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

NCT00076557 | Terminated Phase 1

• 1 other identifier: BB IND 9398
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 3

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 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).

Conditions

Hemophilia B

Keywords

Hemophilia B; Factor IX; Gene Transfer; Adeno-Associated Virus (AAV)

Interventions

Adeno-Associated Viral with Human Factor IX GENETIC

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

* 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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

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

Study Sites (3)

Stanford University

Palo Alto, California, 94305, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

The Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213, United States

Location

Related Links

• Avigen Ongoing Clinical Trials

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: January 26, 2004
• First Posted: January 28, 2004
• Study Start: January 1, 2004
• Last Updated: April 4, 2007

Record last verified: 2004-01

Locations

US (3)