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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ZVS101e Treatment Arm | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ZVS101e | Drug | ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
Subjects will be excluded if they meet any 1 of the following exclusion criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jinlu Zhang, MD | Contact | +86-15810570898 | zhangjinlu@chinagene.cc | |
| Tingting Wu, BS | Contact | +86-18500191916 | wutingting@chinagene.cc |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Boao Super Hospital | Qionghai | Hainan | China | |||
| Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38653979 | Result | 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. | |
| 35925866 |
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| ID | Term |
|---|---|
| C535440 | Bietti Crystalline Dystrophy |
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| Qionghai |
| Hainan |
| China |
| Result |
| 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. |