Clinical Trial

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Recruiting Phase 1/2
View on ClinicalTrials.gov →
Summary
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2023-04-10; most recent amendment 2025-07-01.
Trial Details
NCT Number NCT05824169
Lead Sponsor GeneCradle Inc
Conditions Spinal Muscular Atrophy
Enrollment 18 participants
Start Date 2023-02-25
Primary Completion 2026-12 (estimated)
Study Completion 2026-12 (estimated)
Updated on ClinicalTrials.gov 2025-07-03