Clinical trial NCT00076557: Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Hemophilia B
Keywords: Hemophilia B
Factor IX
Gene Transfer
Adeno-Associated Virus (AAV)
Lead sponsor: Avigen
Sponsor class: INDUSTRY
Collaborators: name: Stanford University
name: Children's Hospital of Philadelphia
name: The Hemophilia Center of Western Pennsylvania
name: University of Washington
name: The University of Texas Health Science Center, Houston
name: University of Campinas, Brazil
name: Christian Medical College, Vellore, India
name: Royal Prince Alfred Hospital, Sydney, Australia
First posted: 2004-01-28
Enrollment: 15
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: The Hemophilia Center of Western Pennsylvania
state: Pennsylvania
Study officials:
References: