Study of a Gene Therapy Treatment for Hemophilia A

NCTID	NCT06297486 (View at clinicaltrials.gov)
Description	The purpose of this study is to evaluate the efficacy of SPK-8011 in preventing bleed episodes compared with FVIII prophylaxis in participants with hemophilia A without FVIII inhibitors on routine FVIII prophylaxis. (Show More)
Indication	Hemophilia A
Compound Name	Dirloctogene samoparvovec/SPK-8011 (AAV LK03 capsid + TTRmut-hFVIII-X07)
Sponsor	Spark Therapeutics, Inc.
Funder Type	Industry
Status	Withdrawn
Enrollment Count

Target Gene/Variant	F8
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	LK03
Editor Type	none
Dose 1	5E11 vg/kg
Dose 2	1E12 vg/kg
Dose 3	1.5E12 vg/kg
Dose 4	2E12 vg/kg
Dose 5

No.of Trial Sites	27
Locations	United States

Has US IND	True
Recent Updates	Product development discontinued, Study was withdrawn by Sponsor (no participants were enrolled)