Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Hemophilia B
About this trial
This is an interventional treatment trial for Hemophilia B focused on measuring Hemophilia B, Factor IX, Gene Transfer, Adeno-Associated Virus (AAV)
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.
Sites / Locations
- Stanford University
- The Children's Hospital of Philadelphia
- The Hemophilia Center of Western Pennsylvania