Trial Summary
What is the purpose of this trial?
The purpose of this study is to determine the safety of giving a normal factor IX gene to treat individuals who have an abnormal or no factor IX gene. Recruitment will be limited to adults (≥ 18 years) with a confirmed diagnosis of hemophilia B (HB), resulting from a missense mutation in the coagulation factor IX (FIX) gene or a nonsense mutation that has not been associated with an inhibitor. Only subjects who have no evidence of active hepatitis or anti-hFIX antibodies, and who have been treated/exposed to Factor IX concentrates for at least ten years and have had an average of 3 bleeding episodes per year requiring FIX administration will be enrolled. Patients will be recruited within the United States for treatment at St. Jude Children's Research Hospital, and patients will be recruited in England and other countries for treatment in London by our British collaborators.
Research Team
Ulrike Reiss, MD
Principal Investigator
St. Jude Children's Research Hospital
Eligibility Criteria
Adult males (18+) with severe Hemophilia B, treated with FIX products for over 10 years or have had at least 50 exposure days. Candidates must average a minimum of three bleeding episodes per year requiring FIX infusions and be free of inhibitors to FIX protein. They should not have active hepatitis, HIV infections under control are eligible, and must agree to use barrier contraception.Inclusion Criteria
Exclusion Criteria
Treatment Details
Interventions
- scAAV2/8-LP1-hFIXco (Virus Therapy)
Find a Clinic Near You
Who Is Running the Clinical Trial?
St. Jude Children's Research Hospital
Lead Sponsor
Dr. James R. Downing
St. Jude Children's Research Hospital
Chief Executive Officer since 2014
MD from University of Michigan Medical School
Dr. Ellis J. Neufeld
St. Jude Children's Research Hospital
Chief Medical Officer since 2017
MD, PhD from Harvard Medical School
Children's Hospital of Philadelphia
Collaborator
Joseph W. St. Geme III
Children's Hospital of Philadelphia
Chief Medical Officer since 2021
MD, PhD, MPH
Madeline Bell
Children's Hospital of Philadelphia
Chief Executive Officer since 2015
BSc in Nursing from Villanova University, MSc in Organizational Dynamics from the University of Pennsylvania
University College, London
Collaborator
Dr. Emma Morris
University College, London
Chief Medical Officer
MD from University College London
Dr. Michael Spence
University College, London
Chief Executive Officer since 2021
PhD in Chemical Engineering from University College London
Hemophilia of Georgia, Inc.
Collaborator
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator
Dr. Gary H. Gibbons
National Heart, Lung, and Blood Institute (NHLBI)
Chief Executive Officer since 2012
MD from Harvard Medical School
Dr. James P. Kiley
National Heart, Lung, and Blood Institute (NHLBI)
Chief Medical Officer since 2011
MD from University of California, San Francisco