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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Efficacy of ZVS101e in Patients With Bietti 's Crystalline Dystrophy
NCTID
NCT05832684
(View at clinicaltrials.gov)
Description
The purpose of this study was to evaluate the safety and efficacy of ZVS101e administered by subretinal injection in subjects with Bietti's crystalline dystrophy (BCD) and to select the optimal effective dose.
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Development Status
Active
Indication
Bietti Crystalline Dystrophy
Disease Ontology Term
DOID:0050664
Compound Name
ZVS101e
Compound Description
rAAV2/8-hCYP4V2
Sponsor
Chigenovo Co., Ltd
Funder Type
Network
Recruitment Status
Active not recruiting
Enrollment Count
24
Results Posted
Not Available
Therapy Information
Target Gene/Variant
CYP4V2
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2/8
Editor Type
none
Dose 1
7.5E10 vg
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2023-04-02
Completion Date
2028-12
Last Update
2024-08-28
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
China
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Clinical Publications
Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial
Preclinical Publications
AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice
Related NCTID
Early Phase 1: NCT05714904
Early Phase 1: NCT04722107