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
Phase 1/Phase 2
18 Years
N/A
18 Years
N/A
Not Enrolling
Male
Hemophilia B
Male
Hemophilia B
Trial Information
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
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.
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Authority:
United States: Food and Drug Administration
Study ID:
BB IND 9398
NCT ID:
NCT00076557
Start Date:
January 2004
Completion Date:
Related Keywords:
- Hemophilia B
- Hemophilia B
- Factor IX
- Gene Transfer
- Adeno-Associated Virus (AAV)
- Hemophilia B
- Hemophilia A
Name | Location |
|---|---|
| Stanford University | Stanford, California 94305 |
| The Children's Hospital of Philadelphia | Philadelphia, Pennsylvania 19104 |
| The Hemophilia Center of Western Pennsylvania | Pittsburgh, Pennsylvania 15213 |
