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

NCT ID: NCT05694598

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

PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-27

Study Completion Date

2028-09-13

Brief Summary

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A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

Detailed Description

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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 at least 52 weeks 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

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Bietti Crystalline Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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VGR-R01

Single-dose Subretinal Administration of VGR-R01

Group Type EXPERIMENTAL

VGR-R01

Intervention Type GENETIC

CYP4v2-coding gene delivered by AAV vector

Interventions

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VGR-R01

CYP4v2-coding gene delivered by AAV vector

Intervention Type GENETIC

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. 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

Principal Investigators

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Wenbin Wei

Role: PRINCIPAL_INVESTIGATOR

Beijing Tongren Hospital

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-R01-101

Identifier Type: -

Identifier Source: org_study_id