Clinical Trial

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Recruiting Phase 1/2
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Summary
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2024-05-14; most recent amendment 2025-07-01.
Trial Details
NCT Number NCT06421831
Lead Sponsor GeneCradle Inc
Conditions Spinal Muscular Atrophy Type 3
Enrollment 21 participants
Start Date 2024-05-10
Primary Completion 2026-12 (estimated)
Study Completion 2028-12 (estimated)
Updated on ClinicalTrials.gov 2025-07-03