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

A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

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

Study Overview

• Status: Active, not recruiting
• Conditions: Bietti Crystalline Dystrophy
• Intervention / Treatment: Genetic: VGR-R01

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 1

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: 18 years to 69 years (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. 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: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VGR-R01; Single-dose Subretinal Administration of VGR-R01	Genetic: VGR-R01; CYP4v2-coding gene delivered by AAV vector

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Best-Corrected Visual Acuity (BCVA)	BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured.	Week 52
Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes	The VFI can range from 100% (normal visual field) to 0% (perimetrically blind field).	Week 52
Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes	Normal values are typically within 0dB and -2dB.	Week 52
Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes	A typical "normal" dB reading is around 30. The numeric dB graph should be studied next. The dBs tested by the Humphrey analyzer range between 0 and 50 dB (0 is the brightest and 50 is the dimmest).	Week 52
Changes from baseline in Mobility testing scores	The mobility score range is between -1 (the worst functional vision) and 6 (the best).	Week 52

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient reported outcome: Changes of NEI-VFQ-25	NEI-VFQ-25 questionnaire measures dimensions of self-reported vision-targeted health status that are most important to persons with eye disease. Total score ranges from 0-100, where a score of 0 represents the worst outcome and 100 represents the best outcome.	Week 52

Collaborators and Investigators

• Sponsor: Shanghai Vitalgen BioPharma Co., Ltd.
• Investigators: Principal Investigator: Wenbin Wei, Beijing Tongren Hospital

Study record dates

Study Major Dates

• Study Start (Actual): March 27, 2023
• Primary Completion (Actual): September 13, 2024
• Study Completion (Estimated): September 13, 2028

Study Registration Dates

• First Submitted: January 11, 2023
• First Submitted That Met QC Criteria: January 20, 2023
• First Posted (Actual): January 23, 2023

Study Record Updates

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

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Corneal Diseases; Bietti Crystalline Dystrophy
• Other Study ID Numbers: VGR-R01-101

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.
• IPD Sharing Time Frame: IPD will be shared with other researchers when VGR-R01 is fully approved.
• IPD Sharing Access Criteria: IPD will be shared with other researchers when VGR-R01 is fully approved.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No