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Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
This study is currently recruiting patients.
Sponsored by: | Avigen
Stanford University
Children's Hospital of Philadelphia
The Hemophilia Center of Western Pennsylvania
University of Washington
University of Texas
University of Campinas, Brazil
Christian Medical College, Vellore, India
Royal Prince Alfred Hospital, Sydney, Australia
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Information provided by: | Avigen |
Purpose
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).
Condition | Treatment or Intervention | Phase |
---|---|---|
Hemophilia B |
Gene Transfer: Adeno-Associated Viral with Human Factor IX |
Phase I Phase II |
MedlinePlus related topics: Bleeding Disorders; Blood and Blood Disorders; Genetic Disorders
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study
Official Title: A Phase I Safety Study in Subjects with Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX into the Liver
Expected Total Enrollment: 15
Study start: January 2004
Eligibility
Ages Eligible for Study: 18 Years and above, Genders Eligible for Study: Male
Criteria
Location and Contact Information
More Information
Avigen Ongoing Clinical Trials
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