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40 Study Matches
Quantifying Motor Network Dynamics to Predict and Enhance Outcomes in Pediatric Dystonia
Karlee Migneault - karlee.migneault@cchmc.org
ALL
6 years to 21 years old
NA
NCT07325175
Inclusion Criteria:
* For dystonia subjects:
* Dx of dystonia (with and without DBS)
* willingness and ability to complete study protocols.
* For Typically Developing Controls:
* normal developmental milestones
* absence of any neuropsychiatric disorder
* no significant medical condition.
Exclusion Criteria:
* history of epilepsy
* presence of implanted medical devices (except DBS in dystonia subjects)
* lack of cognitive or physical ability to complete study protocol. DEVICE: DBS
Dystonia, Pediatric, Deep Brain Stimulation, Motor Development
Studying Health Outcomes After Treatment in Patients With Retinoblastoma (RIVERBOAT)
Amanda Pfeiffer - amanda.pfeiffer@cchmc.org
ALL
NCT03932786
* Unilateral or bilateral intraocular retinoblastoma
* Diagnosis between the ages of 0 - 17.99 years
* Diagnosis on or after January 1, 2008
* No exclusions based on primary or secondary treatment modalities
* Retrospective group patients must be ≥ 6 months post end of treatment at study entry
* For those already at this timepoint, they are now eligible
* For those in treatment, or otherwise not yet at this timepoint, they are eligible once at they are ≥ 6 months post end of treatment
* Prospective group patients must not have begun treatment
* Patients with diminished capacity will not be enrolled.
* Language: Patients must be able to communicate in English, French, or Spanish
* Sibling Cohort: One sibling, not affected by retinoblastoma will be enrolled, preference for the sibling closest in age to the RB patient.
* Regulatory Requirements: All patients and/or their parents or legal guardians must sign a written informed consent. All institutional, FDA, and NCI requirements for human studies must be met.
PROCEDURE: Biospecimen collection, OTHER: Vision assessment, OTHER: Questionnaire administration, OTHER: Quality of life assessment, OTHER: Laboratory Biomarker Analysis
Retinoblastoma, Cancer Survivor, Biological Sibling, Intraocular Retinoblastoma, Unilateral Retinoblastoma
A Safety and Immunogenicity Study of CHIKV VLP Vaccine in Children.
Julie Kulhanek - Julie.Kulhanek@cchmc.org
ALL
1 year to 11 years old
PHASE3
NCT07003984
Inclusion Criteria:
• Males or females between 1 and \<12 years of age at Day 1 (day of vaccination). Note: Screening should only occur in the active/open cohorts. Please see Section 6.1 for details
• Body weight ≥6.5 kg.
• In general good health, in the opinion of the investigator, based on medical history and physical examination.
• Able and willing to provide informed assent for study participation and primary caregiver is able and willing to provide informed consent for study participation, in accordance with the Institutional Review Board (IRB)/Independent Ethics Committee (IEC) determination and applicable federal and local regulations and guidelines.
• Able and willing to complete all scheduled visits and comply with all study procedures.
Exclusion Criteria:
• Participation or planned participation in an investigational clinical study (eg, vaccine, drug) within 30 days before Day 1 and for the duration of the study. Note: Participation in an observational study or follow-up phase of a study may be allowed; these instances should be discussed with the sponsor's medical monitor and written agreement obtained prior to enrollment.
• Current acute illness, with or without fever.
• Current or recent CHIKV infection indicated by positive immunoglobulin M (IgM) and negative immunoglobulin G (IgG) rapid diagnostic test (RDT) results at screening in the Philippines only; participants in the US will not be tested using the RDT.
• History of any known or suspected allergy or history of anaphylaxis to any component of the investigational product.
• History of any known congenital or acquired immunodeficiency or immunosuppressive condition that could impact response to vaccination.
• Prior receipt or anticipated use of systemic immunomodulatory or immunosuppressive medications from 180 days prior to screening through Day 22. Note: Systemic corticosteroid use at a dose or equivalent dose of 20 mg or greater (≥0.5 mg/kg for children \<40 kg) of prednisone for 14 consecutive days or more within 90 days of screening through Day 22 is exclusionary. The use of inhaled, intranasal, topical, or ocular steroids is allowed.
• Receipt or anticipated receipt of immunoglobulin from 180 days prior to screening through the duration of the study.
• Any administration or planned administration of: * A licensed live attenuated vaccine within 28 days before administration of investigational product and until Visit 2 (Day 15 or 22, as applicable) has occurred. * Other licensed (not live) vaccine within 14 days before administration of investigational product and until Visit 2 (Day 15 or 22, as applicable) has occurred. * Another licensed or investigational CHIKV vaccine.
