Bietti Crystalline Corneoretinal DystrophyNCT07307469
Compassionate Administration of ZVS101e for Extended Treatment
A study testing ZVS101e in people with Bietti Crystalline Corneoretinal Dystrophy, run by Chigenovo Co., Ltd at 5 sites in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- 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
- 32 participants
- Where
- 5 sitesChina
- Study dates
- Jul 2026to Jul 2027
- 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
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
China5 sites
Peking University Third Hospital
Beijing, Beijing Municipality
West China Hospital, Sichuan University
Chengdu, Sichuan
Zhongshan Ophthalmic Center, Sun Yat-sen University
Guangzhou, Guangdong
Tianjin Medical University Eye Hospital
Tianjin, Tianjin Municipality
Henan Provincial People's Hospital
Zhengzhou, Henan
About this study
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).
Read the detailed description
The trial period lasts for a total of 8 weeks, including a 4-week screening period, a administration period, and visits on the 1st day, 7th day, and 4th week after administration. The patients will be followed up at the research center on the 1st day, 7th day and 4th week after administration to evaluate the safety.
- Being tested
- ZVS101e
- 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 is a recombinant adeno-associated virus serotype 8 (rAAV8) vector expressing human CYP4V2 protein
| Study group | Role | What they receive |
|---|---|---|
| ZVS101e treatment | Experimental | The study eye of the subject receives a subretinal injection of ZVS101e. Receives: Drug: ZVS101e |
What is being measured
The primary outcome is the main question the study is designed to answer.
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:
- 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;
- Any newly occurring adverse medical conditions (including symptoms, signs, or newly diagnosed diseases);
- Abnormal laboratory test values or results with clinical significance, or other safety assessments (e.g., vital signs measurements).
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2025
- Study startJul 2026 (estimated)
- Primary completionJan 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Bietti Crystalline Corneoretinal 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
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chigenovo Co., Ltd
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07307469
- Sponsor's protocol ID
- ZYA-2025-004
- Status last verified
- Jun 2026
- First submitted
- 14 Dec 2025
- Last updated
- 17 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 42025802Jia 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 40482806Jia 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 35925866Wang 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 38653979Yang 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 41520174
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Compassionate Administration of ZVS101e for Extended Treatment
- ClinicalTrials.gov ID
- NCT07307469
- Acronym / sponsor ID
- ZYA-2025-004
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Chigenovo Co., LtdResearch network
- Study start
- Jul 2026 (estimated)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 29 Dec 2025 (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.
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).
Detailed description
The trial period lasts for a total of 8 weeks, including a 4-week screening period, a administration period, and visits on the 1st day, 7th day, and 4th week after administration. The patients will be followed up at the research center on the 1st day, 7th day and 4th week after administration to evaluate the safety.
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 | Experimental | Drug: ZVS101eThe study eye of the subject receives a subretinal injection of ZVS101e. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
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 is a recombinant adeno-associated virus serotype 8 (rAAV8) vector expressing human CYP4V2 protein | Drug | ZVS101e treatment |
Primary outcome
Incidence and severity of adverse events and serious adverse events
Time frame: 4 weeks
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).
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 17 Jun 2026.
- Background: PubMed 42025802Yang 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.
- 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 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.
- 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 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.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
5 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 Crystalline Corneoretinal Dystrophy, run by Chigenovo Co., Ltd at 5 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 3 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: enrolling by invitation, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events and serious adverse events (time frame: 4 weeks). 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 NCT07307469, which also lists 5 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 17 Jun 2026.