Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
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Purpose
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).
| Condition | Intervention | Phase |
|---|---|---|
|
Hemophilia B |
Genetic: Adeno-Associated Viral with Human Factor IX |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
- 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.
Contacts and Locations| United States, California | |
| Stanford University | |
| Palo Alto, California, United States, 94305 | |
| United States, Pennsylvania | |
| The Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| The Hemophilia Center of Western Pennsylvania | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
More Information
Additional Information:
No publications provided
| ClinicalTrials.gov Identifier: | NCT00076557 History of Changes |
| Other Study ID Numbers: | BB IND 9398 |
| Study First Received: | January 26, 2004 |
| Last Updated: | April 2, 2007 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Avigen:
|
Hemophilia B Factor IX Gene Transfer Adeno-Associated Virus (AAV) |
Additional relevant MeSH terms:
|
Hemophilia B Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |
ClinicalTrials.gov processed this record on May 23, 2013