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Showing 1–24 of 127 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Neuromuscular Diseases in Children Clear all
Active, not recruiting Not applicable Interventional

Sickle Cell Children's Exercise Study (SuCCESs)

NCT06074198
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
SuCCESs
Behavioral
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
6 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
2
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Jul 24, 2026, 7:46 AM 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 10, 2011 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Phase 3 Interventional Results available

Dose-response Study of Efficacy and Safety of Botulinum Toxin Type A to Treat Spasticity of the Arm(s) or of Arm(s) and Leg(s) in Cerebral Palsy

NCT02002884
View directory record Official record
Conditions
Cerebral Palsy, Spasticity
Interventions
IncobotulinumtoxinA (8 Units per kg body weight), IncobotulinumtoxinA (6 Units per kg body weight), IncobotulinumtoxinA (2 Units per kg body weight)
Drug
Lead sponsor
Merz Pharmaceuticals GmbH
Industry
Eligibility
2 Years to 17 Years
Enrollment
351 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
6
States / cities
Gulf Breeze, Florida • Loxahatchee Groves, Florida • Savannah, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Not applicable Interventional Results available

Fibromyalgia Integrative Training for Adolescents With Juvenile Fibromyalgia

NCT03268421
View directory record Official record
Conditions
Fibromyalgia, Myofascial Pain Syndrome, Muscular Diseases, Musculoskeletal Disease, Rheumatic Diseases
Interventions
Fibromyalgia Integrative Training for Teens, Cognitive Behavioral Therapy, Graded Aerobic Exercise
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
12 Years to 18 Years
Enrollment
389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Hartford, Connecticut • Indianapolis, Indiana • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2025 · Synced Jul 24, 2026, 7:46 AM 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 7, 2025 · Synced Jul 24, 2026, 7:46 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Behavioral Effects of Obstructive Sleep Apnea in Children

NCT00060723
View directory record Official record
Conditions
Sleep-disordered Breathing, Sleep Apnea, Obstructive, ADHD
Interventions
Not listed
Lead sponsor
University of Michigan
Other
Eligibility
5 Years to 12 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2004
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 5, 2016 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Phase 3 Interventional Results available

Dysport® Pediatric Lower Limb Spasticity Study

NCT01249417
View directory record Official record
Conditions
Cerebral Palsy, Muscle Spasticity, Children
Interventions
Botulinum toxin type A, Placebo
Biological · Drug
Lead sponsor
Ipsen
Industry
Eligibility
2 Years to 17 Years
Enrollment
241 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
8
States / cities
Aurora, Colorado • Chicago, Illinois • New Orleans, Louisiana + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2022 · Synced Jul 24, 2026, 7:46 AM EDT
Recruiting Phase 2Phase 3 Interventional

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

NCT04833894
View directory record Official record
Conditions
Generalized Myasthenia Gravis
Interventions
Efgartigimod IV
Biological
Lead sponsor
argenx
Industry
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
3
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Not applicable Interventional Results available

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials

NCT03531814
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Medication Event Monitoring System (MEMS^TM), Questionnaires
Behavioral · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 59 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 10, 2024 · Synced Jul 24, 2026, 7:46 AM 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 26, 2025 · Synced Jul 24, 2026, 7:46 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
View directory record Official record
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 8, 2025 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 Mutations

NCT02124772
View directory record Official record
Conditions
Cancer
Interventions
Trametinib, Dabrafenib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Month to 17 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
10
States / cities
Phoenix, Arizona • San Francisco, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2021 · Synced Jul 24, 2026, 7:46 AM EDT
Terminated No phase listed Observational

Heart Imaging in Children With Muscular Dystrophy

NCT01633242
View directory record Official record
Conditions
Muscular Dystrophy
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
8 Years to 100 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 12, 2019 · Synced Jul 24, 2026, 7:46 AM EDT
Recruiting Phase 2Phase 3 Interventional

A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis

NCT05265273
View directory record Official record
Conditions
Myasthenia Gravis
Interventions
Nipocalimab
Drug
Lead sponsor
Janssen Research & Development, LLC
Industry
Eligibility
2 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
11
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Not applicable Interventional

A New Training to Enhance Physical Activity in Adolescents With Cerebral Palsy

NCT05865418
View directory record Official record
Conditions
Cerebral Palsy, Muscle Disorder, Physical Disability, Posture Disorders in Children, Spina Bifida
Interventions
Loaded high intensity training, High intensity circuit training (HICT)
Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
11 Years to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated May 24, 2025 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Not applicable Interventional

Evaluating a New Knee-Ankle-Foot Brace to Improve Gait in Children With Movement Disorders

NCT01961557
View directory record Official record
Conditions
Incomplete Spinal Cord Injury, Muscular Dystrophy, Spina Bifida, Cerebral Palsy
Interventions
EA-KAFO
Device
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
5 Years to 100 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Recruiting No phase listed Observational

Patterns of Neurodevelopmental Disorders

NCT06213090
View directory record Official record
Conditions
Neurodevelopmental Disorders, Autism Spectrum Disorder, Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection, Pediatric Acute-Onset Neuropsychiatric Syndrome, Down Syndrome, Epilepsy, Mitochondrial Encephalomyopathies, Cerebral Folate Deficiency
Interventions
Not listed
Lead sponsor
Richard Frye
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

NCT05309668
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Selumetinib granule formulation, Selumetinib capsule formulation
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
1 Year to 6 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
4
States / cities
Akron, Ohio • Philadelphia, Pennsylvania • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

NCT01362803
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, Plexiform
Interventions
AZD6244
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 18 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2032
U.S. locations
4
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Recruiting Phase 2Phase 3 Interventional

A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis

NCT06392386
View directory record Official record
Conditions
Generalized Myasthenia Gravis
Interventions
Efgartigimod PH20 SC
Biological
Lead sponsor
argenx
Industry
Eligibility
2 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
3
States / cities
Charlotte, North Carolina • Denton, Texas • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Recruiting Phase 2Phase 3 Interventional

IntraVenous Iron in Kids With Iron Deficiency and Scoliosis Study

NCT07217873
View directory record Official record
Conditions
Scoliosis Correction, Iron Deficiency, Transfusion Blood, Surgery Complications, Spinal Fusion, Scoliosis Idiopathic Adolescent, Scoliosis Neuromuscular
Interventions
Ferric carboxymaltose IV, Normal Saline (Placebo)
Drug
Lead sponsor
Columbia University
Other
Eligibility
10 Years to 19 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 25, 2025 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy Study in Preschool Children Aged 4-5 Years With Attention-deficit/Hyperactivity Disorder (ADHD)

NCT03260205
View directory record Official record
Conditions
Attention Deficit Hyperactivity Disorder (ADHD)
Interventions
Placebo, SPD489 (Lisdexamfetamine dimesylate), SPD489
Drug
Lead sponsor
Shire
Industry
Eligibility
4 Years to 5 Years
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
48
States / cities
Dothan, Alabama • Little Rock, Arkansas • Colton, California + 39 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced Jul 24, 2026, 7:46 AM 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 26, 2026 · Synced Jul 24, 2026, 7:46 AM EDT
Completed Not applicable Interventional

A Single Center Pilot Study to Assess the Intra-observer Reliability of Measuring Muscle Strength Using a Hand Held Dynamometer in Children and Adults With Neurofibromatosis Type 1 (NF1) and Type 2 (NF2)

NCT03672838
View directory record Official record
Conditions
Neurofibromatosis 1 and 2 (NF1 and NF2)
Interventions
hand-held dynamometer
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Jul 24, 2026, 7:46 AM EDT