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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)
NCTID
NCT06345898
(View at clinicaltrials.gov)
Description
This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002.
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Indication
Retinoschisis, X-linked
Compound Name
JWK002
Sponsor
West China Hospital
Funder Type
Other
Status
Recruiting
Enrollment Count
18
Therapy Information
Target Gene/Variant
RS1
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
AAV8
Editor Type
Dose 1
Undisclosed low dose
Dose 2
Undisclosed medium dose
Dose 3
Undisclosed high dose
Dose 4
Dose 5
Study Record Dates
Current Stage
Early phase1
Submit Date
2024-03-28
Completion Date
2029-12-30
Last Update
2024-07-23
Participation Criteria
Eligible Age
5 Years - 18 Years
Standard Ages
Child, Adult
Eligible Sex
MALE
Locations
No.of Trial Sites
1
Locations
China
Regulatory Information
Has US IND
False
Recent Updates
Investigator-initiated trial conducted at West China Hospital
Resources/Links
Resources/Links
No External Links Available.