drugset / Trial / NCT06699108
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy
RandomizedParallel-groupSingle-blindTreatment
Summary
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects.
Timeline
- Start
- 2024-12-13
- Primary completion
- 2026-12-01
- Completion
- 2027-06-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VGR-R01 | Gene therapy (AAV / viral vector) | 0.1 ml | Other |