Clinical Trial

Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III

Recruiting Phase 3
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Summary
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives are to assess the efficacy, safety, and pharmacokinetic (PK) profile of GC101 in treating Type 2 SMA.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-05-06.
Trial Details
NCT Number NCT06971094
Lead Sponsor GeneCradle Inc
Conditions SMA - Spinal Muscular Atrophy
Enrollment 50 participants
Start Date 2025-05-27
Primary Completion 2026-12-31 (estimated)
Study Completion 2026-12-31 (estimated)
Updated on ClinicalTrials.gov 2025-06-05