• Patient who does not plan to return to the enrolling center or another participating center for stage II palliation, complete surgical repair, or definitive catheterization procedure
• Contraindications to peri-procedural anticoagulation
• Known to be non-responsive to aspirin or other antiplatelet therapies
• Known hypersensitivity or allergy to Nickel
• Known hypersensitivity to contrast media that cannot be adequately pre-medicated
DEVICE: Ductus Arteriosus Stent
Congenital Heart Disease, Congenital Heart Disease (CHD)
Ductus Arteriosus Stent, pediatric stent, Lifeline Stent
Early Signals of the Transition From Immune Quiescence to Activation in the Liver Allograft Microenvironment and in the Circulation (iSYNAPSE)
Erin Chapman - Erin.Chapman@cchmc.org
ALL
3 years to 13 years old
PHASE2
NCT07549503
Inclusion Criteria:
• Participant and parent or guardian must be able to understand and provide informed assent and consent, respectively
• Recipient of a living or deceased donor Liver transplant (LTx) at \<7 years of age
• \> 3 years but \<7 years after LTx at the time of study enrollment
• Stable liver tests defined as baseline serum alanine aminotransferase (ALT) level \< 30 IU/l and gamma-glutamyl transferase (GGT) level \< 50 IU/l (based on the average of the 3 most recent values prior to screening; all must be within 1 year of screening; 2 must be within 6 months of screening)
• No Acute rejection (AR) or chronic rejection within 12 months of enrollment
• Tacrolimus monotherapy for \> 6 months with baseline 12-hour trough levels \<8 ng/mL (based on the average of 3 values prior to screening; all must be within 1 year of screening; 2 must be within 6 months of screening)
• Participants of childbearing potential must have a negative pregnancy test upon study entry
Exclusion Criteria:
• Liver transplant (LTx) for autoimmune disease, including autoimmune hepatitis or primary sclerosing cholangitis
• LTx for hepatitis B or hepatitis C
• Recipient of any other organ transplant or liver re-transplant, except for patients who have a repeat LTx within 30 days of first LTx who are eligible for enrollment
• \>=50 percent dose increase in tacrolimus within 12 months of enrollment
• Discontinued a second Immunosuppression (IS) agent within 12 months of enrollment
• Systemic illness requiring chronic or recurrent use of IS for which there is a risk of reactivation if tacrolimus is reduced
• Use of medication to treat systemic conditions which in the judgement of the investigator could influence results of the study
• Active or chronic infection requiring treatment
• Inability or unwillingness to comply with the study protocol
• Use of investigational drug within 4 weeks (or 5 half-lives of investigational drug, whichever is longer) of enrollment
• Has any condition that, in the opinion of the investigator, will interfere with safe participation in the trial
DRUG: Immunosuppression Reduction
Liver Transplant
Liver transplant, Immunosuppression
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Laurie Bailey - laurie.bailey@cchmc.org
ALL
NCT00358943
Inclusion Criteria:
ICGG Gaucher Registry
* All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented β-glucocerebrosidase deficiency and/or mutation in the β-glucocerebrosidase gene.
* For all patients, appropriate patient authorization will be obtained.
Gaucher Pregnancy Sub-registry:
* be enrolled in the ICGG Gaucher Registry.
* be pregnant, or have been pregnant with appropriate medical documentation available.
* provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.
Exclusion Criteria:
\- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry. Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
Gaucher Disease, Glucocerebrosidase Deficiency Disease
Improving Outcomes in Pediatric Obstructive Sleep Apnea With Computational Fluid Dynamics (OSA-MRI)
Alex Sierra, MS - alex.sierra@cchmc.org
ALL
3 years to 18 years old
PHASE4
NCT04991389
Inclusion Criteria:
* Male or Female
* Subjects between the ages of 5 to 18 only for Aim 1 and xenon use
* Subjects 3-18 years of age for Aims 2 and 3
* Subjects with persistent moderate or severe OSA after adenotonsillectomy. - -- Persistent moderate or severe OSA will be defined as an oAHI \> 5 per hour of sleep.