• Known infection with human immunodeficiency virus, hepatitis C virus (HCV), or hepatitis B virus. Note: Positive anti-HCV antibodies and negative HCV polymerase chain reaction would NOT be exclusionary. Polymerase chain reaction testing will not be performed as part of this protocol.
• Bleeding disorder or receipt of anticoagulants in the 21 days before Day 1, contraindicating intramuscular vaccination, as judged by the investigator.
• Receipt or anticipated receipt of blood products from 90 days before Day 1 through the duration of the study.
• Onset of menarche prior to study vaccination.
• Planned medical or surgical procedure that could adversely impact the participant's participation or the conduct of the study.
• Identified as an immediate family member of the investigator or employee with direct involvement in the study. Bavarian Nordic staff members and their families, contractors, agents, business partners, and anyone with a financial interest in the outcome of the study.
• Any other medical condition, including severe malnutrition, that, in the opinion of the investigator, could adversely impact the participant's participation or conduct of the study.
BIOLOGICAL: CHIKV VLP vaccine, BIOLOGICAL: Placebo
Chikungunya Virus
Chikungunya, PXVX0317, CHIKV VLP, vaccine, immunogenicity, VIMKUNYA, children
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG-Preschool Age
Elizabeth Smith - elizabeth.smith3@cchmc.org
ALL
24 months to 4 years old
NA
NCT05957549
Inclusion Criteria:
* Diagnoses of Fragile X Syndrome, Typical Development, or History of Premature Birth
* able to sit independently
* English is spoken at home
Exclusion Criteria:
* For all participants: no seizures in the past 6 months
* For typical development group and Fragile X group: not born prior to 32 weeks gestation OTHER: Speech discrimination
Fragile X Syndrome
Neuroimaging Reveals Treatment-related Changes in DLD
Kristi Barnett - Kristi.Barnett@cchmc.org
ALL
48 months to 71 months old
NA
NCT05268341
Inclusion Criteria:
* ages 48-71 months
* average nonverbal intelligence quotient (IQ)
* enrolled in participating center
* typically developing (receptive and expressive language, social, articulation, other)
* DLD for expressive grammar
* typical oral motor function
* typical social/pragmatics skills
* average hearing thresholds
* monolingual and native Standard English speakers
* does not have any uncorrected vision challenges
Exclusion Criteria:
* receiving co-occurring speech-language or other intervention for communication
* not MRI safe
* special education placement of child based on ability or behavior BEHAVIORAL: My Sentence Builder
Developmental Language Disorder
Heart Institute Biobank & Registry for Adult Congenital Heart Disease and Related Disorders (HIBR-ACHD)
Olivia Croweak - 0livia.croweak@cchmc.org
ALL
16 years and over
NCT07477197
Inclusion Criteria:
• Any person ≥ 16 years-old suspected of having or diagnosed with congenital heart disease (CHD), other cardiovascular disease (CVD), pulmonary hypertension, connective tissue disease, or genetic syndrome/diagnosis.
• Additionally, a cohort (Control group A) of control subjects will be enrolled, again ≥16 years-old, self-reported non-smokers without a known history of diabetes mellitus, myocardial infarction, stroke, heart failure, or chronic kidney disease. These controls will be either:
• A family member or other person accompanying a patient to a clinical encounter; or,
• A volunteer recruited via an advertisement; or,
• Another person who volunteers to enroll in HIBR-ACHD.
• A cohort (Control group B) of comparison subjects who do not have CHD, but have a diagnosis of heart failure or pulmonary hypertension.
Exclusion Criteria:
* Unable to provide informed consent/assent personally or via a legal guardian.
* Considered unsafe to collect the biospecimen determined by either a clinical provider or an HIBR-ACHD investigator.
* Overnight hospitalization for non-obstetric reason with discharge in the prior 30 days. Adult Congenital Heart Disease, Pulmonary Hypertension, Connective Tissue Disease, Other Cardiovascular Conditions
ACHD, Adult Congenital Heart Disease
Study to Learn About Safety, Tolerability, and Immune Response of a Catch-up Pneumococcal Vaccine in Children and Adolescents
- VaccineResearch@cchmc.org
ALL
15 months to 17 years old
PHASE3
NCT07660198
Inclusion Criteria:
Cohort 1 (Participants ≥15 Months to \<2 Years of Age) Healthy toddlers and children ≥15 months to \<2 years of age with documentation of prior receipt of PCV.
Cohort 2 (Participants ≥2 Years to \<5 Years of Age) Healthy toddlers and children ≥2 years to \<5 years of age with documentation of prior receipt of PCV if applicable.
