Trials / Active Not Recruiting
Active Not RecruitingNCT05694598
Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy
A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
- Status
- Active Not Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- Sponsor
- Shanghai Vitalgen BioPharma Co., Ltd. · Industry
- Sex
- All
- Age
- 18 Years – 69 Years
- Healthy volunteers
- Not accepted
Summary
A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
Detailed description
VGR-R01 is a novel AAV vector carrying the human CYP4V2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo at least 52 weeks of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGR-R01 for a total of five years.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | VGR-R01 | CYP4v2-coding gene delivered by AAV vector |
Timeline
- Start date
- 2023-03-27
- Primary completion
- 2024-09-13
- Completion
- 2028-09-13
- First posted
- 2023-01-23
- Last updated
- 2025-12-24
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT05694598. Inclusion in this directory is not an endorsement.