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	Recruiting
Enrollment Count	85

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
Dose 1	5 x 10^11 - 2 x 10^12 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	27
Locations	United States

Has US IND	True
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