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

This study has been terminated.
Sponsor:
Collaborators:
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
First received: January 26, 2004
Last updated: April 2, 2007
Last verified: January 2004
  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
Genetic: Adeno-Associated Viral with Human Factor IX
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Additional Information:
No publications provided

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

Keywords provided by Avigen:
Hemophilia B
Factor IX
Gene Transfer
Adeno-Associated Virus (AAV)

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on November 25, 2014