HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
NCT ID: NCT03569891
Last Updated: 2025-05-04
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
67 participants
INTERVENTIONAL
2018-06-27
2025-03-19
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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AMT-061
Single infusion of AMT-061
Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline. After study drug administration (post study drug), subjects will be monitored for tolerance to the study drug and detection of potential immediate AEs at the clinical trial site for a few hours after dosing.
AAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)
FIX replacement (Lead-in Period)
During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.
Factor IX (FIX)
During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.
Interventions
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AAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua (AMT-061)
Factor IX (FIX)
During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Age ≥18 years
3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis
4. \>150 previous exposure days of treatment with factor IX protein
Exclusion Criteria
2. Positive factor IX inhibitor test at screening
3. Select screening laboratory value \>2 times upper limit of normal
4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
5. Active infection with hepatitis B or C virus at screening
6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
7. Previous gene therapy treatment
8. Receipt of an experimental agent within 60 days prior to screening
9. 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
18 Years
MALE
No
Sponsors
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CSL Behring
INDUSTRY
Responsible Party
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Principal Investigators
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Steven Pipe, MD
Role: PRINCIPAL_INVESTIGATOR
University of Michigan
Locations
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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
Amsterdam UMC - Locatie AMC
Amsterdam, , Netherlands
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Erasmus MC
Rotterdam, , Netherlands
UMC Utrecht, Van Creveldkliniek
Utrecht, , Netherlands
Center for Thrombosis and Hemostasis Skåne University Hospital Malmö
Malmo, , Sweden
The Cambridge Haemophilia and Thrombophilia Centre Camridge University Hospitals NHS Foundation Trust - Box 217 Addenbrooke's Hospital
Cambridge, , United Kingdom
The Royal London Hospital (Barts Health NHS Trust)
London, , United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, , United Kingdom
Countries
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References
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O'Connell N, van der Valk P, Le Quellec S, Gomez E, Monahan PE, Crary SE, Coppens M, Lemons R, Castaman G, Klamroth R, Symington E, Quon DV, Kampmann P. Invasive procedures and surgery following etranacogene dezaparvovec gene therapy in people with hemophilia B. J Thromb Haemost. 2025 Jan;23(1):73-84. doi: 10.1016/j.jtha.2024.08.027. Epub 2024 Sep 26.
Coppens M, Pipe SW, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan PE, Leebeek FWG; HOPE-B Investigators. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial. Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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CSL222_3001 (CT-AMT-061-02)
Identifier Type: -
Identifier Source: org_study_id
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