Clinical Trial

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

Active, Not 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 2 (SMA 2) patients.
Protocol Amendment History 6 amendments
This ClinicalTrials.gov record has been amended 6 times since 2023-06-04; most recent amendment 2025-06-30.
Status change: Recruiting → Active, Not Recruiting 2025-06-30
Status change: Not Yet Recruiting → Recruiting 2023-09-11
Trial Details
NCT Number NCT05901987
Lead Sponsor GeneCradle Inc
Conditions SMA II
Enrollment 33 participants
Start Date 2023-08-01
Primary Completion 2025-04-22 (estimated)
Study Completion 2028-12 (estimated)
Updated on ClinicalTrials.gov 2025-07-03