* Clinical indication or suspicion of upper-airway obstruction. Examples include but not limited to hypertrophy of the lingual tonsils, disproportionately large tongue, or micrognathia.
* Subjects who have failed a trial of CPAP.
* Subjects whose parents elect to pursue surgery without a trial of CPAP.
* Subjects who require a surgical procedure for OSA based on the clinical assessment of the surgeon (otolaryngologist or plastic surgeon).
Exclusion Criteria:
* Children adequately treated with CPAP.
* Children with braces/metal rods.
* Children who have a contraindication to sedative.
* Standard MRI exclusion criteria as set forth by the CCHMC Department of Radiology. DRUG: 129-Xe
Obstructive Sleep Apnea
OSA, 129Xe, MRI, CFD simulations
Integrative Training Program for Pediatric Sickle Cell Pain (I-STRONG SCD)
Katie Katie Beasley - Katie.Beasley@cchmc.org
ALL
12 years to 18 years old
NA
NCT06691867
Inclusion Criteria:
* Provision of signed and dated informed consent form for adolescents 18 years old and caregivers' participation
* For children \<18, informed assent and parental informed consent to participate in the study
* Willingness to comply with all study procedures and lifestyle considerations and availability for the duration of the study
* Males and females; Ages 12-18 years for adolescents; no age limitations for caregivers
* Documented diagnosis of sickle cell disease (any genotype) for adolescents
* Adolescent scores at least 3 on the Pediatric Pain Screening Tool
* Adolescent reports typical pain intensity in the past week at least 4 on a 0-10 cm Visual Analog Scale
* Adolescent on stable disease-modifying treatments, if applicable (e.g., hydroxyurea, glutamine, voxelotor, crizanlizumab) as defined by not newly initiated or significantly increased dosages (mg/kg) in the past 3 months
* Speak and read English
Exclusion Criteria:
* An adolescent has comorbid medical conditions typically associated with pain but unrelated to SCD (e.g., rheumatologic disorders or inflammatory bowel disease)
* Adolescent has undergone genetic or hematopoietic stem cell therapy
* Presence of a condition(s) or diagnosis, either physical or psychological, or physical exam finding that precludes participation
* Adolescents receiving active treatment (e.g., weekly appointments with a provider) for nonpharmacological therapies (e.g., structured behavioral pain management, physical therapy, or acupuncture program) that overlap with the active phase of the study intervention BEHAVIORAL: I-STRONG, OTHER: Enhanced Usual Care (EUC)
Sickle Cell Disease
Sickle Cell Pain, Lifestyle considerations, Pediatrics, Behavioral intervention, Mind-body intervention
Evaluation of the SMART IBD App in Pediatric IBD
Jessica King - jessica.king1@cchmc.org
ALL
13 years to 17 years old
NA
NCT07195123
Inclusion Criteria:
* Confirmed diagnosis of IBD (Crohn's Disease, ulcerative colitis, or indeterminate colitis)
* Prescribed at least one daily or weekly medication for treatment of IBD
* \<86% adherence to prescribed medication
* Access to internet via Wi-Fi or data plan and access to smartphone
* English fluency for patient and caregiver
Exclusion Criteria:
* Diagnosis of pervasive developmental disorder in patient or caregiver as determined by medical chart review
* Diagnosis of serious mental illness (e.g., schizophrenia) in patient or caregiver as determined by medical chart review BEHAVIORAL: SMART-IBD
Inflammatory Bowel Disease (IBD), IBD (Inflammatory Bowel Disease), IBD - Inflammatory Bowel Disease, Ulcerative Colitis (UC), Indeterminate Colitis, Crohn Disease (CD), IBD
IBD, Inflammatory Bowel Disease, Ulcerative Colitis, Indeterminate Colitis, Crohns Disease
Biomarker Validation in Motor System Physiology in Attention Deficit Hyperactivity Disorder (AMPAIII)
Karlee Migneault - karlee.migneault@cchmc.org
ALL
8 years to 12 years old
PHASE4
NCT04421248
Inclusion Criteria:
* Either gender, any race, ethnicity or socioeconomic status
* Currently between 8 years 0 months and 12 years, 11 months, 30 days
* Willing to answer questions about ADHD and related diagnoses
* For children with ADHD prescribed stimulant medications, willing to suspend taking medications as specified in the study procedures
* For children with ADHD, willing to participate in the single dose, randomized crossover study to probe acute effects of methylphenidate on biomarkers
* Right hand dominant (predominately right-handed)
* Able to participate in and sign an informed consent
* ADHD inclusion: The diagnosis of ADHD will be based on Diagnostic and Statistical Manual version 5 (DSM-5) criteria using standard rating scales and a structured diagnostic interview. Oppositional Defiant Disorder is permitted; Conduct disorder is excluded.
