drugset / Trial / NCT07188012
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Phase 0
Recruiting
9 enrolled
Shanghai Siponuoyin Biotechnology Co Ltd
Shanghai Children's Medical Center · collab
NaSingle-groupOpen-labelTreatment
Summary
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients. In addition, this study will preliminarily investigate the changes in dystrophin nucleic acid concentration, dystrophin protein expression and engraftment, anti-dystrophin antibodies and cytokine profiles, as well as fat tissue mas and lean tissue mass following SPOT-03 administrations.
Timeline
- Start
- 2025-08-06
- Primary completion
- 2026-12
- Completion
- 2026-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | SPOT-03 | Unknown | 4e+11 unknown | Intravenous |