NCT05399069

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

An Open-Label Safety Study of Single-dose VGR-R01 in Patients With Bietti Crystalline Dystrophy

• Status: Unknown
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 3 (estimated)
• Sponsor: Beijing Tongren Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot 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 365(±7) days 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

• Bietti Crystalline Dystrophy

Interventions

Type	Name	Description
DRUG	VGR-R01	Gene Replacement Therapy

Timeline

• Start date: 2022-09-15
• Primary completion: 2024-05-01
• Completion: 2024-05-01
• First posted: 2022-06-01
• Last updated: 2022-11-09

Locations

1 site across 1 country: China