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S256 : 26 WEEK EFFICACY AND SAFETY OF ETRANACOGENE DEZAPARVOVEC (AAV5-PADUA HFIX VARIANT; AMT-061) IN ADULTS WITH SEVERE OR MODERATE-SEVERE HEMOPHILIA B TREATED IN THE PHASE 3 HOPE-B CLINICAL TRIAL

Researchers

Presenter

  • Steven Pipe

Principal Investigators

  • Michael Recht

  • Nigel S Key

  • Susan Lattimore

  • Giancarlo Castaman

  • Paul van der Valk

  • Kathelinje Peerlinck

  • Michiel Coppens

  • Niamh M O'Connell

  • K John Pasi

  • Peter Kampmann

  • Karina Meijer

  • Annette von Drygalski

  • Guy Young

  • Cedric Hermans

  • Jan Astermark

  • Robert Klamroth

  • Richard Lemons

  • Nathan Visweshwar

  • Shelley Crary

  • Syed R S Kazmi

  • Emily Symington

  • Miguel A Escobar

  • Esteban Gomez

  • Rebecca Kruse-Jarres

  • Adam Kotowski

  • Doris Quon

  • Michael Wang

  • Alison P Wheeler

  • Eileen K Sawyer

  • David Cooper

  • Valerie Ferriera

  • Wolfgang Miesbach

Medical Centers

  • Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, United States

  • uniQure Inc, Lexington, United States

  • Department of Epidemiology, University of North Carolina, Chapel Hill, NC, United States

  • Surgery, Skane University Hospital, Malmo, Sweden

  • Erasmus University Medical Center, Rotterdam, Netherlands

  • University of South Florida Health, Tampa, United States

  • Rigshospitalet, Copenhagen, Denmark

  • University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom

  • University Hospital Frankfurt, Frankfurt, Germany

  • Cambridge University Health Partners, Addenbrookes Hospital, Cambridge, United Kingdom

  • Department of Vascular Medicine and Haemostasis, University Hospitals Leuven, Leuven, Belgium

  • Oregon Health &amp

  • WNY Bloodcare,New York,United States

  • Center for Bleeding Disorders, Careggi UniversityHospital, Florence, Italy

  • Phoenix Children's Hospital, Phoenix, Arizona, United States

  • Universitair Medisch Centrum, Utrecht, Netherlands

  • Universitair Medisch Centrum Groningen, Groningen, Netherlands

  • Vivantes Klinikum Am Friedrichshain, Berlin, Germany

  • University of California San Diego, CA, United States

  • uniQure Inc, Lexington, United States

  • National Coagulation Centre, St James's Hospital, Dublin, Ireland

  • Bloodworks Northwest, Seattle, United States

  • uniQure B.V., Amsterdam, Netherlands

  • The University of Texas Health Science Center at Houston, Houston, TX

  • University of Utah, Salt Lake City, Utah, United States

  • University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States

  • Science University, Portland, Oregon, United States

  • Department of Vascular Medicine, Amsterdam University Medical Centres, Amsterdam

  • University of Colorado Hemophilia and Thrombosis Center,Anschutz Medical Campus,Aurora,United States

  • Royal London Haemophilia Centre, Barts and the London School of Medicine and Dentistry, London, United Kingdom

  • Cliniques Universitaires Saint-Luc, Universite Catholique de Louvain, Brussels, Belgium

  • The University of Michigan, Ann Arbor, Michigan, United States

  • Vanderbilt University, Medical Center, Nashville, United States

  • Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA

Locations

  • United States

  • Sweden

  • Netherlands

  • Denmark

  • United Kingdom

  • Germany

  • Belgium

  • Italy

  • Ireland

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Hemophilia

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Open Label

  • Single Arm

  • Prospective

Phase

  • III

Study Id's

  • NCT03569891

Sponsors

  • N/A

Result

  • N/A

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