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ClinicalTrials.gov public records Last synced Jul 23, 2026, 5:32 PM EDT

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Showing 25–48 of 24 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 Accepts healthy volunteers

Airway Management of Pediatric Patients With Klippel-Feil Syndrome

NCT03741790
View directory record Official record
Conditions
Klippel-Feil Syndrome
Interventions
Ventilation, Intubation
Procedure
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
22
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Recruiting Phase 2 Interventional

Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System

NCT06080074
View directory record Official record
Conditions
Heart Failure, Cardiogenic Shock, Congenital Heart Disease, Cardiomyopathies
Interventions
Cardiohelp device (VA-ECMO), Heparin, Bivalirudin
Device · Drug
Lead sponsor
Stanford University
Other
Eligibility
0 Years to 16 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Palo Alto, California • New York, New York • Durham, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Recruiting No phase listed Observational

Biomarkers of Resiliency In Childhood Cancer Surgery

NCT06674811
View directory record Official record
Conditions
Pediatric Cancer, Neuroblastoma, Sarcoma, Wilms Tumor and Other Childhood Kidney Tumors, Childhood Soft Tissue Sarcoma, Childhood Bone Sarcoma, Childhood Malignant Intestinal Neoplasm, Childhood Malignant Liver Neoplasm, Childhood Malignant Lung Neoplasm, Childhood Malignant Ovarian Neoplasm, Childhood Malignant Pleural Neoplasm, Childhood Neuroblastic Tumor, Childhood Sarcoma
Interventions
Wearing Smartwatch, Patient Report Outcomes, Blood Draw, Clinical Data Review
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
3 Years to 25 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 2 Interventional Results available

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

NCT00074958
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
7 Years to 15 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 1, 2015 · Synced Jul 23, 2026, 5:32 PM EDT
No Longer Available No phase listed Expanded access

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

NCT03639844
View directory record Official record
Conditions
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
Interventions
rivogenlecleucel, rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
3 Months to 21 Years
U.S. locations
2
States / cities
Los Angeles, California • Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2020 · Synced Jul 23, 2026, 5:32 PM EDT
Enrolling by invitation Not applicable Interventional

EXCOR Active Driving System for the EXCOR Pediatric VAD IDE Study

NCT05610787
View directory record Official record
Conditions
Heart Failure, Transplant; Failure, Heart, Congenital Heart Disease
Interventions
EXCOR Active Driving System for Pediatric VAD
Device
Lead sponsor
Berlin Heart, Inc
Industry
Eligibility
Up to 21 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
15
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 3 Interventional Results available

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

NCT00006400
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell
Interventions
Hydroxyurea, Placebo
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
9 Months to 18 Months
Enrollment
193 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2020 · Synced Jul 23, 2026, 5:32 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475
View directory record Official record
Conditions
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Interventions
PBGM01
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 24 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
4
States / cities
Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 1 Interventional

Delgocitinib Cream for the Treatment of Moderate to Severe Atopic Dermatitis During 8 Weeks in Adults, Adolescents, and Children

NCT03826901
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
delgocitinib
Drug
Lead sponsor
LEO Pharma
Industry
Eligibility
2 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
12
States / cities
Birmingham, Alabama • Phoenix, Arizona • Fountain Valley, California + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2025 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 1Phase 2 Interventional

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

NCT04259281
View directory record Official record
Conditions
Angelman Syndrome
Interventions
GTX-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
4 Years to 17 Years
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
6
States / cities
Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
No Longer Available No phase listed Expanded access

HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

NCT05154851
View directory record Official record
Conditions
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
Interventions
HB-adMSCs
Biological
Lead sponsor
Hope Biosciences Research Foundation
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Sugar Land, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

NCT02603562
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
ATYR1940
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
16 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
4
States / cities
Stanford, California • Iowa City, Iowa • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Jul 23, 2026, 5:32 PM EDT
Terminated No phase listed Observational

Screening for Lysosomal Acid Lipase Deficiency

NCT02926872
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
There is no intervention in the study
Other
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 16 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Atlanta, Georgia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 15, 2017 · Synced Jul 23, 2026, 5:32 PM EDT
Completed No phase listed Observational

