Compassionate Administration of ZVS101e Injection 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 crystalline retinal degeneration (BCD).

Conditions

Patients With Bietti Crystalline Corneoretinal Dystrophy (Carrying Biallelic CYP4V2 Mutations)

Outcome Measures

Primary Outcomes (1)

• Safety evaluation

  Incidence of adverse events

  4 weeks

Study Arms (1)

Safety evaluation

EXPERIMENTAL

Adverse events that occurred during the study period

Drug: ZVS101e

Interventions

ZVS101e DRUG

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

Safety evaluation

Eligibility Criteria

• Age: 18 Years - 75 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 crystalline retinal degeneration (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

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• 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: March 1, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: December 29, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (5)