NCT07653971

Brief Summary

The goal of this clinical trial is to evaluate the safety and early effectiveness of ZVS101e in patients with Bietti crystalline dystrophy who meet the eligibility criteria for treatment under the translational application program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone. Participants will: undergo screening and baseline assessments to confirm eligibility; receive a single subretinal injection of ZVS101e in the study eye; complete follow-up visits over 4 weeks after treatment for safety monitoring and assessment of early effectiveness.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
300

participants targeted

Target at P75+ for not_applicable

Timeline
114mo left

Started Jul 2026

Longer than P75 for not_applicable

Geographic Reach
1 country

2 active sites

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 12, 2026

Completed
5 days until next milestone

First Posted

Study publicly available on registry

June 17, 2026

Completed
1 month until next milestone

Study Start

First participant enrolled

July 30, 2026

Expected
4.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 30, 2030

4.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2035

Last Updated

June 17, 2026

Status Verified

June 1, 2026

Enrollment Period

4.4 years

First QC Date

June 12, 2026

Last Update Submit

June 12, 2026

Conditions

Bietti's Crystalline DystrophyBietti Crystalline Corneoretinal Dystrophy

Keywords

Bietti's Crystalline DystrophyZVS101eAAV8-hCYP4V2gene replacement treatmentBietti Crystalline Corneoretinal Dystrophy

Outcome Measures

Primary Outcomes (2)

  • Change from baseline in Best corrected visual acuity (BCVA)

    From enrollment to the end of treatment at 52 weeks

  • The incidence and severity of adverse events and serious adverse events

    From enrollment to the end of treatment at 52 weeks

Study Arms (1)

ZVS101e Treatment Arm

EXPERIMENTAL
Drug: ZVS101e

Interventions

ZVS101eDRUG

ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.

ZVS101e Treatment Arm

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Voluntarily participate in the research and sign the informed consent form, and be willing to complete the entire process according to the protocol requirements;
  • Clinically diagnosed with Bietti's Crystalline Dystrophy (BCD), age ≥ 18 years;
  • Confirmed by genetic testing to carry homozygous or compound heterozygous mutations in CYP4V2, and not complicated with other ophthalmic genetic diseases;
  • Target eye BCVA ≤ 60 ETDRS letters.

You may not qualify if:

  • The target eye currently has or has a history of macular lesions; suffers from ocular diseases that may hinder surgery or interfere with the interpretation of study endpoints;
  • The target eye has previously undergone retinal reattachment surgery, vitrectomy, or any intraocular surgery within 3 months before enrollment;
  • Within 1 month before enrollment, suffered from viral infectious diseases that may affect the evaluation of the efficacy and safety of the investigational drug, or received antiviral vaccines;
  • The target eye has previously received gene therapy or stem cell therapy for BCD or other ocular diseases;
  • Known allergy to the drugs planned for use in the study;
  • The laboratory abnormalities are considered clinically significant;
  • Pregnant or lactating females.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Boao Super Hospital

Qionghai, Hainan, China

Location

Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University

Qionghai, Hainan, China

Location

Related Publications (2)

  • 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: 38653979RESULT

  • 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: 35925866RESULT

MeSH Terms

Conditions

Bietti Crystalline Dystrophy

Central Study Contacts

Jinlu Zhang, MD

CONTACT

+86-15810570898zhangjinlu@chinagene.cc

Tingting Wu, BS

CONTACT

+86-18500191916wutingting@chinagene.cc

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

June 12, 2026

First Posted

June 17, 2026

Study Start (Estimated)

July 30, 2026

Primary Completion (Estimated)

December 30, 2030

Study Completion (Estimated)

December 1, 2035

Last Updated

June 17, 2026

Record last verified: 2026-06

Data Sharing

IPD Sharing
Will not share

Locations

CN(2)