NCT06699108

Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients With Bietti Crystalline Dystrophy

A Multi-Center, Randomized, Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of VGR-R01 in Subjects With Bietti Crystalline Dystrophy

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 45 (estimated)
• Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 69 Years
• Healthy volunteers: Not accepted

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.

Detailed description

VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to assess the efficacy of VGR-R01 in subjects with Bietti crystalline dystrophy (BCD) based on the change from baseline in best corrected visual acuity (BCVA) of the study eyes. 30 subjects will be enrolled in the intervention group and will receive monocular administration of VGR-R01, and 15 subjects in the control group will not receive any intervention. After completion of the trial, the untreated eyes will receive administration of VGR-R01 in subsequent clinical trial.

Conditions

• Bietti Crystalline Dystrophy
• Inherited Retinal Diseases

Interventions

Type	Name	Description
DRUG	VGR-R01	Subretinal injection of VGR-R01 (0.1 mL)

Timeline

• Start date: 2024-12-13
• Primary completion: 2026-12-01
• Completion: 2027-06-30
• First posted: 2024-11-21
• Last updated: 2025-12-24

Locations

1 site across 1 country: China