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

Tracking Information
First Received Date ^ICMJE	January 26, 2004
Last Updated Date	April 2, 2007
Start Date ^ICMJE	January 2004
Primary Completion Date
Current Primary Outcome Measures ^ICMJE
Original Primary Outcome Measures ^ICMJE
Change History	Complete list of historical versions of study NCT00076557 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE
Original Secondary Outcome Measures ^ICMJE

Descriptive Information
Brief Title ^ICMJE	Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Official Title ^ICMJE	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
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).
Detailed Description
Study Phase	Phase I, Phase II
Study Type ^ICMJE	Interventional
Study Design ^ICMJE	Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study
Condition ^ICMJE	Hemophilia B
Intervention ^ICMJE	Gene Transfer: Adeno-Associated Viral with Human Factor IX
Study Arms / Comparison Groups
Publications *
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Terminated
Enrollment ^ICMJE	15
Completion Date
Primary Completion Date
Eligibility Criteria ^ICMJE	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.
Gender	Male
Ages	18 Years and older
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT ID ^ICMJE	NCT00076557
Responsible Party
Study ID Numbers ^ICMJE	BB IND 9398
Study Sponsor ^ICMJE	Avigen
Collaborators ^ICMJE	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
Investigators ^ICMJE
Information Provided By	Avigen
Verification Date	January 2004
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP