drugset / Trial / NCT07554924
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
NaSequentialOpen-labelTreatment
Summary
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
Timeline
- Start
- 2026-05-15
- Primary completion
- 2029-05-25
- Completion
- 2029-05-25
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | SKG0201 | Gene therapy (AAV / viral vector) | — | — |