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

NCT06743646 | Recruiting Phase 3

• 1 other identifier: ZYA-2024-001
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 8

Brief Summary

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

Conditions

Bietti Crystalline Corneoretinal Dystrophy; Bietti Crystalline Dystrophy

Keywords

gene replacement therapy; ZVS101e; AAV8-hCYP4V2; phase 3; Bietti crystalline corneoretinal dystrophy

Outcome Measures

Primary Outcomes (1)

• 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.

  From baseline to Week 52

Secondary Outcomes (7)

• 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

Study Arms (2)

Treatment group

EXPERIMENTAL

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

Drug: ZVS101e

Control group

NO INTERVENTION

Participants in the control group will receive no treatment

Interventions

ZVS101e DRUG

subretinal injection of ZVS101e

Also known as: AAV8-hCYP4V2, AAV2/8-hCYP4V2, Puliretgene parvec

Treatment group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Chigenovo Co., Ltd: lead

Study Sites (8)

Peking University Third Hospital

Beijing, Beijing Municipality, China

NOT YET RECRUITING

Zhongshan Ophthalmic Center, Sun Yat-sen University

Guangzhou, Guangdong, China

NOT YET RECRUITING

The First Affiliated Hospital of Harbin Medical University

Haerbin, Heilongjiang, China

NOT YET RECRUITING

Henan Provincial People's Hospital

Zhengzhou, Henan, China

NOT YET RECRUITING

Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China

NOT YET RECRUITING

West China Hospital of Sichuan University

Chengdu, Sichuan, China

NOT YET RECRUITING

Tianjin Medical University Eye Hospital

Tianjin, Tianjin Municipality, China

RECRUITING

Eye Hospital, Wenzhou Medical University

Wenzhou, Zhejiang, China

NOT YET RECRUITING

Related Publications (2)

• 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.

  PMID: 35925866 RESULT

• 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.

  PMID: 38653979 RESULT

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Officials

• Jinlu Zhang, MD

  Chigenovo Co., Ltd

  STUDY DIRECTOR

Central Study Contacts

Jinlu Zhang, MD

CONTACT

+86-15810570898; zhangjinlu@chinagene.cc

Tingting Wu, BS

CONTACT

+86-18500191916; wutingting@chinagene.cc

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2024
• First Posted: December 20, 2024
• Study Start: December 27, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2030
• Last Updated: January 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (8)