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)
Development Status	Active
Indication	Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term	DOID:11723
Compound Name	JWK007
Sponsor	West China Hospital
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	6
Results Posted	Not Available

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	none
Dose 1	1.0E14 vg/kg
Dose 2	2.0E14 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

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

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