Cohort 3 (Participants ≥5 Years to \<18 Years of Age) Healthy children ≥5 years to \<18 years of age with documentation of prior receipt of PCV (if applicable). A negative urine pregnancy test is required for individuals of childbearing potential (IOCBP). IOCBP or participants able to father must also agree to use a highly effective method of birth control.
Exclusion Criteria:
All Cohorts (Participants ≥15 Months to \<18 Years of Age)
Children with significant medical, psychiatric, or neurological conditions (eg, immunodeficiency or history of seizure); or a history of confirmed invasive pneumococcal infection in the past will be excluded from enrolment in the trial BIOLOGICAL: PG4, BIOLOGICAL: 20-valent pneumococcal conjugate vaccine (20vPnC)
Pneumococcal Disease
Assessing Pulmonary Changes in HSCT
Ashley Borden, MPH - ashley.borden@cchmc.org
ALL
3 years and over
PHASE1
NCT07823036
Inclusion Criteria:
* Ages 3 years and older
Exclusion Criteria:
* Pregnancy or positive pregnancy test
* Baseline oximetry at MRI visit of less than 90% on room air or less than 90% on a previously prescribed dosage of oxygen delivered by nasal cannula. In some cases of cardiorespiratory disease, if the participant is referred to this research study by their treating physician and considered stable for imaging, the cardiorespiratory criteria will be 5% of their clinical baseline. This should be documented in the research record upon referral of a participant that meets this scenario.
* Any condition in the opinion of the investigator that would make the participant unable to tolerate the MRI procedure (e.g., neurocognitive delay, behavioral issues).
* Standard MRI exclusions (e.g., metal, incompatible implants). DRUG: Hyperpolarized 129 Xenon
HSCT, Respiratory, Gas Exchange Impairment
Lorlatinib for Newly-Diagnosed High-Grade Glioma With ROS or ALK Fusion
Diarra Diop - diarra.diop@cchmc.org
ALL
1 year to 21 years old
EARLY_PHASE1
NCT06333899
Inclusion Criteria:
• Patients must be ≥ 12 months and ≤ 21 years of age at the time of study enrollment on TarGeT-SCR.
• Diagnosis: Patients with newly diagnosed high-grade glioma (HGG), including diffuse intrinsic pontine gliomas (DIPG), whose tumors harbor an ALK or ROS-1 fusion alteration are eligible. Patients must have had histologically verified high-grade glioma from diagnostic biopsy or resection. For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2/3 of the pons, with histopathology consistent with diffuse WHO Grade 2-4. All other HGGs must be Grade 3 or 4.
• Disease Status: Patients with disseminated DIPG or HGG are eligible only if the patient is to receive chemotherapy only, i.e. no craniospinal RT is intended to be given. MRI of spine must be performed if disseminated disease is suspected clinically by the treating physicians. Patients with primary spinal tumors are eligible only if the patient is to receive either chemotherapy or focal radiation therapy, i.e., no craniospinal RT is intended to be given. Patients with leptomeningeal disease only, with no definitive identifiable primary tumor, and documented ALK or ROS-1 fusion, must be discussed with the Study Chair on a case-by-case basis.
• Performance Level: Karnofsky ≥ 50% for patients \> 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age (See Appendix I). 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.
• Prior Therapy: * Patients must not have received any prior anti-cancer chemotherapy. * Prior use of corticosteroids is allowed (see below Exclusion Criteria)
• Organ Function Requirements 6.1 Adequate Bone Marrow Function Defined as: * Peripheral absolute neutrophil count (ANC) ≥ 1000/μL * Platelet count ≥ 100,000/μL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment) * Hemoglobin \>8 g/dL (may receive transfusions) 6.2 Adequate Renal Function Defined as: * Serum creatinine within normal institutional limits OR Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 6.3 Adequate Liver Function Defined as: * Total bilirubin ≤ 2 × institutional upper limit of normal * AST(aspartate aminotransferase)/ALT(alanine transaminase) ≤ 2.5 × institutional upper limit of normal 6.4 Adequate Pulmonary Function Defined as: Pulse oximetry \> 94% on room air if there is clinical indication for determination (e.g. dyspnea at rest).
• 5Adequate Cardiac Function Defined as: QTc ≤ 470 msec (by Bazett formula) 6.6 Adequate Neurologic Function Defined as: Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled.
• 7 Informed Consent: 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 or breast-feeding women will not be entered on this study due to unknown risks of fetal and teratogenic adverse events as seen in animal/human studies. 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 an effective contraceptive method. Females of reproductive potential must use an effective non-hormonal method of contraception, since lorlatinib can render hormonal contraceptives ineffective, during study treatment and for at least 6 months after the final dose. Males with female partners of reproductive potential must use effective contraception during treatment with lorlatinib and for 3 months after the final dose.
