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Showing 1–2 of 2 matching trials from the live ClinicalTrials.gov search.
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
Conditions
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Interventions
VCA-894A
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Madison, Wisconsin