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Gene therapy, Open-Label, Dose-escalation study of SPK-9001 [adeno-associated viral vector with human factor IX gene] in subjects with hemophilia B

Clinical Trial Details

The approach being tested in this study uses a novel recombinant adeno-associated virus (AAV), which in nature causes no disease, to deliver the human factor IX (hFIX) gene to the liver cells where FIX is normally made. Recent data of a gene therapy study showed preliminary encouraging results with the approach of using an AAV vector carrying the factor IX gene. This study will seek to determine the safety and kinetics of a single IV infusion of SPK-9001 (a novel AAV vector carrying a high specific activity factor IX variant). 

Study contact by location

Upper East Side - Manhattan

Contact(s)

Ilene Goldberg
(212) 746-3403
igoldber@med.cornell.edu

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:

1510016677

Status

Open to Enrollment

Age Group

Adult , Pediatric

Disease