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

NCT05694598 | Active Not Recruiting Phase 1

• 1 other identifier: VGR-R01-101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Bietti Crystalline Dystrophy

Outcome Measures

Primary Outcomes (5)

• Incidence of adverse events

  An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

  Baseline up to Week 52

• Incidence of serious adverse events

  A serious adverse event (SAE) is any untoward medical occurrence at any dose that resulted in death; life threatening; require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect.

  Baseline up to Week 52

• Number of Participants with Clinically Significant Change from Baseline in Vital Signs

  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 5 minutes. Clinical significance of vital signs will be determined at the investigator's discretion.

  Baseline up to Week 52

• Number of Participants with Clinically Laboratory Abnormalities

  Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, 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 Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings

  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

Secondary Outcomes (5)

• Best-Corrected Visual Acuity (BCVA)

  Week 52

• Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes

  Week 52

• Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes

  Week 52

• Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes

  Week 52

• Changes from baseline in Mobility testing scores

  Week 52

Other Outcomes (1)

• Patient reported outcome: Changes of NEI-VFQ-25

  Week 52

Study Arms (1)

VGR-R01

EXPERIMENTAL

Single-dose Subretinal Administration of VGR-R01

Genetic: VGR-R01

Interventions

VGR-R01 GENETIC

CYP4v2-coding gene delivered by AAV vector

VGR-R01

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);
• 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 Officials

• Wenbin Wei

  Beijing Tongren Hospital

  PRINCIPAL INVESTIGATOR

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: January 11, 2023
• First Posted: January 23, 2023
• Study Start: March 27, 2023
• Primary Completion: September 13, 2024
• Study Completion (Estimated): September 13, 2028
• Last Updated: December 24, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: IPD will be shared with other researchers when VGR-R01 is fully approved.
• Access Criteria: IPD will be shared with other researchers when VGR-R01 is fully approved.

Locations

CN (1)