Scientists gather data to pave way for hemophilia gene therapy

NCT ID NCT03587116

Summary

This study aimed to collect detailed information on how well current standard treatments work for adults with severe hemophilia A or B. Researchers tracked bleeding rates and treatment use in 212 participants for at least six months. This data serves as a crucial baseline to compare against future gene therapy treatments being tested in separate Phase 3 trials.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Acibadem Adana Hospital, Department of Pediatric Hematology

    Adana, 01130, Turkey (Türkiye)

  • Akdeniz University Medical Faculty Hospital

    Antalya, 07070, Turkey (Türkiye)

  • Alliance for Childhood Diseases

    Las Vegas, Nevada, 89135, United States

  • Bloodworks NW

    Seattle, Washington, 98104, United States

  • CHRU de Brest - La Cavale Blanche

    Brest, 29200, France

  • CHU Hôtel-Dieu

    Nantes, 44093, France

  • Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes

    Vitória, Espírito Santo, 29047-105, Brazil

  • Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP

    Campinas, São Paulo, 13083-878, Brazil

  • Centro de Hemoterapia e Hematologia do Para - Fundação HEMOPA

    Belém, Pará, 66033-000, Brazil

  • Changhua Christian Hospital

    Changhua, 500, Taiwan

  • Chung Shan Medical University Hospital

    Taichung, 40201, Taiwan

  • Clinical Research Facility

    Glasgow, G31 2ER, United Kingdom

  • Clinical and Translational Research Unit (CTRU)

    Palo Alto, California, 94304, United States

  • Cliniques universitaires Saint-Luc/Unité d'Hématology et Hémostase

    Brussels, 1200, Belgium

  • Department of Haematology

    Glasgow, G4 0SF, United Kingdom

  • Ege Universitesi Tip Fakultesi Cocuk Sagligi Ve Hastaliklari Anabilim Dali Pediatric Hematoloji

    Bornova, İ̇zmir, 35040, Turkey (Türkiye)

  • Ege Universitesi Tip Fakultesi Hematoloji BD

    Bornova, İ̇zmir, 35040, Turkey (Türkiye)

  • Emory University Hospital

    Atlanta, Georgia, 30322, United States

  • Emory University Hospital Midtown

    Atlanta, Georgia, 30308, United States

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150, Australia

  • Gaziantep University Sahinbey Training and Research Hospital

    Gaziantep, 27310, Turkey (Türkiye)

  • General Hospital of Athens "Hippokration"

    Athens, Attica, 11527, Greece

  • General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center

    Athens, Attica, 11527, Greece

  • Guy's and St Thomas' NHS Foundation Trust

    London, SE1 7EH, United Kingdom

  • H.U. La Paz.

    Madrid, 28046, Spain

  • H.U. Rio Hortega

    Valladolid, 47012, Spain

  • Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • Hopital Necker, Hematologie Adultes

    Paris, 75015, France

  • Hospital Das Clínicas Da Faculdade de Medicina de Ribeirao Preto Da Universidade de Sao Paulo

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hospital Universitari Vall d´Hebrón

    Barcelona, 08035, Spain

  • Hospital Universitario Virgen de la Arrixaca

    El Palmar, Murcia, 30120, Spain

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

    São Paulo, 05403-000, Brazil

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 08025, Spain

  • Hôpital Cardiologique Louis Pradel

    Bron, 69677, France

  • IRCCS - AOU di Bologna, Policlinico di Sant'Orsola

    Bologna, BO, 40138, Italy

  • Indiana Hemophilia & Thrombosis Center, Inc.

    Indianapolis, Indiana, 46260, United States

  • Investigational Drug Service

    Atlanta, Georgia, 30322, United States

  • Istanbul University Oncology Institute

    Istanbul, 34093, Turkey (Türkiye)

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • King Abdulaziz Medical City

    Riyadh, 11426, Saudi Arabia

  • King Faisal Specialist Hospital & Research Center

    Riyadh, Saudi Arabia

  • Kyung Hee University Hospital At Gangdong

    Seoul, 05278, South Korea

  • Kyungpook National University Hospital

    Daegu, 41944, South Korea

  • Lucile Packard Childrens Hospital

    Palo Alto, California, 94304, United States

  • McMaster University Medical Centre - Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5, Canada

  • Mississippi Center for Advanced Medicine

    Madison, Mississippi, 39110, United States

  • Nagoya University Hospital - Transfusion Medicine

    Nagoya, Aichi-ken, 466-8560, Japan

  • Nara Medical University Hospital

    Kashihara, Nara, 634-8522, Japan

  • National Center for Child Health and Development

    Setagaya-ku, Tokyo, 157-8535, Japan

  • National Taiwan University Hospital

    Taipei, 10002, Taiwan

  • Newcastle upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom

  • Non Malignant Haematology Research Unit

    Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom

  • Ogikubo Hospital

    Suginami-ku, Tokyo, 167-0035, Japan

  • Penn Blood Disorder Center

    Philadelphia, Pennsylvania, 19104, United States

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029, Australia

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050, Australia

  • SODc. Malattie Emorragiche e della Coagulazione Centro di Riferimento Regionale per le

    Florence, 50134, Italy

  • Saitama Medical University Hospital

    Iruma-gun, Saitama, 350-0495, Japan

  • Sapporo Tokushukai Hospital

    Sapporo, Hokkaido, 004-0041, Japan

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722, South Korea

  • Skåne University Hospital

    Malmo, 205 02, Sweden

  • St. Michael's Hospital

    Toronto, Ontario, M5B 1W8, Canada

  • Stanford Health Care

    Stanford, California, 94305, United States

  • Taichung Veterans General Hospital

    Taichung, 40705, Taiwan

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • The Chaim Sheba Medical Center, The National Hemophilia Center

    Tel Litwinsky, 5262000, Israel

  • UOC Medicina Interna - Malattie Emorragiche e Trombotiche

    Naples, Naples, 80131, Italy

  • Universitaetsklinikum Giessen

    Giessen, 35392, Germany

  • Universitatsklinikum Bonn. Anstalt des oeffentlichen Rechts

    Bonn, 53127, Germany

  • University of California, San Francisco - Outpatient Hematology Clinic

    San Francisco, California, 94143, United States

  • Università degli studi di Roma "La Sapienza"- Policlinico Umberto I

    Roma, 00161, Italy

  • Universität und Universitätsklinikum des Saarlandes

    Homburg/Saar, 66421, Germany

  • Vivantes Klinikum Friedrichshain

    Berlin, 10249, Germany

  • lnstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO

    Rio de Janeiro, 20211-030, Brazil

Conditions

Explore the condition pages connected to this study.