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

Chigenovo

Status and phase: Enrolling; Phase 3

Conditions: Bietti Crystalline Dystrophy; Bietti Crystalline Corneoretinal Dystrophy

Treatments: Drug: ZVS101e

Study type: Interventional

Funder types: NETWORK

Identifiers

NCT06743646

ZYA-2024-001

Details and patient eligibility

About

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

Full 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.

Enrollment: 62 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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;

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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62 participants in 2 patient groups

Treatment group

Experimental group

Description:

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

Treatment:

Drug: ZVS101e

Control group

No Intervention group

Description:

Participants in the control group will receive no treatment