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ClinicalTrials.gov public records Last synced Oct 13, 2026, 2:48 PM EDT

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Showing 25–48 of 128 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neuromuscular Diseases in Children Clear all
Active, not recruiting Phase 1 Interventional

Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT05101148
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Selumetinib
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
12 Years to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
2
States / cities
Rochester, Minnesota • Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 20, 2026 · Synced Oct 13, 2026, 2:48 PM EDT
Completed No phase listed Observational

Cardiac Outcome Measures in Children With Muscular Dystrophy

NCT01066455
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
8 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
5
States / cities
Sacramento, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2013 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Phase 1 Interventional

Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders

NCT00010335
View directory record Official record
Conditions
Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis, Juvenile Rheumatoid Arthritis, Autoimmune Diseases
Interventions
Stem Cell Transplantation, CD34 selection
Procedure · Device
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
2 Years to 18 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 6, 2015 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of SHP465 at 6.25 mg in the Treatment of Attention-Deficit/Hyperactivity Disorder (ADHD) in Children Aged 6-12 Years

NCT03325881
View directory record Official record
Conditions
Attention Deficit Hyperactivity Disorder (ADHD)
Interventions
SHP465, Placebo
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Years to 12 Years
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
41
States / cities
Dothan, Alabama • Little Rock, Arkansas • Anaheim, California + 36 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2021 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Not applicable Interventional

Load Experienced While Using a Stander in Children With Cerebral Palsy

NCT01952496
View directory record Official record
Conditions
Cerebral Palsy
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
Up to 17 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 17, 2016 · Synced Oct 13, 2026, 2:48 PM EDT
Recruiting Phase 1 Interventional

Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...

NCT02390752
View directory record Official record
Conditions
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Promyelocytic, Acute, Sarcoma
Interventions
TURALIO(R)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 35 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Early Phase 1 Interventional

Ketones & Mitochondrial Heteroplasmy

NCT01252979
View directory record Official record
Conditions
MELAS Syndrome, Mitochondrial Diseases
Interventions
Medium-Chain Triglycerides
Dietary Supplement
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 6, 2012 · Synced Oct 13, 2026, 2:48 PM 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 18, 2026 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Not applicable Interventional

Methylphenidate in Children and Adolescents With Pervasive Developmental Disorders

NCT00025779
View directory record Official record
Conditions
Attention Deficit Disorder With Hyperactivity, Autistic Disorder, Pervasive Development Disorders
Interventions
methylphenidate, guanfacine
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
5 Years to 14 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
5
States / cities
Los Angeles, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2009 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Phase 2 Interventional Results available

Lorvotuzumab Mertansine in Treating Younger Patients With Relapsed or Refractory Wilms Tumor, Rhabdomyosarcoma, Neuroblastoma, Pleuropulmonary Blastoma, Malignant Peripheral Nerve Sheath Tumor, or Synovial Sarcoma

NCT02452554
View directory record Official record
Conditions
Pleuropulmonary Blastoma, Recurrent Malignant Peripheral Nerve Sheath Tumor, Recurrent Neuroblastoma, Recurrent Rhabdomyosarcoma, Recurrent Synovial Sarcoma, Wilms Tumor
Interventions
Laboratory Biomarker Analysis, Lorvotuzumab Mertansine, Pharmacological Study
Other · Biological
Lead sponsor
Children's Oncology Group
Network
Eligibility
12 Months to 30 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
69
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Downey, California + 60 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2022 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Phase 2 Interventional Results available

Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults

NCT02101736
View directory record Official record
Conditions
NF1, Neurofibromatosis, Plexiform Neurofibromas
Interventions
Cabozantinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
12
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated May 24, 2023 · Synced Oct 13, 2026, 2:48 PM EDT
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 21, 2026 · Synced Oct 13, 2026, 2:48 PM EDT
Terminated Phase 3 Interventional Results available

Safety and Tolerability Study of SHP465 in Children Aged 4 to 12 Years Diagnosed With Attention-deficit/Hyperactivity Disorder (ADHD)

NCT03325894
View directory record Official record
Conditions
Attention Deficit Hyperactivity Disorder (ADHD)
Interventions
SHP465
Drug
Lead sponsor
Shire
Industry
Eligibility
4 Years to 12 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
48
States / cities
Dothan, Alabama • Little Rock, Arkansas • Anaheim, California + 43 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2021 · Synced Oct 13, 2026, 2:48 PM 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 – 2027
U.S. locations
3
States / cities
Charlotte, North Carolina • Denton, Texas • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 13, 2026, 2:48 PM 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 31, 2026 · Synced Oct 13, 2026, 2:48 PM 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 6, 2016 · Synced Oct 13, 2026, 2:48 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NCT00439569
View directory record Official record
Conditions
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
5
States / cities
Stanford, California • St Louis, Missouri • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2010 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Not applicable Interventional Results available

Progressive Strength Training in Spinal Muscular Atrophy

NCT01233817
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Progressive strength training
Other
Lead sponsor
University of Utah
Other
Eligibility
5 Years to 21 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jun 14, 2017 · Synced Oct 13, 2026, 2:48 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Development of a Proxy Motor Outcome Measure in Young Children With Neuromuscular Disease

NCT02007213
View directory record Official record
Conditions
Neuromuscular Disease
Interventions
Not listed
Lead sponsor
National Institute of Nursing Research (NINR)
NIH
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 13, 2026, 2:48 PM EDT
Recruiting Phase 2 Interventional

Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

NCT07766980
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD, Muscular Dystrophy
Interventions
VM100 Nutritional Supplement
Drug
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 13, 2026, 2:48 PM EDT
Completed Not applicable Interventional

Therapeutic Potential of Myofascial Structural Integration in Children With Cerebral Palsy

NCT01815814
View directory record Official record
Conditions
Spastic Cerebral Palsy
Interventions
Rolfing / Myofascial Structural Integration
Other
Lead sponsor
Stanford University
Other
Eligibility
1 Year to 3 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jul 8, 2015 · Synced Oct 13, 2026, 2:48 PM EDT
Recruiting Not applicable Interventional

HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness

NCT07488338
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST)
Other
Lead sponsor
Teachers College, Columbia University
Other
Eligibility
5 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 13, 2026, 2:48 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional Results available

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)

NCT03199469
View directory record Official record
Conditions
X-Linked Myotubular Myopathy
Interventions
Resamirigene bilparvovec
Genetic
Lead sponsor
Astellas Gene Therapies
Industry
Eligibility
Up to 5 Years · Male only
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 13, 2026, 2:48 PM 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 5, 2021 · Synced Oct 13, 2026, 2:48 PM EDT