Gene Therapy for Hemophilia B
Trial Summary
What is the purpose of this trial?
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile.The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.
Research Team
Steven Pipe, MD
Principal Investigator
University of Michigan
Eligibility Criteria
This trial is for males over 18 with severe or moderately severe hemophilia B, who have had at least 150 days of prior treatment with factor IX protein. Participants must not have a history of gene therapy, active hepatitis B or C infections, uncontrolled HIV, or recent use of experimental drugs.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Lead-in Period
Subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary
Treatment
Single infusion of AAV5-hFIXco-Padua (AMT-061) administered at baseline
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term Follow-up
Follow up and assess any adverse events reported for safety
Treatment Details
Interventions
- AAV5-hFIXco-Padua
- Factor IX (FIX)
Find a Clinic Near You
Who Is Running the Clinical Trial?
UniQure Biopharma B.V.
Lead Sponsor
CSL Behring
Lead Sponsor
Dr. Paul McKenzie
CSL Behring
Chief Executive Officer since 2023
PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania
Dr. Bill Mezzanotte
CSL Behring
Chief Medical Officer since 2021
MD from Duke University