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

NCT ID: NCT06699108

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-13

Study Completion Date

2027-06-30

Brief Summary

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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

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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

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Bietti Crystalline Dystrophy Inherited Retinal Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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interventional group

Group Type EXPERIMENTAL

VGR-R01

Intervention Type DRUG

Subretinal injection of VGR-R01 (0.1 mL)

Control group

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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VGR-R01

Subretinal injection of VGR-R01 (0.1 mL)

Intervention Type DRUG

Eligibility Criteria

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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;

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;
Minimum Eligible Age

18 Years

Maximum Eligible Age

69 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shanghai Vitalgen BioPharma Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Shanghai Vitalgen Biopharma Co.,Ltd.

Shanghai, Shanghai Municipality, China

Site Status

Countries

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China

Other Identifiers

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VGR-R01-301

Identifier Type: -

Identifier Source: org_study_id