Compassionate Administration of ZVS101e for Extended Treatment

NCT07307469 | Enrolling By Invitation

• 1 other identifier: ZYA-2025-004
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 5

Brief Summary

To provide treatment options for the control group of subjects who completed the 52-week follow-up of the Phase III clinical trial (Protocol number: ZYA-2024-001) of ZVS101e in subjects with Bietti crystalline corneoretinal dystrophy (BCD).

Conditions

Bietti Crystalline Corneoretinal Dystrophy

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events and serious adverse events

  This study specifies that the recording of adverse events begins on Day 0 (D0). Adverse events (AEs) include, but are not limited to, the following: 1. Worsening of pre-existing medical conditions/diseases (i.e., conditions present prior to enrollment in the clinical trial), including exacerbation of symptoms, signs, frequency of episodes, or abnormal laboratory test results; 2. Any newly occurring adverse medical conditions (including symptoms, signs, or newly diagnosed diseases); 3. Abnormal laboratory test values or results with clinical significance, or other safety assessments (e.g., vital signs measurements).

  4 weeks

Study Arms (1)

ZVS101e treatment

EXPERIMENTAL

The study eye of the subject receives a subretinal injection of ZVS101e.

Drug: ZVS101e

Interventions

ZVS101e DRUG

ZVS101e is a recombinant adeno-associated virus serotype 8 (rAAV8) vector expressing human CYP4V2 protein

ZVS101e treatment

Eligibility Criteria

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

You may qualify if:

• Subjects who were randomly assigned as the control group in a multicenter, randomized controlled, Phase III clinical trial (Protocol number: ZYA-2024-001) on the efficacy and safety of a single subretinal injection of ZVS101e in subjects with BCD and completed a 52-week safety follow-up were included.
• Voluntarily participate in clinical trials and sign informed consent forms, and be willing to complete all trial procedures as required by the protocol.
• Study the BCVA of the eye ≤60 ETDRS letters.

You may not qualify if:

• The study eye currently has or has had macular degeneration such as macular holes or neovascularization in the macular area; Other ocular diseases such as glaucoma and diabetic retinopathy that may hinder surgery or interfere with the interpretation of the study endpoint.
• The study eye had undergone retinal repositioning surgery, vitrectomy, or had undergone any intraocular surgery within 3 months prior to enrollment, such as phacoemulsification for cataract.
• Within one month prior to enrollment, the patient had a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or had received an antiviral vaccine.
• Any eye has previously received gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to other viral vector gene therapies, mRNA therapies, etc.
• Known to be allergic to the drugs planned to be used in the study.
• The following laboratory test abnormalities have clinical significance:
• Liver function: ALT or AST\>2 times the upper limit of the normal value; Abnormal coagulation function (prothrombin time ≥ the upper limit of normal value by 3 seconds, activated partial thromboplastin time ≥ the upper limit of normal value by 10 seconds).
• Pregnant or lactating women.

Sponsors & Collaborators

• Chigenovo Co., Ltd: lead

Study Sites (5)

Peking University Third Hospital

Beijing, Beijing Municipality, China

Location

Zhongshan Ophthalmic Center, Sun Yat-sen University

Guangzhou, Guangdong, China

Location

Henan Provincial People's Hospital

Zhengzhou, Henan, China

Location

West China Hospital, Sichuan University

Chengdu, Sichuan, China

Location

Tianjin Medical University Eye Hospital

Tianjin, Tianjin Municipality, China

Location

Related Publications (5)

• Yang L, Li W, Zhang J, Chen S, Qiao J, Qu B, Zhang F, Shen X, Leung HYE, Lam DSC. ZVS101e: AAV-mediated gene replacement therapy for Bietti crystalline corneoretinal dystrophy (BCD). Asia Pac J Ophthalmol (Phila). 2026 May-Jun;15(3):100320. doi: 10.1016/j.apjo.2026.100320. Epub 2026 Apr 21.

  PMID: 42025802 BACKGROUND

• Jia R, Chen S, Li W, Zhang J, Qu B, Qiao J, Meng X, Yu S, Liu X, Xu B, Chen T, Shen X, Sun W, Dou H, Mahajan VB, Zhang Q, Yang L. Unravelling CYP4V2: Clinical features, genetic insights, pathogenic mechanisms and therapeutic strategies in Bietti crystalline corneoretinal dystrophy. Prog Retin Eye Res. 2025 Jul;107:101377. doi: 10.1016/j.preteyeres.2025.101377. Epub 2025 Jun 5.

  PMID: 40482806 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• Yang L, Xing D, Zhang J, Liu B, Jiang X, Chen S, Li W, Qu B, Qiao J, Feng X, Jia H, Hu X, Yu S, Jia R, Wang L, Mahajan VB, Dou H, Lu F, Li X. Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial. Mol Ther. 2026 Mar 4;34(3):1743-1755. doi: 10.1016/j.ymthe.2026.01.005. Epub 2026 Jan 10.

  PMID: 41520174 BACKGROUND

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2025
• First Posted: December 29, 2025
• Study Start: July 1, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: June 17, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (5)