Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

Hemophilia B Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00076557
• Other study ID #: BB IND 9398
• Status: Terminated
• Phase: Phase 1/Phase 2
• First received: January 26, 2004
• Last updated: April 2, 2007
• Start date: January 2004

Study information

• Verified date: January 2004
• Source: Avigen
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

• 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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Genetic:
• Adeno-Associated Viral with Human Factor IX

Locations

Country	Name	City	State
United States	Stanford University	Palo Alto	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	The Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania

Sponsors (9)

Lead Sponsor	Collaborator
Avigen	Children's Hospital of Philadelphia, Christian Medical College, Vellore, India, Royal Prince Alfred Hospital, Sydney, Australia, Stanford University, The Hemophilia Center of Western Pennsylvania, The University of Texas Health Science Center, Houston, University of Campinas, Brazil, University of Washington

Country where clinical trial is conducted

United States,