NCT06191354

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

A Multicenter, Open, Dose-escalation Clinical Study Evaluating the Safety, Initial Efficacy, and Immunogenicity of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Kun Sun · Academic / Other
• Sex: All
• Age: 180 Days
• Healthy volunteers: Not accepted

Summary

This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).

Detailed description

This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).

Conditions

• Spinal Muscular Atrophy 1

Interventions

Type	Name	Description
GENETIC	SKG0201 Injection	SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based in vivo gene therapeutic product.

Timeline

• Start date: 2023-06-25
• Primary completion: 2025-12-01
• Completion: 2025-12-01
• First posted: 2024-01-05
• Last updated: 2025-11-21

Locations

3 sites across 1 country: China