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
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Locations
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Acibadem Adana Hospital, Department of Pediatric Hematology
Adana, 01130, Turkey (Türkiye)
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Akdeniz University Medical Faculty Hospital
Antalya, 07070, Turkey (Türkiye)
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Alliance for Childhood Diseases
Las Vegas, Nevada, 89135, United States
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Bloodworks NW
Seattle, Washington, 98104, United States
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CHRU de Brest - La Cavale Blanche
Brest, 29200, France
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CHU Hôtel-Dieu
Nantes, 44093, France
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Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes
Vitória, Espírito Santo, 29047-105, Brazil
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Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP
Campinas, São Paulo, 13083-878, Brazil
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Centro de Hemoterapia e Hematologia do Para - Fundação HEMOPA
Belém, Pará, 66033-000, Brazil
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Changhua Christian Hospital
Changhua, 500, Taiwan
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Chung Shan Medical University Hospital
Taichung, 40201, Taiwan
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Clinical Research Facility
Glasgow, G31 2ER, United Kingdom
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Clinical and Translational Research Unit (CTRU)
Palo Alto, California, 94304, United States
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Cliniques universitaires Saint-Luc/Unité d'Hématology et Hémostase
Brussels, 1200, Belgium
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Department of Haematology
Glasgow, G4 0SF, United Kingdom
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Ege Universitesi Tip Fakultesi Cocuk Sagligi Ve Hastaliklari Anabilim Dali Pediatric Hematoloji
Bornova, İ̇zmir, 35040, Turkey (Türkiye)
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Ege Universitesi Tip Fakultesi Hematoloji BD
Bornova, İ̇zmir, 35040, Turkey (Türkiye)
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Emory University Hospital
Atlanta, Georgia, 30322, United States
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Emory University Hospital Midtown
Atlanta, Georgia, 30308, United States
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Fiona Stanley Hospital
Murdoch, Western Australia, 6150, Australia
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Gaziantep University Sahinbey Training and Research Hospital
Gaziantep, 27310, Turkey (Türkiye)
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General Hospital of Athens "Hippokration"
Athens, Attica, 11527, Greece
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General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center
Athens, Attica, 11527, Greece
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Guy's and St Thomas' NHS Foundation Trust
London, SE1 7EH, United Kingdom
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H.U. La Paz.
Madrid, 28046, Spain
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H.U. Rio Hortega
Valladolid, 47012, Spain
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Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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Hopital Necker, Hematologie Adultes
Paris, 75015, France
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Hospital Das Clínicas Da Faculdade de Medicina de Ribeirao Preto Da Universidade de Sao Paulo
Ribeirão Preto, São Paulo, 14051-140, Brazil
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Hospital Universitari Vall d´Hebrón
Barcelona, 08035, Spain
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Hospital Universitario Virgen de la Arrixaca
El Palmar, Murcia, 30120, Spain
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Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, 05403-000, Brazil
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Hospital de la Santa Creu i Sant Pau
Barcelona, 08025, Spain
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Hôpital Cardiologique Louis Pradel
Bron, 69677, France
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IRCCS - AOU di Bologna, Policlinico di Sant'Orsola
Bologna, BO, 40138, Italy
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Indiana Hemophilia & Thrombosis Center, Inc.
Indianapolis, Indiana, 46260, United States
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Investigational Drug Service
Atlanta, Georgia, 30322, United States
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Istanbul University Oncology Institute
Istanbul, 34093, Turkey (Türkiye)
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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King Abdulaziz Medical City
Riyadh, 11426, Saudi Arabia
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King Faisal Specialist Hospital & Research Center
Riyadh, Saudi Arabia
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Kyung Hee University Hospital At Gangdong
Seoul, 05278, South Korea
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Kyungpook National University Hospital
Daegu, 41944, South Korea
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Lucile Packard Childrens Hospital
Palo Alto, California, 94304, United States
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McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5, Canada
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Mississippi Center for Advanced Medicine
Madison, Mississippi, 39110, United States
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Nagoya University Hospital - Transfusion Medicine
Nagoya, Aichi-ken, 466-8560, Japan
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Nara Medical University Hospital
Kashihara, Nara, 634-8522, Japan
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National Center for Child Health and Development
Setagaya-ku, Tokyo, 157-8535, Japan
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National Taiwan University Hospital
Taipei, 10002, Taiwan
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Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom
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Non Malignant Haematology Research Unit
Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom
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Ogikubo Hospital
Suginami-ku, Tokyo, 167-0035, Japan
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Penn Blood Disorder Center
Philadelphia, Pennsylvania, 19104, United States
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Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
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Royal Brisbane and Women's Hospital
Herston, Queensland, 4029, Australia
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Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050, Australia
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SODc. Malattie Emorragiche e della Coagulazione Centro di Riferimento Regionale per le
Florence, 50134, Italy
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Saitama Medical University Hospital
Iruma-gun, Saitama, 350-0495, Japan
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Sapporo Tokushukai Hospital
Sapporo, Hokkaido, 004-0041, Japan
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Severance Hospital, Yonsei University Health System
Seoul, 03722, South Korea
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Skåne University Hospital
Malmo, 205 02, Sweden
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St. Michael's Hospital
Toronto, Ontario, M5B 1W8, Canada
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Stanford Health Care
Stanford, California, 94305, United States
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Taichung Veterans General Hospital
Taichung, 40705, Taiwan
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The Alfred Hospital
Melbourne, Victoria, 3004, Australia
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The Chaim Sheba Medical Center, The National Hemophilia Center
Tel Litwinsky, 5262000, Israel
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UOC Medicina Interna - Malattie Emorragiche e Trombotiche
Naples, Naples, 80131, Italy
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Universitaetsklinikum Giessen
Giessen, 35392, Germany
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Universitatsklinikum Bonn. Anstalt des oeffentlichen Rechts
Bonn, 53127, Germany
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University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, 94143, United States
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Università degli studi di Roma "La Sapienza"- Policlinico Umberto I
Roma, 00161, Italy
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Universität und Universitätsklinikum des Saarlandes
Homburg/Saar, 66421, Germany
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Vivantes Klinikum Friedrichshain
Berlin, 10249, Germany
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lnstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
Rio de Janeiro, 20211-030, Brazil
Conditions
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