One-Time gene therapy could free hemophilia b patients from frequent infusions
NCT ID NCT03861273
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 27 times
Summary
This study tests a one-time gene therapy for adult men with moderate to severe hemophilia B, a bleeding disorder caused by a missing or faulty Factor IX gene. The therapy delivers a working copy of the gene to help the body produce its own clotting factor. Researchers will track how well it reduces bleeding episodes compared to standard preventive treatments over several years.
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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.
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
Adana, 01130, Turkey (Türkiye)
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ApoEx AB
Malmö, 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 & St Thomas' NHS Foundation Trust, St Thomas' Hospital
London, SE1 7EH, 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
Malmö, 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.