NCT03569891CompletedPhase 3
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B
Source: ClinicalTrials.gov · Last updated 27 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 67 (actual)
- First submitted
- 14 Jun 2018
- Start date
- 27 Jun 2018
- Primary completion date
- 22 Sept 2021
- Lead sponsor
- CSL Behring
- Diseases
- Haemophilia
- NCT number
- NCT03569891
- EU CT number
- 2024-510738-42-00
- EudraCT number
- 2017-004305-40
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 67
- From 27 Jun 2018 to the primary completion date, 22 Sept 2021
- 32 sites in 8 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Positive factor IX inhibitor test at screening
- 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
Other illness
- History of factor IX inhibitors
- 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
Lab values
- Select screening laboratory value \>2 times upper limit of normal
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: Male · Does not accept healthy volunteers
Sites (32)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| UZ Leuven Coordinator of this site? Confirm its status | Leuven | Belgium | Completed |
| Cliniques Universitaires Saint-Luc Coordinator of this site? Confirm its status | Sint-Lambrechts-Woluwe | Belgium | Completed |
| Vivantes Netzwerk fuer Gesundheit GmbH Coordinator of this site? Confirm its status | Berlin | Germany | Completed |
| Rigshospitalet Coordinator of this site? Confirm its status | Copenhagen Oe | Denmark | Completed |
| The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's Hospital Coordinator of this site? Confirm its status | Cambridge | United Kingdom | Unknown status |
| The Royal London Hospital - Barts Health Nhs Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| University Hospital Southampton NHS Foundation Trust Coordinator of this site? Confirm its status | Southampton | United Kingdom | Unknown status |
| St James's Hospital Coordinator of this site? Confirm its status | Dublin 8 | Ireland | Completed |
| Academisch Medisch Centrum Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Completed |
| Universitair Medisch Centrum Groningen Coordinator of this site? Confirm its status | Groningen | Netherlands | Completed |
| Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) Coordinator of this site? Confirm its status | Rotterdam | Netherlands | Completed |
| Universitair Medisch Centrum Utrecht Coordinator of this site? Confirm its status | Utrecht | Netherlands | Completed |
| Region Skane Skanes Universitetssjukhus Coordinator of this site? Confirm its status | Malmo | Sweden | Completed |
| University of Michigan Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| University of Colorado Denver Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| Hemophilia Center of Western New York Coordinator of this site? Confirm its status | Buffalo | United States | Unknown status |
| University of North Carolina, Chapel Hill Coordinator of this site? Confirm its status | Chapel Hill | United States | Unknown status |
| University of Texas Health Science Center & Medical School Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| Arkansas Children's Hospital Coordinator of this site? Confirm its status | Little Rock | United States | Unknown status |
| Children's Hospital of Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Los Angeles Orthopedic Hospital Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| University of Tennessee Health Science Center Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
| Vanderbilt University Medical Center Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Phoenix Children s Hospital Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| Oregon Health Science University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| University of California - Davis Coordinator of this site? Confirm its status | Sacramento | United States | Unknown status |
| University of Utah Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
| University of California San Diego Coordinator of this site? Confirm its status | San Diego | United States | Unknown status |
| Washington Institute for Coagulation Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| Bloodworks Northwest Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| University of South Florida Coordinator of this site? Confirm its status | Tampa | United States | Unknown status |
| Children's National Medical Center Hematology and Oncology Coordinator of this site? Confirm its status | Washington D.C. | United States | Unknown status |
Related publications
- Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.N Engl J Med · 2026 · PMID 41358585
This page is information, not medical advice. Discuss any trial with your doctor.