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

NCT ID: NCT05399069

Last Updated: 2022-11-09

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-09-15
• Study Completion Date: 2024-05-31

Brief Summary

An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

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 365(±7) days 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

Bietti Crystalline Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

VGR-R01

Subretinal injection of VGR-R01

Group Type: EXPERIMENTAL

VGR-R01

Intervention Type: DRUG

Gene Replacement Therapy

Interventions

VGR-R01

Gene Replacement Therapy

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. ≥18 years and <80 years of age;

2. Confirmed diagnosis of Bietti Crystalline Dystrophy;

3. Molecular diagnosis of CYP4v2 mutations (homozygotes or compound heterozygotes);

4. BCVA ≤ 20/200 in the study eye;

5. -8 D <diopters< +8 D, 21 mm < axial lengths ≤ 28 mm in the study eye;

6. Normal liver function and renal function；

7. Agree to use reliable barrier contraception for 1 year after administration of VGR-R01;

8. Able to provide informed consent and comply with requirements of the study. -

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. Prior medications which may interfere with the interpretation of study endpoints within six months before screening, eg. anti-VEGF drugs;

6. Have or potentially require of systemic medications that may cause eye injure;

7. Live attenuated vaccines is expected to be required during the study;

8. Participation in a clinical study with an investigational drug or medical device within three months before enrollment;

9. History of allergy or sensitivity to investigational drug, medications planned for use in the study;

10. Use of anticoagulants, or after 10 days cessation of anti-platelet agents the platelet function does not recover;

11. Use of any corticosteroids, other immunosuppressive drug(s) or antipsychotic drugs (eg. antidepressant, etc.) within 3 months prior to enrollment;

12. Have contraindications for corticosteroids or immunosuppressant;

13. Have complicating systemic diseases that would preclude the planned follow-up;

14. Abnormal coagulation function or other clinically significant abnormal laboratory results;

15. Have malignancies or history of malignancies;

16. History of immunodeficiency (acquired or congenital);

17. Females in lactation period;

18. Have a history of alcohol or illicit drug addiction;

19. Unable or unwilling to comply with the schedule of visits. -

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Beijing Tongren Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Wenbin Wei

Role: PRINCIPAL_INVESTIGATOR

Vice President of Beijing Tongren Hospital

Locations

Beijing Tongren Hospital

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Wenbin Wei

Role: CONTACT

tr_weiwenbin@163.com

010-58269516

Xiuli Zhao

Role: CONTACT

xiulizhao@medmail.com.cn

010-58268486-8008

Facility Contacts

Wen-Bin Wei, MD

Role: primary

weiwenbintr@163.com

Other Identifiers

VGR-R01-001

Identifier Type: -

Identifier Source: org_study_id