One-Shot gene therapy aims to free patients from lifelong bleeding disorder treatments
NCT ID NCT03861273
Summary
This study is testing a one-time gene therapy for adult men with severe hemophilia B, a genetic disorder that prevents blood from clotting properly. The therapy aims to give the body long-lasting instructions to make its own clotting factor, potentially reducing or eliminating the need for regular infusions. Researchers will compare bleeding rates and treatment use for over a year after the single infusion to the participants' previous standard care.
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Contacts and locations
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Locations
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Acibadem Adana Hospital
Adana, 01130, Turkey (Türkiye)
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ApoEx AB
Malmo, 211 24, Sweden
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Azienda Ospedaliera Universitaria Federico II
Naples, 80131, Italy
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Azienda Ospedaliero Universitaria Careggi
Florence, 50134, Italy
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Center for Human Phenomic Science
Philadelphia, Pennsylvania, 19104, United States
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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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Changhua Christian Hospital
Changhua, 500, Taiwan
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Chung Shan Medical University
Taichung, 40201, Taiwan
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Chung Shan Medical University Hospital
Taichung, 40201, Taiwan
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Ege University Medical Faculty Hospital
Izmir, 35040, Turkey (Türkiye)
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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 "LAIKO", 2nd Regional Blood Transfusion Center
Athens, 11527, Greece
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Glasgow Royal Infirmary
Glasgow, G4 0SF, United Kingdom
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Guy's and St. Thomas' NHS Foundation Trust
London, SE1 7EH, United Kingdom
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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 Cardiologique Louis Pradel - CRTH
Bron, 69677, France
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Hopital Necker
Paris, 75015, France
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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, São Paulo, 05403-010, Brazil
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Indiana Hemophilia & Thrombosis Center, Inc.
Indianapolis, Indiana, 46260, United States
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Indiana Hemophilia and Thrombosis Center, Inc
Indianapolis, Indiana, 46260, United States
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Innovative Hematology, Inc.
Indianapolis, Indiana, 46260, United States
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Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO
Rio de Janeiro, 20211-030, Brazil
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Investigational Drug Service
Philadelphia, Pennsylvania, 19104, United States
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Istanbul University Oncology Institute
Istanbul, 34093, Turkey (Türkiye)
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Juravinski Hospital - Hamilton Health Sciences
Hamilton, Ontario, L8V 1C3, Canada
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Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807, Taiwan
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King Abdulaziz Medical City-Ministry of national guard
Riyadh, Kingdom of Saudi Arabia, 14611, Saudi Arabia
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King Abdullah International Medical Research Center
Riyadh, 11481, Saudi Arabia
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King Faisal Specialist Hospital and Research Centre
Riyadh, 11211, Saudi Arabia
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Kyung Hee University Hospital at Gangdong
Seoul, 05278, South Korea
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Madison Radiological Group
Madison, Mississippi, 39110, 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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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 FT
Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom
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Newcastle upon Tyne Hospitals NHS FT
Newcastle upon Tyne, TYNE & WEAR, NE2 4HH, United Kingdom
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Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE2 4HH, United Kingdom
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Penn Blood Disorder Center
Philadelphia, Pennsylvania, 19104, United States
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Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts
Aurora, Colorado, 80045, 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, 4029, Australia
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Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050, Australia
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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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Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics
Malmo, 205 02, Sweden
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St. Michael's Hospital
Toronto, Ontario, M5B 1W8, Canada
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St. Vincent Hospital & Health Care Center, Inc.
Indianapolis, Indiana, 46260, 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 Regents of the University of California, San Francisco campus
San Francisco, California, 94158, United States
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The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal
Philadelphia, Pennsylvania, 19104, United States
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UCSF IDS Pharmacy
San Francisco, California, 94143, United States
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University of California, San Francisco - Clinical Research Center
San Francisco, California, 94143, United States
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University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, 94143, United States
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Universitätsklinikum Bonn
Bonn, 53127, Germany
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Universitätsklinikum Bonn Institut für Klinische Chemie und Klinische Pharmakologie -Phase 1-Einheit
Bonn, 53127, Germany
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Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts
Bonn, 53127, Germany
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Vivantes Klinikum Friedrichshain
Berlin, 10249, Germany
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Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
Conditions
Explore the condition pages connected to this study.