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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

January 30, 2019

Primary Completion Date

October 14, 2020

Study Completion Date

January 19, 2023

Conditions
Adeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, InheritedCoagulation Protein DisordersFactor VIII (FVIII)Factor VIII (FVIII) DeficiencyFactor VIII (FVIII) GeneFactor VIII (FVIII) ProteinGenetic Diseases, InbornGenetic Diseases, X-LinkedGene TherapyGene TransferHematologic DiseasesHemorrhagic DisordersRecombinantVectorInhibitors
Interventions
GENETIC

SPK-8016

adeno-associated viral vector

Trial Locations (11)

10065

Weill Cornell Medicine, New York

15213

Hemophilia Center of Western Pennsylvania, Pittsburgh

17033

Penn State Health, Hershey

19104

Children's Hospital of Philadelphia, Philadelphia

19107

Jefferson University Hospitals, Philadelphia

23219

Virginia Commonwealth University School of Medicine, Richmond

39110

Mississippi Center for Advanced Medicine, Madison

48109

University of Michigan, Ann Arbor

61615

Illinois Bleeding and Clotting Disorders Institute, Peoria

90007

Orthopaedic Institute for Children, Los Angeles

97239

Oregon Health & Science University, Portland

All Listed Sponsors
lead

Spark Therapeutics, Inc.

INDUSTRY

NCT03734588 - Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors | Biotech Hunter | Biotech Hunter