* Typically Developing (healthy control) inclusion: Free of ADHD or other developmental or psychiatric disorders based on DSM-5 criteria using standard rating scales and a structured diagnostic interview.
Exclusion Criteria:
* Known diagnosis of mental retardation, cerebral palsy, Autism Spectrum Disorder, traumatic brain injury, brain tumor, epilepsy, or other serious neurological disorder.
* Major Depression, Bipolar Disorder, Conduct Disorder, Adjustment Disorder, other Anxiety Disorders, or other developmental psychiatric diagnoses.
* For females, onset of menses, pregnancy.
* Current use of antidepressants, non-stimulant ADHD medications, dopamine blocking agents, mood stabilizers.
* Implanted brain stimulator, vagal nerve stimulator, ventriculo-peritoneal shunt, cardiac pacemaker, or implanted medication port.
* Diagnosis of a speech/language disorder or a Reading Disability (RD). DRUG: Methylphenidate, DRUG: Placebo
Attention Deficit Hyperactivity Disorder Combined
Treating Young Children With Attention Deficit Hyperactivity Disorder (TYCA)
Hannah Bush, MA - ddbp.adhd.research@cchmc.org
ALL
3 years to 5 years old
PHASE4
NCT07300956
Inclusion Criteria:
• Males or females 3 to 5 years of age, inclusive (e.g. children age 5 years, 11 months are eligible).
• Child has a confirmed diagnosis of moderate or severe Attention-Deficit/Hyperactivity Disorder (diagnostic code: F90.9) based on Diagnostic and Statistical Manual of Mental Disorders - 5th Edition (DSM-5) criteria, review of supportive evidence (caregiver interview, behavioral observations, Vanderbilt Attention-Deficit/Hyperactivity Disorder Rating Scale results, any school or daycare information provided by caregiver).
• Clinician-rated Clinical Global Impairment-Severity (see description below) rating of ≥4 (denoting moderate or greater impairment due to their Attention-Deficit/Hyperactivity Disorder symptoms).
• The child's clinician and caretakers have decided on or are considering initiating drug therapy (with either Methylphenidate or Guanfacine).
• Primary language is English or Spanish.
Exclusion Criteria:
• History of taking Methylphenidate or Guanfacine. Prior use of other Attention-Deficit/Hyperactivity Disorder medications or other psychotropic medications is allowed.
• Current use of other Attention-Deficit/Hyperactivity Disorder medications (e.g., amphetamines), other psychotropic medications (e.g., atypical antipsychotics or serotonin reuptake inhibitors), or centrally active depressants (such as phenothiazines, barbiturates, or benzodiazepines)
• Electronic Health Record documentation of moderate to severe global developmental delay (F88) or intellectual disability (F70), or a score of \<55 on the Vineland Adaptive Behavior Scales-3.
