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

This study has been terminated.

Sponsors and Collaborators:	Avigen Stanford University Children's Hospital of Philadelphia The Hemophilia Center of Western Pennsylvania University of Washington The University of Texas Health Science Center, Houston University of Campinas, Brazil Christian Medical College, Vellore, India Royal Prince Alfred Hospital, Sydney, Australia
Information provided by:	Avigen
ClinicalTrials.gov Identifier:	NCT00076557

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	Gene Transfer: Adeno-Associated Viral with Human Factor IX	Phase I Phase II

Genetics Home Reference related topics: hemophilia L1 syndrome

Drug Information available for: Factor IX

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study
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

Further study details as provided by Avigen:

Estimated Enrollment:	15
Study Start Date:	January 2004

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00076557

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

Sponsors and Collaborators

Avigen

Stanford University

Children's Hospital of Philadelphia

The Hemophilia Center of Western Pennsylvania

University of Washington

The University of Texas Health Science Center, Houston

University of Campinas, Brazil

Christian Medical College, Vellore, India

Royal Prince Alfred Hospital, Sydney, Australia

More Information

Avigen Ongoing Clinical Trials This link exits the ClinicalTrials.gov site

Study ID Numbers:	BB IND 9398
Study First Received:	January 26, 2004
Last Updated:	April 2, 2007
ClinicalTrials.gov Identifier:	NCT00076557
Health Authority:	United States: Food and Drug Administration

Keywords provided by Avigen:

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

Study placed in the following topic categories:

Virus Diseases
Hemophilia B
Hemorrhagic Disorders
Genetic Diseases, Inborn
Hematologic Diseases

Blood Coagulation Disorders
Hemophilia A
Genetic Diseases, X-Linked
Hemostatic Disorders

Additional relevant MeSH terms:

Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders

ClinicalTrials.gov processed this record on January 15, 2009