Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
NCTID
NCT06519552
(View at clinicaltrials.gov)
Description
This study is a single-center, single-arm, non-randomized, open-label, non controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK008 injection in patients with MPS I.
(Show More)
Development Status
Active
Indication
Mucopolysaccharidosis Type I (Hurler Syndrome)
Disease Ontology Term
DOID:12802
Compound Name
JWK-008
Sponsor
West China Hospital
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
6
Results Posted
Not Available
Therapy Information
Target Gene/Variant
IDUA
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravenous
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV5
Editor Type
Dose 1
5.0E12 vg/kg
Dose 2
2.0E13 vg/kg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2024-06-23
Completion Date
2029-06-22
Last Update
2024-07-25
Participation Criteria
Eligible Age
>=18 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
Resources/Links
Resources/Links
No External Links Available.