Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

Phase 1

Terminated

• Conditions: Hemophilia B

• Registration Number: NCT00076557
• Lead Sponsor: Avigen

Brief Summary

In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).

Detailed Description

Not available

Study Timeline

• Status Verified: 2004-01
• Study Start: 2004-01
• Study First Submitted: 2004-01-26
• Study First Submitted QC: 2004-01-27
• Study First Posted: 2004-01-28
• Last Update Submitted: 2007-04-02
• Last Update Posted: 2007-04-04

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Trial Locations

Locations (3)

Stanford University; 🇺🇸 Palo Alto, California, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

The Hemophilia Center of Western Pennsylvania; 🇺🇸 Pittsburgh, Pennsylvania, United States