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

NCT ID: NCT06743646

Last Updated: 2025-01-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-27

Study Completion Date

2030-06-30

Brief Summary

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

Detailed Description

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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.

Conditions

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Bietti Crystalline Corneoretinal Dystrophy Bietti Crystalline Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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Treatment group

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

Group Type EXPERIMENTAL

ZVS101e

Intervention Type DRUG

subretinal injection of ZVS101e

Control group

Participants in the control group will receive no treatment

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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ZVS101e

subretinal injection of ZVS101e

Intervention Type DRUG

Other Intervention Names

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AAV8-hCYP4V2 AAV2/8-hCYP4V2 Puliretgene parvec

Eligibility Criteria

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Inclusion Criteria

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;
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Chigenovo Co., Ltd

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jinlu Zhang, MD

Role: STUDY_DIRECTOR

Chigenovo Co., Ltd

Locations

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Peking University Third Hospital

Beijing, Beijing Municipality, China

Site Status NOT_YET_RECRUITING

Zhongshan Ophthalmic Center, Sun Yat-sen University

Guangzhou, Guangdong, China

Site Status NOT_YET_RECRUITING

The First Affiliated Hospital of Harbin Medical University

Haerbin, Heilongjiang, China

Site Status NOT_YET_RECRUITING

Henan Provincial People's Hospital

Zhengzhou, Henan, China

Site Status NOT_YET_RECRUITING

Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China

Site Status NOT_YET_RECRUITING

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Site Status NOT_YET_RECRUITING

Tianjin Medical University Eye Hospital

Tianjin, Tianjin Municipality, China

Site Status RECRUITING

Eye Hospital, Wenzhou Medical University

Wenzhou, Zhejiang, China

Site Status NOT_YET_RECRUITING

Countries

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China

Central Contacts

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Jinlu Zhang, MD

Role: CONTACT

+86-15810570898

Tingting Wu, BS

Role: CONTACT

+86-18500191916

Facility Contacts

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Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

Tingting Wu, BS

Role: primary

+86-18500191916

References

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Jia R, Meng X, Chen S, Zhang F, Du J, Liu X, Yang L. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice. Hum Mol Genet. 2023 Jan 1;32(1):122-138. doi: 10.1093/hmg/ddac181.

Reference Type RESULT
PMID: 35925866 (View on PubMed)

Wang J, Zhang J, Yu S, Li H, Chen S, Luo J, Wang H, Guan Y, Zhang H, Yin S, Wang H, Li H, Liu J, Zhu J, Yang Q, Sha Y, Zhang C, Yang Y, Yang X, Zhang X, Zhao X, Wang L, Yang L, Wei W. Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. Signal Transduct Target Ther. 2024 Apr 24;9(1):95. doi: 10.1038/s41392-024-01806-3.

Reference Type RESULT
PMID: 38653979 (View on PubMed)

Other Identifiers

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ZYA-2024-001

Identifier Type: -

Identifier Source: org_study_id

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