HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Completed · Phase 3

Conditions studied: Hemophilia B

In brief

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.

Key facts

Study ID

NCT03569891

Run by

CSL Behring

People needed

67

Starts

2018-06-27

Expected to finish

2025-03-19

Last updated by the study team

2026-03-27

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male
• Age ≥18 years
• Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis
• >150 previous exposure days of treatment with factor IX protein

You may not qualify if…

• History of factor IX inhibitors
• Positive factor IX inhibitor test at screening
• Select screening laboratory value >2 times upper limit of normal
• Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
• Active infection with hepatitis B or C virus at screening
• History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
• Previous gene therapy treatment
• Receipt of an experimental agent within 60 days prior to screening
• Current participation or anticipated participation within one year after study drug administration in this trial in any other interventional clinical trial involving drugs or devices

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Los Angeles Orthopedic Hospital — Los Angeles, California, United States
• Children's Hospital of Los Angeles — Los Angeles, California, United States
• University of California, Davis — Sacramento, California, United States
• University of California, San Diego — San Diego, California, United States
• University of Colorado Denver — Aurora, Colorado, United States
• Children's National Medical Center Hematology and Oncology — Washington D.C., District of Columbia, United States
• University of South Florida — Tampa, Florida, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Hemophilia Center of Western New York — Buffalo, New York, United States
• University of North Carolina, Chapel Hill — Chapel Hill, North Carolina, United States
• Oregon Health & Science University — Portland, Oregon, United States
• University of Tennessee Health Science Center — Memphis, Tennessee, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• University of Texas Health Science Center & Medical School — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Washington Institute for Coagulation — Seattle, Washington, United States
• Bloodworks Northwest — Seattle, Washington, United States
• Cliniques Universitaires Saint-Luc — Brussels, Belgium
• University Hospital Leuven — Leuven, Belgium
• Righospitalet — Copenhagen, Denmark
• Vivantes Klinikum im Friedrichshain — Berlin, Germany
• Klinikum der Johann Wolfgang Goethe Universitat — Frankfurt am Main, Germany
• National Coagulation Centre, St James's Hospital — Dublin, Ireland

Full record on ClinicalTrials.gov

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