• Medical contraindications to taking Methylphenidate or Guanfacine, including cardiac risk factors (e.g. known structural cardiac abnormalities, cardiomyopathy, serious cardiac arrhythmias, coronary artery disease, or other serious cardiac disease), increased intraocular pressure, glaucoma, verbal tics, Tourette's Syndrome, psychosis
DRUG: Guanfacine (GUA), DRUG: Methylphenidate (MPH)
Attention-Deficit/Hyperactivity Disorder (ADHD)
Attention-Deficit/Hyperactivity Disorder, Methylphenidate, Guanfacine, Preschool Children, Comparative Effectiveness, Prospective Study
Testing a Standardized Approach to Surgery and Chemotherapy for Type I Pleuropulmonary Blastoma or the Addition of an Anti-cancer Drug, Topotecan, to the Usual Treatment for Types II and III Pleuropulmonary Blastoma
- cancer@cchmc.org
ALL
Up to 21 years old
PHASE3
NCT06647953
Inclusion Criteria:
* 21 years of age or younger
* Newly diagnosed PPB. Note that patients with known germline DICER1 variant or mosaicism with a large, solid unresectable thoracic mass with imaging features characteristic for Type II or III PPB are eligible without histologic confirmation of the diagnosis if a biopsy of the mass is not considered safe or feasible
* Individuals are eligible based on institutional diagnosis of Type I, Ir, II or III PPB diagnosed within 60 days prior to enrollment. Children with Type II or III PPB at risk for clinical decompensation may receive protocol therapy while awaiting rapid central pathology review. Children with Type I or Ir PPB will be assigned to chemotherapy vs. observation based on imaging and central pathology review diagnosis. Type I and Ir patients should not begin chemotherapy prior to return of central pathology results
* For patients with Type II or III PPB (within 7 days prior to enrollment): A serum creatinine based on age/sex 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) OR - A 24 hour urine creatinine clearance ≥ 60 mL/min/1.73 m\^2 OR - A glomerular filtration rate (GFR) ≥ 60 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
* Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
* For patients with Type II or III PPB (within 7 days prior to enrollment): Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
* For patients with Type II or III PPB (within 7 days prior to enrollment): Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) ≤ 135 U/L
* Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
* Shortening fraction of ≥ 27% by echocardiogram, or ejection fraction of ≥ 50% by radionuclide angiogram (within 21 days prior to start of protocol therapy)
* HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible as long as they are NOT receiving anti-retroviral agents that are strong inhibitors or inducers of CYP3A4
Exclusion Criteria:
* Administration of prior PPB-directed chemotherapy is an exclusion criterion. Prior treatment for another malignancy is not an exclusion criterion
* Patients with known Charcot-Marie-Tooth disease
* 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
* 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, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Scan, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, BIOLOGICAL: Dactinomycin, DRUG: Dexrazoxane, DRUG: Doxorubicin, PROCEDURE: Echocardiography Test, DRUG: Ifosfamide, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Multigated Acquisition Scan, OTHER: Patient Observation, PROCEDURE: Positron Emission Tomography, DRUG: Topotecan, PROCEDURE: Ultrasound Imaging, DRUG: Vincristine
Pleuropulmonary Blastoma
Regional Monitoring of CF Lung Disease
Carrie Stevens, BS - carrie.stevens@cchmc.org
ALL
12 years to 21 years old
EARLY_PHASE1
NCT06339593
Inclusion Criteria:
* 1 Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
2 Use of highly effective modulators for more than 30 days (ie. Trikafta) 3 Willingness and ability to adhere to the study visit schedule and other protocol requirements.
4 Documentation of a CF diagnosis with prescription of Mechanical ACT 5 Ages 12-21 inclusive, at the time of consent. 6 Clinically stable with no respiratory tract infection or recent exacerbations. 7 Treating CF physician agreeable to study procedures. Only applicable to Aim 3.
8 No change in chronic maintenance therapies in the 28 days prior to enrollment.
9 Ability to cooperate with MRI procedures.
Exclusion Criteria:
• Standard MRI exclusions (metal implants, claustrophobia).
• For females of childbearing potential: Positive urine pregnancy test or Lactating.
• Acute respiratory symptoms (e.g., wheezing) at the time of the MRI
• Chronic lung or liver or pancreatic disease not related to CF.
• Any other condition that, in the opinion of the Investigator, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives.
