A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

NCTID	NCT06114056 (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 JWK007 injection in pediatric patients with Duchenne Muscular Dystrophy (DMD). (Show More)
Indication	Duchenne Muscular Dystrophy
Compound Name	JWK007
Sponsor	West China Hospital
Funder Type	Other
Status	Recruiting
Enrollment Count	6

Target Gene/Variant	Micro-dystrophin
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh74
Editor Type
Dose 1	1.0 x 10^14 vg/kg
Dose 2	2.0 x 10^14 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
Recent Updates	Investigator-initiated trial conducted at West China Hospital

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