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ClinicalTrials.gov public records Last synced Jul 23, 2026, 2:31 PM EDT

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Showing 1–24 of 695 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Congenital Pediatric Disorders Clear all
Active, not recruiting No phase listed Observational

Pulmonary Vascular Disease in CF

NCT04549077
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years to 21 Years
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Indianapolis, Indiana • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

NCT06152237
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Adolescent, Caregiver, and Young Adult Perspectives of the Transition From Pediatric to Adult Care for Sickle Cell Disease: A Preliminary Evaluation of the Sickle Cell Disease Transition Program

NCT01569971
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Assessment
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Years to 30 Years
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 23, 2014 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Exercise Capacity in Pediatric Sickle Cell Anemia

NCT01527799
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
8 Years to 21 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 3, 2016 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational

Genotype-Phenotype Associations in Pediatric Cardiomyopathy (PCM GENES)

NCT01873963
View directory record Official record
Conditions
Dilated Cardiomyopathy, Hypertrophic Cardiomyopathy, Restrictive Cardiomyopathy
Interventions
Not listed
Lead sponsor
Wayne State University
Other
Eligibility
Not listed
Enrollment
544 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
11
States / cities
Aurora, Colorado • Miami, Florida • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2018 · Synced Jul 23, 2026, 2:31 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

NCT03769116
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Los Angeles, California • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational

Automated Cardioverter Defibrillator in Children

NCT00268021
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 21 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 1, 2014 · Synced Jul 23, 2026, 2:31 PM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

NCT02715115
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
5 Years to 15 Years · Female only
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2020 · Synced Jul 23, 2026, 2:31 PM EDT
Active, not recruiting Phase 1 Interventional

Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)

NCT04236479
View directory record Official record
Conditions
Congenital Heart Disease (CHD)
Interventions
BM-MSC
Biological
Lead sponsor
Catherine Bollard
Other
Eligibility
Up to 6 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational

Re-operative Surgery in Children:A Technique for Sternal Re-Entry

NCT00328146
View directory record Official record
Conditions
Congenital Disorders
Interventions
Observation of sternal reentry
Other
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 18 Years
Enrollment
802 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 28, 2017 · Synced Jul 23, 2026, 2:31 PM EDT
Recruiting Phase 1Phase 2 Interventional

Alpha/Beta T and B Cell Depletion With Zoledronic Acid for Solid Tumors

NCT06625190
View directory record Official record
Conditions
Neuroblastoma, Rhabdomyosarcoma, Synovial Sarcoma, Peripheral Nerve Sheath Tumors, Clear Cell Sarcoma, Alveolar Soft Part Sarcoma, Desmoplastic Small Round Cell Tumor, Chordoma, Rhabdoid Tumor, Epithelioid Sarcoma, Myoepithelial Tumor, Osteosarcoma, Ewing Sarcoma, Wilms Tumor
Interventions
Miltenyi CliniMACS Prodigy ® system, Zoledronic acid
Device · Drug
Lead sponsor
University of Florida
Other
Eligibility
6 Months to 25 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Metabolic and Hemodynamic Reserve in Pediatric SCA

NCT04406818
View directory record Official record
Conditions
Child, Only, Brain Diseases, Sickle Cell Disease, Anemia, Sickle Cell
Interventions
Carbon Dioxide
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
4 Years to 21 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Jul 23, 2026, 2:31 PM EDT
Recruiting No phase listed Observational

A Prospective Sub-Study of the Global Hypophosphatasia Registry

NCT05234567
View directory record Official record
Conditions
Hypophosphatasia
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
12
States / cities
Hartford, Connecticut • Chicago, Illinois • Boston, Massachusetts + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Terminated Phase 2 Interventional Results available

Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome

NCT03732534
View directory record Official record
Conditions
Tourette Syndrome
Interventions
NBI-98854
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 18 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
9
States / cities
Anaheim, California • San Diego, California • Gulf Breeze, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 18, 2022 · Synced Jul 23, 2026, 2:31 PM EDT
Completed Phase 2 Interventional Results available

Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)

NCT02797821
View directory record Official record
Conditions
Hypophosphatasia
Interventions
Asfotase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
3
States / cities
St Louis, Missouri • Durham, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 16, 2019 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational

Iron Overload in Stem Cell Transplant Recipients

NCT00806715
View directory record Official record
Conditions
Iron Overload
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
5 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2018 · Synced Jul 23, 2026, 2:31 PM EDT
Completed Phase 2 Interventional Results available

Arsenic Trioxide in Treating Patients With Advanced Neuroblastoma or Other Childhood Solid Tumors

NCT00024258
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Retinoblastoma, Sarcoma
Interventions
arsenic trioxide
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 40 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 24, 2015 · Synced Jul 23, 2026, 2:31 PM EDT
Recruiting Phase 3 Interventional

Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy

NCT07220785
View directory record Official record
Conditions
Generalized Lipodystrophy
Interventions
Mibavademab, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Ann Arbor, Michigan • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

NCT05255445
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Pediatric Cancer
Interventions
Red Blood Cell (RBC) Transfusion
Biological
Lead sponsor
Westat
Other
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Oakland, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Jul 23, 2026, 2:31 PM EDT
Recruiting Not applicable Interventional

The ORIGIN-FH Study

NCT07470723
View directory record Official record
Conditions
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Familial Hypercholesterolemia
Interventions
Screening for FH
Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

NCT03974113
View directory record Official record
Conditions
Hemophilia
Interventions
Fitusiran
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
1 Year to 11 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
4
States / cities
Los Angeles, California • Hackensack, New Jersey • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Completed No phase listed Observational

Natural History Study of SLC25A46 Mutation-related Mitochondriopathy

NCT04594590
View directory record Official record
Conditions
Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
Interventions
Mutation analysis
Genetic
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
0 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Mar 7, 2024 · Synced Jul 23, 2026, 2:31 PM EDT
Recruiting No phase listed Observational

Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

NCT06847282
View directory record Official record
Conditions
Muscular Dystrophy, Facioscapulohumeral
Interventions
Not listed
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
5 Years to 17 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
6
States / cities
Palo Alto, California • Iowa City, Iowa • Kansas City, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2026 · Synced Jul 23, 2026, 2:31 PM EDT
Active, not recruiting Phase 3 Interventional

Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)

NCT04944979
View directory record Official record
Conditions
Primary Immunodeficiency Disease
Interventions
Kedrion IVIG 10%
Biological
Lead sponsor
Kedrion S.p.A.
Industry
Eligibility
2 Years to 16 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
8
States / cities
San Francisco, California • Centennial, Colorado • St. Petersburg, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jul 23, 2026, 2:31 PM EDT