Brief Title
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
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
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).
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Condition
Hemophilia B
Intervention
Adeno-Associated Viral with Human Factor IX
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
15
Start Date
January 2004
Eligibility Criteria
- Males with severe hemophilia B with Factor IX activity level < 1% of normal.
- Life expectancy of > 1 year.
- Age > 18 years old.
- Ability to give informed consent.
- Greater than twenty exposure days of treatment with Factor IX protein.
- No history or presence of an inhibitor to Factor IX protein.
- Subjects must be able to receive Factor IX protein on a home infusion protocol.
- Subjects must have a normal protime (PT).
- Hepatitis C infected subjects will be evaluated for liver fibrosis based on liver
biopsy data graded on a scale of 0-4 (Poynard et. al., 1997). Subjects who are
Hepatitis C antibody and RNA positive and have not had a liver biopsy within the last
36 months will be required to have one.
- Subjects must have low AAV titer.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT00076557
Organization ID
BB IND 9398
Study Sponsor
Avigen
Collaborators
Stanford University
Study Sponsor
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Verification Date
January 2004