Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy

Phase 3Active Not RecruitingNCT06743646

Chigenovo Co., Ltd62 enrolled

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Bietti Crystalline Corneoretinal Dystrophy Bietti Crystalline Dystrophy

Interventions

ZVS101eDRUG

subretinal injection of ZVS101e

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. All of the following inclusion criteria should be met for enrollment into the trial: 2. 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; 3. Clinical diagnosis of Bietti's crystalline dystrophy (BCD), age≥18 years ; 4. Genetic testing confirmed biallelic CYP4V2 mutations without other ophthalmic genetic diseases; 5. Best-corrected visual acuity of 5-60 ETDRS letters. Exclusion Criteria: 1. 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 2. The study eye had received the following intraocular surgical treatments: retinal reattachment, vitrectomy; 3. The study eye had received any intraocular surgery, such as phacoemulsification 3 months prior to enrollment; 4. 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; 5. Pregnant or lactating women;

Locations (8)

Peking University Third Hospital

Beijing, Beijing Municipality, China

Zhongshan Ophthalmic Center, Sun Yat-sen University

Guangzhou, Guangdong, China

The First Affiliated Hospital of Harbin Medical University

Haerbin, Heilongjiang, China

Henan Provincial People's Hospital

Outcomes

Primary Outcomes

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

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.