• Concomitant Medications * Investigational Agents/Drugs: Patients who have previously received or are currently receiving another investigational drug are not eligible. * Anti-cancer Agents: Patients who have previously received or are currently receiving other anti-cancer agents, including chemotherapy, immunotherapy, monoclonal antibodies, biologic or targeted therapy, are not eligible
• Infection: Patients must not have any active, uncontrolled systemic bacterial, viral or fungal infection.
• Patients who have received prior solid organ transplantation are not eligible.
• Patients must not have malabsorption syndrome or other condition affecting oral absorption.
• Patients must not be receiving any treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor or inducer. Discontinue strong CYP3A inducers for 3 plasma half-lives of the strong CYP3A inducer prior to treatment with loraltinib. Moderate inducers of CYP3A4 should be avoided
• Avoid concomitant use of lorlatinib with certain CYP3A substrates, for which minimal concentration changes may lead to serious therapeutic failures. If concomitant use is unavoidable, increase the CYP3A substrate dosage in accordance with approved product labeling.
• P-glycoprotein (P-gp) substrates: Lorlatinib is considered a moderate P-gp inducer. Co-administration of lorlatinib with P-gp substrates including but not limited to digoxin should be avoided as the concentration of these drugs may be reduced by lorlatinib.
• 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 a known personal history of acute or chronic severe psychiatric disorders or current history of suicidal ideation and history of suicide attempt.
DRUG: Lorlatinib, DRUG: Lorlatinib with chemotherapy1, DRUG: Lorlatinib with chemotherapy 2, DRUG: Lorlatinib post Radiation
High Grade Glioma, Diffuse Intrinsic Pontine Glioma, Anaplastic Astrocytoma, Infant Type Hemispheric Glioma, Glioblastoma, Glioblastoma Multiforme, WHO Grade III Glioma, WHO Grade IV Glioma, Diffuse Midline Glioma, H3K27-altered
ALK fusion, ROS fusion, High Grade Glioma, Diffuse Intrinsic Pontine Glioma
BEATRIX: A Study to Learn About a Group B Streptococcus Vaccine in Healthy Pregnant Women and Their Babies
Natalie Brinkmann - natalie.brinkmann@cchmc.org
ALL
1 day to 49 years old
PHASE3
NCT07160244
Key Inclusion criteria- Maternal:
* Healthy pregnant women ≤49 years of age who are between 24 0/7 and 36 0/7 weeks of gestation on the day of planned vaccination, with an uncomplicated, singleton pregnancy, and who have no known increased risk of complications.
* Had a fetal anomaly ultrasound examination with no significant fetal abnormalities observed.
* Documented negative human immunodeficiency virus (HIV) antibody test, syphilis test, and hepatitis B virus (HBV) surface antigen test during this pregnancy and prior to randomization.
* Capable of giving personal signed informed consent.
* Willing to give informed consent for her infant to participate in the study.
Key Exclusion criteria- Maternal:
* Prepregnancy body mass index (BMI) of \>40 kg/m2.
* Current pregnancy complications or abnormalities that may increase the risk associated with the participation in and completion of the study.
* Prior pregnancy complications or abnormalities that, based on the investigator's judgment, may increase the risk associated with the participation in and completion of the study.
* History of microbiologically proven invasive disease caused by GBS in the current pregnancy.
* A known or suspected infection during the current pregnancy that may increase the risk of complications in pregnancy (eg, active tuberculosis, syphilis, primary genital herpes simplex, malaria).
Key Inclusion criteria- Infant Participants
\- Evidence of a signed and dated ICD signed by the parent(s)/legally authorized representative or legal guardian
Key Exclusion Criteria - Infant Participants:
\- Children or grandchildren who are direct descendants of investigator site staff or sponsor and sponsor delegate employees directly involved in the conduct of the study.
Key Exclusion Criteria - Infant immunogenicity subset Participants:
\- Children with a known or suspected contraindication to any vaccine administered in the infant vaccine immunogenicity subset.
Refer to the study contact for further eligibility details
BIOLOGICAL: Multivalent Group B streptococcus vaccine, BIOLOGICAL: Placebo, BIOLOGICAL: Infanrix hexa, BIOLOGICAL: Prevenar 20, BIOLOGICAL: Pediarix, BIOLOGICAL: Prevnar 20, BIOLOGICAL: Infanrix
Healthy
group B streptococcus, maternal immunization, vaccine