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

NCT06699108 | Active Not Recruiting Phase 3

• 1 other identifier: VGR-R01-301
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Bietti Crystalline Dystrophy; Inherited Retinal Diseases

Outcome Measures

Primary Outcomes (1)

• Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS

  Change in the BCVA assessment from screening in the study eye of BCD subjects will be compared to controls

  Baseline up to Week 52

Secondary Outcomes (6)

• Change from baseline in BCVA

  Baseline up to Week 52

• Change from baseline in multi-luminance mobility test (MLMT) score of the study eye

  Baseline up to Week 52

• Changes from baseline in Microperimetry indexes

  Baseline up to Week 52

• Change from baseline in optical coherence tomography (OCT)

  Baseline up to Week 52

• Counts, frequencies and percentages of AE, SAE and other safety evaluation

  Baseline up to Week 52

Other Outcomes (2)

• Changes from baseline in visual field index

  Baseline up to Week 52

• Change from baseline in PRO measures

  Baseline up to Week 52

Study Arms (2)

interventional group

EXPERIMENTAL

Drug: VGR-R01

Control group

NO INTERVENTION

Interventions

VGR-R01 DRUG

Subretinal injection of VGR-R01 (0.1 mL)

interventional group

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able to provide informed consent and comply with requirements of the study;
• ≥18 years and \<70 years of age;
• Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
• Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study eye;

You may not qualify if:

• Have insufficient viable retinal photoreceptor cells based on investigator's decision;
• Have current ocular or periocular infections, or endophthalmitis;
• Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;
• Have intraocular surgery history except cataract surgery in the study eye;
• Have or potentially require of systemic medications that may cause eye injure;
• Have contraindications for corticosteroids or immunosuppressant;
• Unwilling or unable to have the planned follow-up;
• Abnormal coagulation function or other clinically significant abnormal laboratory results;
• Have malignancies or history of malignancies;

Sponsors & Collaborators

• Shanghai Vitalgen BioPharma Co., Ltd.: lead

Study Sites (1)

Shanghai Vitalgen Biopharma Co.,Ltd.

Shanghai, Shanghai Municipality, China

Location

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2024
• First Posted: November 21, 2024
• Study Start: December 13, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: December 24, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)