A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)

NCT07586306 | Not Yet Recruiting Phase 1

• 1 other identifier: VGR-R01-102
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

Brief Summary

This is a multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.

Conditions

Bietti Crystalline Dystrophy

Keywords

VGR-R01; CYP4V2; gene therapy

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events and serious adverse events

  Ocular/non-ocular adverse events are collected. The ophthalmic examination will include Best Corrected Visual Acuity (BCVA), Intraocular Pressure (IOP), slit lamp examination, angiography and Optical Coherence Tomography (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.

  Up to Year 5

• Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings

  Vital signs (temperature, respiratory rate, pulse rate, systolic and diastolic blood pressure) will be obtained with participant in the seated position, after having sat calmly for at least 10 minutes. Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, etc. Ophthalmic Examination will include BCVA, IOP, slit lamp examination, angiography and OCT, etc. Clinical significance of the above signs will be determined at the investigator's discretion.

  Up to Year 5

Secondary Outcomes (7)

• Change from baseline in BCVA

  Year 5

• Change from baseline in multi-luminance mobility test (MLMT) score

  Year 5

• Change from baseline in optical coherence tomography (OCT)

  Year 5

• Number of subjects with the presence of immunogenicity

  Year 5

• Number of subjects with the presence of vector shedding

  Year 5

Other Outcomes (4)

• Visual field index (VFI)

  Year 5

• Central threshold test

  Year 5

• Change from baseline in NEI-VFQ-25 score

  Year 5

Study Arms (1)

VGR-R01 group

EXPERIMENTAL

Single-dose Subretinal Administration of VGR-R01

Biological: VGR-R01

Interventions

VGR-R01 BIOLOGICAL

CYP4v2-coding gene delivered by AAV vector

VGR-R01 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:

• Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
• Able to provide informed consent and comply with requirements of the study;
• Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated 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;
• 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 (2)

Beijing Tongren Hospital

Beijing, Beijing Municipality, China

Location

Shanghai General Hospital (Shanghai First People's Hospital)

Shanghai, Shanghai Municipality, China

Location

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Officials

• Wenbin Wei

  Beijing Tongren Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yan Jiang

CONTACT

086-15802234907; yan.jiang@vitalgen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2026
• First Posted: May 14, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): July 31, 2027
• Study Completion (Estimated): July 31, 2031
• Last Updated: May 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)