NCT06971094

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

A Multicenter, Randomized, Open-Label, Standard-of-Care-Controlled, Phase III Clinical Trial to Evaluate the Safety and Efficacy of Intrathecal (IT) Injection of GC101 Adeno-Associated Virus Injection in the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: GeneCradle Inc · Industry
• Sex: All
• Age: 2 Years – 12 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The trial is divided into two groups: one group will receive a single intrathecal injection of GC101 at a dose of 1.2E+14 vg per person and discontinue their previous standard-of-care treatment with nusinersen; the other group will continue their previous standard-of-care treatment with nusinersen. Participants will be randomly assigned to the trial group or the control group in a 1:1 ratio.

Conditions

• SMA - Spinal Muscular Atrophy

Interventions

Type	Name	Description
GENETIC	GC101 adeno-associated virus injection	Self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene

Timeline

• Start date: 2025-05-27
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2025-05-14
• Last updated: 2025-06-05

Locations

7 sites across 1 country: China