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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy
NCTID
NCT06699108
(View at clinicaltrials.gov)
Description
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects.
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Development Status
Active
Indication
Bietti Crystalline Dystrophy
Disease Ontology Term
DOID:0050664
Compound Name
VGR-R01
Compound Alias
rAAV2/8-hCYP4V2
Sponsor
Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type
Industry
Recruitment Status
Not yet recruiting
Enrollment Count
45
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
6.0E10 vg/eye
Dose 2
1.2E11 vg/eye (expansion dose)
Dose 3
2.0E11 vg/eye
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase3
Submit Date
2024-11-19
Completion Date
2027-06-30
Last Update
2024-11-21
Participation Criteria
Eligible Age
18 Years - 69 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Phase I/II preliminary results presented at ASGCT 2024
Resources/Links
Clinical Publications
(Abstract #149) Subretinal Gene Therapy in Patients with Bietti Crystalline Dystrophy: Preliminary Results of a Phase 1/2 Clinical Trial - ASGCT 2024
Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial
News and Press Releases
Vitalgen completed the enrollment in the Phase I/II clinical trial of the world's first BCD gene therapy
Preclinical Publications
AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice
Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy
Related NCTID
Phase 1: NCT05694598
Early Phase I: NCT05399069