Status:
TERMINATED
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Lead Sponsor:
Avigen
Collaborating Sponsors:
Stanford University
Children's Hospital of Philadelphia
Conditions:
Hemophilia B
Eligibility:
MALE
18+ years
Phase:
PHASE1
PHASE2
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 vecto...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
January 1 2004
Trial Type :
INTERVENTIONAL
End Date :
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT00076557
Start Date
January 1 2004
Last Update
April 4 2007
Active Locations (3)
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1
Stanford University
Palo Alto, California, United States, 94305
2
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
3
The Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213