Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCTID	NCT03734588 (View at clinicaltrials.gov)
Description	SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII. (Show More)
Development Status	Inactive
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	SPK-8016
Sponsor	Spark Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	4
Results Posted	View Results

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	AAV
Editor Type	none
Dose 1	5E11 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	11
Locations	United States

Has US IND	True
FDA Designations
Recent Updates	Roche (maker of Hemlibra) acquired Spark in 2019, announced they would discontinue development of this program in Q2 2023 update