Bietti's Crystalline DystrophyZVS101e-BCDNCT07653971
ZVS101e in Patients With Bietti's Crystalline Dystrophy
A study testing ZVS101e in people with Bietti's Crystalline Dystrophy and Bietti Crystalline Corneoretinal Dystrophy, run by Chigenovo Co., Ltd at 2 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bietti's Crystalline Dystrophy, Bietti Crystalline Corneoretinal Dystrophy
- What is being tested?
- ZVS101e
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 300 participants
- Where
- 2 sitesChina
- Study dates
- 30 Sep 2026to Dec 2035
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Subjects will be excluded if they meet any 1 of the following exclusion criteria:
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites
Boao Super Hospital
Qionghai, Hainan
Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University
Qionghai, Hainan
About this study
Official title: Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy
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.
- Being tested
- ZVS101e
- Keywords
- Bietti's Crystalline Dystrophy, ZVS101e, AAV8-hCYP4V2, gene replacement treatment, Bietti Crystalline Corneoretinal Dystrophy
- Medical subject headings
- Bietti Crystalline Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZVS101eDrug
ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.
| Study group | Role | What they receive |
|---|---|---|
| ZVS101e Treatment Arm | Experimental | Not described Receives: Drug: ZVS101e |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Best corrected visual acuity (BCVA)
Measured: From enrollment to the end of treatment at 52 weeks
The incidence and severity of adverse events and serious adverse events
Measured: From enrollment to the end of treatment at 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2026
- Study start30 Sep 2026 (estimated)
- Primary completion30 Dec 2030 (estimated)
- Study completionDec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Bietti's Crystalline DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chigenovo Co., Ltd
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07653971
- Sponsor's protocol ID
- ZYA-2025-003
- Acronym
- ZVS101e-BCD
- Status last verified
- Aug 2026
- First submitted
- 12 Jun 2026
- Last updated
- 24 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 40482806Yang 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.
PubMed 41520174Wang 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.
PubMed 38653979Jia 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.
PubMed 35925866
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy
- ClinicalTrials.gov ID
- NCT07653971
- Acronym / sponsor ID
- ZVS101e-BCD / ZYA-2025-003
- Keywords
- Bietti's Crystalline Dystrophy, ZVS101e, AAV8-hCYP4V2, gene replacement treatment, Bietti Crystalline Corneoretinal Dystrophy
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Chigenovo Co., LtdResearch network
- Study start
- 30 Sep 2026 (estimated)
- Primary completion
- 30 Dec 2030 (estimated)
- Study completion
- Dec 2035 (estimated)
- First posted
- 17 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ZVS101e Treatment Arm | Experimental | Drug: ZVS101e |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (8 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZVS101eZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein. | Drug | ZVS101e Treatment Arm |
Primary outcome
Change from baseline in Best corrected visual acuity (BCVA)
Time frame: From enrollment to the end of treatment at 52 weeks
The incidence and severity of adverse events and serious adverse events
Time frame: From enrollment to the end of treatment at 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2026.
- Background: PubMed 40482806Jia 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.
- Background: PubMed 41520174Yang 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.
- Background: PubMed 38653979Wang 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.
- Background: PubMed 35925866Jia 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing ZVS101e in people with Bietti's Crystalline Dystrophy and Bietti Crystalline Corneoretinal Dystrophy, run by Chigenovo Co., Ltd at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jinlu Zhang, MD (+86-15810570898, zhangjinlu@chinagene.cc), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in Best corrected visual acuity (BCVA) (time frame: From enrollment to the end of treatment at 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07653971, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2026.