Hemophilia B Clinical Trial
Official title:
Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
Hemophilia B, or Christmas disease, is a genetic bleeding disorder resulting in the lack of
ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer
repeated bleeding events, which can cause chronic joint disease and sometimes leads to death
due to the inability for blood to clot efficiently. This chronic joint disease can have
significant physical, psychosocial, and quality-of-life effects, including financial burden.
The current treatment is intravenous infusion of FIX protein products, either
prophylactically or in response to bleeding.
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).
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