Contact SCGE




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
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.