Mucopolysaccharidosis Type II Natural History

NCT03529786
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Jul 23, 2026, 5:32 PM EDT
Terminated No phase listed Observational

Incidence of Neo-Aortic Insufficiency

NCT00268073
View directory record Official record
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
Up to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 6, 2007 · Synced Jul 23, 2026, 5:32 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Liver Adiposity Effects on Pediatric Statin

NCT04903223
View directory record Official record
Conditions
Cholesterol; Lipidosis
Interventions
Rosuvastatin 10mg
Drug
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
8 Years to 21 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 2 Interventional Results available

CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

NCT01095510
View directory record Official record
Conditions
Hereditary Angioedema (HAE)
Interventions
CINRYZE
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
8
States / cities
Colorado Springs, Colorado • Tampa, Florida • Chevy Chase, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2021 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Not applicable Interventional

Distracting Through Procedural Pain and Distress

NCT04892160
View directory record Official record
Conditions
Chronic Illness, Hematologic Malignancy, Bone Marrow Transplant Infection, Oncology, Sickle Cell Disease
Interventions
Guided Imagery, Virtual Reality
Other
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
8 Years to 25 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
2
States / cities
Milwaukee, Wisconsin • Wauwatosa, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 18, 2021 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Not applicable Interventional Results available

Neonatal Intensive Care Unit Virtual Family-Centered Rounds

NCT05762835
View directory record Official record
Conditions
Pediatric Disorder, Neonatal Disease
Interventions
Virtual family-centered rounds (FCR)
Behavioral
Lead sponsor
University of California, Davis
Other
Eligibility
Not listed
Enrollment
514 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Aug 16, 2025 · Synced Jul 23, 2026, 5:32 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial

NCT04284774
View directory record Official record
Conditions
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Kidney Wilms Tumor, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Medulloblastoma, Recurrent Melanoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdoid Tumor of the Kidney, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Carcinoma, Recurrent WHO Grade 2 Glioma, Refractory Adrenal Gland Pheochromocytoma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Medulloblastoma, Refractory Melanoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdoid Tumor of the Kidney, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Carcinoma, Refractory WHO Grade 2 Glioma
Interventions
Tipifarnib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
171
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Terminated Not applicable Interventional Results available

Low-dose Epinephrine Infusion Tests in Adolescent and Pediatric Patients

NCT00548886
View directory record Official record
Conditions
Congenital Disorders
Interventions
Epinephrine
Drug
Lead sponsor
Emory University
Other
Eligibility
3 Years to 18 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Jul 23, 2026, 5:32 PM EDT
Recruiting No phase listed Observational

Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders

NCT07493096
View directory record Official record
Conditions
Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD), Neurodevelopmental Disorders and Developmental Abnormalities, Developmental Delay (Disorder), Cerebral Palsy (CP), Cerebral Palsy Hemiparetic Cerebral Palsy Spasticity Gait Disorders, Neurologic Postural Balance Impairment, Cerebral Palsy Infantile, Cerebral Palsy Spastic Hemiplegic, Cerebral Palsy, Dyskinetic, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD, Hypoxic Ischemic Encephalopathy, Hypoxic Ischemic Encephalopathy (HIE), Traumatic Brain Injury (TBI), Sensory Processing Disorder, Chromosomal Abnormalities, Genetic Disorders, Down Syndrome (Trisomy 21), Fragile X Syndrome (FXS), RETT Syndrome With Proven MECP2 Mutation, Williams Syndrome, 22q11.2 Deletion Syndrome, Sensorimotor Integration
Interventions
Not listed
Lead sponsor
Healing Hope International
Other
Eligibility
4 Years to 12 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
The Woodlands, Texas
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Jul 23, 2026, 5:32 PM EDT
Completed Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy and Safety of IDP-124 Lotion for the Treatment of Moderate to Severe Atopic Dermatitis in Pediatric and Adult Subjects

NCT03058783
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
IDP-124 Lotion, IDP-124 Vehicle Lotion
Drug
Lead sponsor
Bausch Health Americas, Inc.
Industry
Eligibility
2 Years and older
Enrollment
328 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fremont, California
Source: ClinicalTrials.gov public record
Updated May 7, 2023 · Synced Jul 23, 2026, 5:32 PM EDT