Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Jun 10, 2026, 3:30 AM EDT

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Showing 1–2 of 2 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Enrolling by invitation Phase 1Phase 2 Interventional

Gene Therapy for IGHMBP2-Related Diseases

NCT05152823
Conditions
SMARD1, CMT2S
Interventions
Gene Therapy
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
2 Months to 14 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Completed No phase listed Observational Accepts healthy volunteers

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

NCT02532244
Conditions
Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
Interventions
sample collection
Other
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
1 Month and older
Enrollment
230 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2024
U.S. locations
3
States / cities
Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida