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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Bietti Crystalline Dystrophy; Inherited Retinal Diseases
• Intervention / Treatment: Drug: VGR-R01

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.

• Study Type: Interventional
• Enrollment (Estimated): 45
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Shanghai Vitalgen Biopharma Co.,Ltd.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

1. Able to provide informed consent and comply with requirements of the study;
2. ≥18 years and <70 years of age;
3. Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
4. Hand Motion ≤ BCVA ≤ 60 ETDRS letters in the study eye;

Key Exclusion Criteria:

1. Have insufficient viable retinal photoreceptor cells based on investigator's decision;
2. Have current ocular or periocular infections, or endophthalmitis;
3. 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;
4. Have intraocular surgery history except cataract surgery in the study eye;
5. Have or potentially require of systemic medications that may cause eye injure;
6. Have contraindications for corticosteroids or immunosuppressant;
7. Unwilling or unable to have the planned follow-up;
8. Abnormal coagulation function or other clinically significant abnormal laboratory results;
9. Have malignancies or history of malignancies;
10. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Control group
Experimental: interventional group	Drug: VGR-R01; Subretinal injection of VGR-R01 (0.1 mL)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in BCVA	BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. Testing Protocol will be followed (confidential). Change in the BCVA assessment from baseline in treated eyes of intervention group will be compared to controls.	Baseline up to Week 52
Change from baseline in multi-luminance mobility test (MLMT) score of the study eye	Subjects will navigate a standardized mobility maze under set conditions as specified times during the study. The mobility testing will follow a standardized administration and data acquisition protocol and may only be administered by site staff certified in the methodology.	Baseline up to Week 52
Changes from baseline in Microperimetry indexes	Number of subjects with change in fixation stability or light sensitivity in treated eyes of intervention group will be compared to controls	Baseline up to Week 52
Change from baseline in optical coherence tomography (OCT)	Change from baseline in central retinal thickness (CRT) as imaged by OCT	Baseline up to Week 52
Counts, frequencies and percentages of AE, SAE and other safety evaluation	Ocular/non-ocular adverse events are collected. The ophthalmic examination will include BCVA, IOP, slitlamp examination, angiography and SD-OCT, etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision	Baseline up to Week 52
Number of subjects with the presence of immunogenicity	Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product	Baseline up to Week 52

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes from baseline in visual field index	Assessed by Humphery static visual field testing.	Baseline up to Week 52
Change from baseline in PRO measures	As assessed by the NEI-VFQ-25 questionnaire	Baseline up to Week 52

Collaborators and Investigators

• Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 13, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: November 19, 2024
• First Submitted That Met QC Criteria: November 19, 2024
• First Posted (Actual): November 21, 2024

Study Record Updates

• Last Update Posted (Actual): December 24, 2025
• Last Update Submitted That Met QC Criteria: December 17, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Corneal Diseases; Eye Diseases, Hereditary; Bietti Crystalline Dystrophy
• Other Study ID Numbers: VGR-R01-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: IPD will be shared with other researchers when VGR-R01 is fully approved

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No