Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Stopped early · Phase 1/Phase 2
Conditions studied: Hemophilia B
In brief
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).
Key facts
- Study ID
- NCT00076557
- Run by
- Avigen
- People needed
- 15
- Starts
- 2004-01-01
- Last updated by the study team
- 2007-04-04
Who can join
Age: 18 and older. Sex: male. Healthy volunteers: not accepted.
Where it is running
- 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
Full record on ClinicalTrials.gov
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