Clinical Trial: A Gene Therapy Study for Hemophilia B

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Gene Therapy, Open-label, Dose-escalation Study of SPK-9001 [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B

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

Detailed Summary:

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).


Sponsor: Spark Therapeutics

Current Primary Outcome: Number of participants experiencing drug-related adverse events [ Time Frame: 1 year ]

As assessed by physical exam, vital signs, standard clinical labs, and Bethesda assay for FIX inhibitor


Original Primary Outcome: Same as current

Current Secondary Outcome: Changes from baseline in circulating FIX activity (IU/dL or % normal) [ Time Frame: 1 Year ]

Original Secondary Outcome: Same as current

Information By: Spark Therapeutics

Dates:
Date Received: June 18, 2015
Date Started: September 2015
Date Completion: January 2019
Last Updated: February 22, 2017
Last Verified: February 2017