PROCEDURE: Airway-clearance vest, DRUG: Hyperpolarized Xe129
Cystic Fibrosis
Survivors Journey+ (SJ)
Zyah Flagg - zyah.flagg@cchmc.org
ALL
15 years to 25 years old
NA
NCT07321353
Inclusion Criteria:
* a diagnosis of a pediatric intracranial tumor
* tumor-directed treatment completed \> 12 months ago (treatment included: surgery, radiation, or chemotherapy)
* lives with parent/guardian(s)
* language: English must be the primary spoken language in the home
Exclusion Criteria:
* history of tuberous sclerosis or neurofibromatosis
* treatment \< 12 months ago and/or treatment did not include surgery, radiation, or chemotherapy
* history of psychiatric hospitalization
* resides outside of the family home
* history of autism, reactive attachment disorder, psychosis, or other psychiatric diagnoses/conditions associated with significant risk of harm to self or others per caregiver
* English is not the primary language spoken in the home BEHAVIORAL: Survivor's Journey+
Pediatric Brain Tumor
pediatric brain tumor survivors, online learning, Adolescent young adult, Family, Digital, Problem Solving
Ferric Citrate and Chronic Kidney Disease in Children (FIT4KID)
Elizabeth Siry - elizabeth.siry@cchmc.org
ALL
6 years to 18 years old
PHASE2
NCT04741646
Inclusion Criteria:
• Ages 6 to 18 years (inclusive);
• Estimated Glomerular Filtration Rate (GFR) of 15-59 ml/min per 1.73 m2 by modified Chronic Kidney disease in Children (CKiD) under 25 (U25) formula;56
• Serum phosphate \<=5.9 mg/dl;
• Serum ferritin \<500 ng/ml and TSAT \<50%;
• For those patients treated with growth hormone, calcitriol, nutritional vitamin D, iron, and/or erythropoiesis-stimulating agents (ESAs) such treatments must have stable dosing for at least 2 weeks prior to screening;
• Able to swallow tablets;
• Able to eat at least two meals a day;
• In the opinion of the investigator, willing and able to follow the study treatment regimen and comply with the site investigator's recommendations.
Exclusion Criteria:
• Patients currently treated with phosphate binders.
• History of allergy to all ingredients (including non-medical ingredients) in both products (i.e. investigational product and placebo)
• Current intestinal malabsorption, documented in the medical record; disease, inflammatory bowel syndrome, and/or Crohn's Disease.
• Anticipated initiation of dialysis or kidney transplantation within 6 months
• Current or planned future systemic immunosuppressive therapy
• Prior solid organ transplantation
• Receipt of bone marrow transplant within two years of screening
• Current pregnancy, lactation or female subjects who have reached menarche, unless using highly-effective contraception as outlined in section 7.1.1 of Protocol
• Patients participating in other interventional study (observational study participation permitted)
• Poor adherence to medical treatments in the opinion of the investigator
• Cystinosis
• Fanconi syndrome
• Hemochromatosis or laboratory tests indicating possible hemochromatosis or other iron overload (primary or secondary) syndrome
DRUG: Ferric Citrate, DRUG: Placebo
Chronic Kidney Diseases
Pediatric, CKD, Phosphate Binder
Safety Study of Unlicensed IND Cord Blood Units Manufactured by the National Cord Blood Program for Unrelated Transplantation
Stephanie Edwards - stephanieL.edwards@cchmc.org
ALL
PHASE2
NCT01656603
Inclusion Criteria:
• Diagnosis: Patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.
• Patients: Patients of any age and either gender
• Cord blood product manufactured by the NCBP (at least one, if the graft contains more than one units)
Exclusion Criteria:
• Patients who are receiving licensed cord blood products (only)
• Patients who are receiving unlicensed cord blood products from other banks (only)
• Patients who are transplanted at non-US transplant centers
• Patients who are receiving cord blood products that will be "manipulated" post-thaw (e.g., ex vivo expansion, incubation in vitro, etc.)
BIOLOGICAL: unlicensed CBU
Infusion Reactions
cord blood, transplantation, stem cells, adverse event