Hemophilia B
Conditions
Keywords
Hemophilia B, Factor IX, Gene Transfer, Adeno-Associated Virus (AAV)
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).
Interventions
GENETICAdeno-Associated Viral with Human Factor IX
Sponsors
Stanford University
Children's Hospital of Philadelphia
The Hemophilia Center of Western Pennsylvania
University of Washington
The University of Texas Health Science Center, Houston
University of Campinas, Brazil
Christian Medical College, Vellore, India
Royal Prince Alfred Hospital, Sydney, Australia
Avigen
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion 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.
Countries
United States
Outcome results
None listed