NCT02484092

A Gene Therapy Study for Hemophilia B

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Pfizer · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.

Detailed description

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).

Conditions

• Hemophilia B

Interventions

Type	Name	Description
BIOLOGICAL	SPK-9001	A novel, bioengineered adeno-associated viral vector carrying human factor IX variant

Timeline

• Start date: 2015-11-18
• Primary completion: 2019-04-08
• Completion: 2019-04-08
• First posted: 2015-06-29
• Last updated: 2020-06-16
• Results posted: 2020-05-19

Locations

10 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study