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
Data Collection
Blood Coagulation Disorders+6
+ Hemophilia B
+ Hematologic Diseases
Treatment Study
Summary
Study start date: January 1, 2004
Actual date on which the first participant was enrolled.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).
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.15 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Male
Biological sex of participants that are eligible to enroll.Over 18 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 3 locations
The Children's Hospital of Philadelphia
Philadelphia, United StatesThe Hemophilia Center of Western Pennsylvania
Pittsburgh, United States