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ClinicalTrials.gov public records Last synced Oct 13, 2026, 3:18 PM EDT

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Showing 49–72 of 128 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neuromuscular Diseases in Children Clear all
Withdrawn No phase listed Observational Accepts healthy volunteers

Stem Cells in NF1 Patients With Tumors of the Central Nervous System

NCT03332030
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tumors of the Central Nervous System
Interventions
Collection of Stem Cells
Diagnostic Test
Lead sponsor
Roger Packer
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Not applicable Interventional

Body Weight Support Harness System in Spinal Muscular Atrophy

NCT05715749
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Interventions
In-home body weight support harness system
Other
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 22, 2024 · Synced Oct 13, 2026, 3:18 PM EDT
Active, not recruiting No phase listed Observational

Defining Endpoints in Becker Muscular Dystrophy

NCT05257473
View directory record Official record
Conditions
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years and older · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Recruiting Phase 3 Interventional

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

NCT07608432
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Duchenne Type, Muscular Dystrophy, Duchenne and Becker Types, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Genetic Disease, Inborn, Genetic Disease, X-Linked, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neuromuscular Diseases (NMD)
Interventions
Zeleciment Rostudirsen (DYNE-251), Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Hillsborough, North Carolina
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1

NCT04481048
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-Acetyl cysteine, Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2024
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)

NCT06987539
View directory record Official record
Conditions
Generalized Myasthenia Gravis
Interventions
Inebilizumab
Drug
Lead sponsor
Amgen
Industry
Eligibility
2 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Philadelphia, Pennsylvania • Austin, Texas • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Recruiting Not applicable Interventional

Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders

NCT05726591
View directory record Official record
Conditions
Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Incomplete Spinal Cord Injury
Interventions
EA-KAFO
Device
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
3 Years to 17 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2025 · Synced Oct 13, 2026, 3:18 PM EDT
Recruiting Phase 2 Interventional

Vasodilator and Exercise Study for DMD (VASO-REx)

NCT06290713
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children, Vasodilation, Exercise, DMD
Interventions
Tadalafil, Placebo, Exercise Training
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Terminated Not applicable Interventional Results available

The Effects of Ankle Foot Orthoses on Gait Efficiency in Children With Acute Lymphoblastic Leukemia and Foot Drop

NCT01001390
View directory record Official record
Conditions
Foot Drop, Acute Lymphoblastic Leukemia
Interventions
AFO Device
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
6 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 27, 2014 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Offset Analgesia as a Measure of Central Sensitization in Children

NCT03445403
View directory record Official record
Conditions
Chronic Pain Syndrome, Chronic Daily Headache, Functional Abdominal Pain Syndrome, Musculoskeletal Pain Disorder, CRPS (Complex Regional Pain Syndromes)
Interventions
Offset analgesia
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
10 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Waltham, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 13, 2026, 3:18 PM EDT
Terminated Not applicable Interventional

Does Wearing Tetra-Grip Improve Arm Function in Children Diagnosed With Neonatal Brachial Plexus Palsy?

NCT03647761
View directory record Official record
Conditions
Neonatal Brachial Plexus Palsy
Interventions
Tetra-grip
Device
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
3 Years to 7 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 5, 2022 · Synced Oct 13, 2026, 3:18 PM EDT
Completed No phase listed Observational

Use of EMG to Assess Clinical Hypertonia

NCT00472914
View directory record Official record
Conditions
Cerebral Palsy, Hypertonia, Spasticity, Dystonia, Rigidity
Interventions
portable surface electromyography
Device
Lead sponsor
University of Southern California
Other
Eligibility
6 Years to 18 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 23, 2014 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Not applicable Interventional Results available

The Summer Camp Study 2: Blood Glucose Control With a Bi-Hormonal Endocrine Pancreas

NCT02105324
View directory record Official record
Conditions
Type 1 Diabetes
Interventions
Bionic Pancreas, Usual Care
Device · Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
6 Years to 11 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 20, 2017 · Synced Oct 13, 2026, 3:18 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2010 · Synced Oct 13, 2026, 3:18 PM EDT
Enrolling by invitation Phase 2Phase 3 Interventional

Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis

NCT05374590
View directory record Official record
Conditions
Generalized Myasthenia Gravis, gMG
Interventions
Efgartigimod IV or Efgartigimod PH20 SC
Biological
Lead sponsor
argenx
Industry
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Chicago, Illinois • Charlotte, North Carolina • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Tideglusib, Placebo
Drug
Lead sponsor
AMO Pharma Limited
Industry
Eligibility
6 Years to 16 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
9
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Phase 1Phase 2 Interventional

Oral Baclofen Pharmacokinetics and Pharmacodynamics in Children With Spasticity

NCT00607542
View directory record Official record
Conditions
Spasticity, Cerebral Palsy
Interventions
baclofen
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 16 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
11
States / cities
Chicago, Illinois • New Orleans, Louisiana • Baltimore, Maryland + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2011 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1

NCT04481035
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
N-acetylcysteine (NAC), Placebo
Drug · Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years to 16 Years
Enrollment
5 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Ultrasonographic Assessment of Carpal Tunnel Syndrome

NCT02196220
View directory record Official record
Conditions
Carpal Tunnel Syndrome
Interventions
Not listed
Lead sponsor
University of Utah
Other
Eligibility
3 Years to 12 Years
Enrollment
44 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2019
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Aug 20, 2019 · Synced Oct 13, 2026, 3:18 PM EDT
Active, not recruiting Phase 1 Interventional

EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults

NCT03618381
View directory record Official record
Conditions
Pediatric Solid Tumor, Germ Cell Tumor, Retinoblastoma, Hepatoblastoma, Wilms Tumor, Rhabdoid Tumor, Carcinoma, Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Synovial Sarcoma, Clear Cell Sarcoma, Malignant Peripheral Nerve Sheath Tumors, Desmoplastic Small Round Cell Tumor, Soft Tissue Sarcoma, Neuroblastoma
Interventions
second generation 4-1BBζ EGFR806-EGFRt, second generation 4-1BBζ EGFR806-EGFRt and a second generation 4 1BBζ CD19-Her2tG
Biological
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
1 Year to 30 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2040
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Spinal Cord Stimulation for Children With Cerebral Palsy

NCT07516067
View directory record Official record
Conditions
Cerebral Palsy, Spasticity
Interventions
Transcutaneous Spinal Cord Stimulation (tSCS)
Device
Lead sponsor
Martin Piazza
Other
Eligibility
5 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Not applicable Interventional

Hippotherapy to Improve the Balance of Children With Movement Disorders

NCT01313325
View directory record Official record
Conditions
Neuromuscular Diseases, Cerebral Palsy, Down Syndrome, Autism, Developmental Coordination Disorder
Interventions
Hippotherapy
Other
Lead sponsor
Central Michigan University
Other
Eligibility
5 Years to 17 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Dansville, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 11, 2011 · Synced Oct 13, 2026, 3:18 PM EDT
Active, not recruiting Phase 2 Interventional

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

NCT05540860
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3, Sevasemten Dose 4, Sevasemten Dose 5, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
4 Years to 9 Years · Male only
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 13, 2026, 3:18 PM EDT
Completed Phase 2 Interventional Results available

Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas

NCT03231306
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibroma
Interventions
Binimetinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Year and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
22
States / cities
Birmingham, Alabama • Los Angeles, California • Oakland, California + 16 more
Source: ClinicalTrials.gov public record
Updated May 2, 2025 · Synced Oct 13, 2026, 3:18 PM EDT