A Multicenter, Randomized，Controlled，Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of ZVS101e Administered as a Single Subretinal Injection in Subjects With Bietti's Crystalline Dystrophy (BCD)

Chigenovo Co., Ltd8 sites in 1 country62 target enrollmentDecember 27, 2024

ConditionsBietti Crystalline Corneoretinal Dystrophy Bietti Crystalline Dystrophy

InterventionsZVS101e

DrugsZVS101e

Overview

Phase: Phase 3
Intervention: ZVS101e
Conditions: Bietti Crystalline Corneoretinal Dystrophy
Sponsor: Chigenovo Co., Ltd
Enrollment: 62
Locations: 8
Primary Endpoint: Proportion of study eyes with a ≥15-letter improvement from baseline in BCVA
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a multi-center, randomized and controlled phase 3 clinical trial.

Detailed Description

Subjects who have signed the written informed consent form and who were eligible for enrollment after screening will be randomly assigned to the treatment group or untreated control group. Each Participant in the treatment group will receive a unilateral subretinal injection of ZVS101e in the study eye. Participants in the control group will receive no treatment during the first 52 weeks of enrollment.

Registry

clinicaltrials.gov

Start Date

December 27, 2024

End Date

June 30, 2030

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Chigenovo Co., Ltd

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•All of the following inclusion criteria should be met for enrollment into the trial:
•Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form, and be able to complete all trial procedures as required by the protocol;
•Clinical diagnosis of Bietti's crystalline dystrophy (BCD), age≥18 years ;
•Genetic testing confirmed biallelic CYP4V2 mutations without other ophthalmic genetic diseases;
•Best-corrected visual acuity of 5-60 ETDRS letters.

Exclusion Criteria

•The study eye has or has had macular lesions such as macular hole or macular neovascularization; glaucoma, diabetic retinopathy, or any other ocular disease that may preclude surgery or interfere with interpretation of the study endpoints
•The study eye had received the following intraocular surgical treatments: retinal reattachment, vitrectomy;
•The study eye had received any intraocular surgery, such as phacoemulsification 3 months prior to enrollment;
•Previously treatment of either eye with gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to viral vector gene therapy, RNA therapy;
•Pregnant or lactating women;

Arms & Interventions

Treatment group

Each Participant in this group will receive a unilateral subretinal injection of ZVS101e in the study eye

Intervention: ZVS101e

Outcomes

Primary Outcomes

Proportion of study eyes with a ≥15-letter improvement from baseline in BCVA

Time Frame: From baseline to Week 52

Best corrected visual acuity (BCVA) will be assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A 15-letter improvement over the baseline is clinical meaningful.

Secondary Outcomes

Change from Baseline in BCVA(From baseline to Week 52)
Proportion of study eyes with a ≥10-letter improvement from baseline in BCVA(From baseline to Week 52)
Change from baseline in multi-luminance mobility test (MLMT) score(From baseline to Week 52)
Change from baseline in 25-Item visual function questionnaire (VFQ-25) score(From baseline to Week 52)
Change from Baseline in microperimetry(From baseline to Week 52)
Change from baseline in OCT(From baseline to Week 52)
Incidence and severity of ocular and systemic adverse events(From baseline to Week 52)