New hope for young boys with rare bleeding disorder

NCT ID NCT02141074

Summary

This trial tested a new, long-acting version of a missing clotting protein (Factor IX) in young boys with severe hemophilia B who had little to no prior treatment. The goal was to see if regular injections could safely prevent bleeding episodes and effectively treat bleeds when they occurred. Researchers measured how well it worked and checked for any safety issues, especially the development of antibodies that could block the treatment.

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

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642, Greece

  • ,,Louis Ţurcanu'' Emergency Hospital for Children

    Timișoara, Timiș County, 300011, Romania

  • 1st Paediatric Department, Fundeni Clinical Institute

    Bucharest, 022328, Romania

  • Aghia Sophia Childrens' Hospital

    Athens, GR-11527, Greece

  • Ap-Hp-Hopital de Bicetre-1

    Le Kremlin-Bicêtre, 94275, France

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Beni Messous Hospital Issaad Hassani

    Algiers, 16000, Algeria

  • Birmingham Children's Hospital

    Birmingham, B4 6NH, United Kingdom

  • CHU Estaing

    Clermont-Ferrand, 63003, France

  • CHU Haute Pierre

    Strasbourg, 67098, France

  • Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

    Nantes, 44093, France

  • Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou

    Rennes, 35033, France

  • Centro Hospitalar Lisboa Norte-HSM

    Lisbon, 1649-035, Portugal

  • Centro Medico Nacional SXXI-Hospital de Pediatria, IMSS

    Mexico City, México, D.F., 06720, Mexico

  • Changhua Christian Hospital

    Changhua, 500, Taiwan

  • Changhua Christian Hospital_Hematology Dept.

    Changhua, 500, Taiwan

  • Children's Hosp-New Orleans

    New Orleans, Louisiana, 70118-5720, United States

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California, 90027, United States

  • Children's Hsptl Of The Kings

    Norfolk, Virginia, 23507, United States

  • China Medical University Children's Hospital

    Taichung, 40447, Taiwan

  • China Medical University Hospital - Children Building

    Taichung, 40447, Taiwan

  • Coagulation Research Center

    Duisburg, 47051, Germany

  • Dayton Children Hemostati Ctr

    Dayton, Ohio, 45404, United States

  • Dipartimento di Ematologia Univ. Firenze

    Florence, 50134, Italy

  • HZRM Haemophilie-Zentrum Rhein Main GmbH

    Frankfurt am Main, 60596, Germany

  • Hamltn Hth Sci/McMstr Child Hosp

    Hamilton, Ontario, L8N 3Z5, Canada

  • Hope for Kids

    Macon, Georgia, 31201, United States

  • Hospital La Fe - Endocrinología y Nutrición

    Valencia, 46026, Spain

  • Hospital Pulau Pinang_Georgetown, Penang

    George Town, Pulau Pinang, 10450, Malaysia

  • Hospital Sant Joan de Déu

    Esplugues Llobregat, 08950, Spain

  • Hospital Tengku Ampuan Afzan

    Kuantan, Pahang, 25100, Malaysia

  • Hospital Tengku Ampuan Rahimah

    Klang, Selangor, 41200, Malaysia

  • Hospital Universitario Dr. José Eleuterio González_Monterrey

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan

    CABA, C1245AAM, Argentina

  • IRCCS Meyer Firenze

    Florence, 50139, Italy

  • Institute for Mother and Child Health Care of Serbia

    Belgrade, 11070, Serbia

  • Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery

    Lviv, 79044, Ukraine

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • Johns Hopkins University_Baltimore_3

    Baltimore, Maryland, 21205, United States

  • Kanagawa Children's Medical Center _ Hematology / Oncology

    Kanagawa, 232-8555, Japan

  • Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics

    Kaohsiung City, 807, Taiwan

  • Kent and Canterbury Hospital - Kent Haemophilia and Thrombosis Centre

    Canterbury, CT1 3NG, United Kingdom

  • King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology

    Bangkok, 10330, Thailand

  • Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)

    Klagenfurt, 9020, Austria

  • LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde

    Salzburg, 5020, Austria

  • Lady Cilento Children's Hospital

    South Brisbane, Queensland, 4101, Australia

  • Landes-Frauen und Kinderklinik Linz

    Linz, 4020, Austria

  • Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

    Leicester, LE1 5WW, United Kingdom

  • Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology

    Chiang Mai, 50200, Thailand

  • Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie

    Graz, 8036, Austria

  • Medical University Of SC

    Charleston, South Carolina, 29425, United States

  • Miller Children's Hospital Long Beach

    Long Beach, California, 90806, United States

  • Nagoya University Hospital_Blood Transfusion

    Aichi, 466-8560, Japan

  • National Blood Centre

    Kuala Lumpur, 50400, Malaysia

  • National Center for Child Health and Development_Hematology

    Tokyo, 157-8535, Japan

  • National Taiwan University Children's Hospital

    Taipei, 100, Taiwan

  • Nemours Chld Clnc Jacksonville

    Jacksonville, Florida, 32207, United States

  • North Shore Long Island Jewish Medical Center

    New Hyde Park, New York, 11040, United States

  • Novant Hlth Vasc Ins Charlotte

    Charlotte, North Carolina, 28204, United States

  • Ogikubo Hospital_Pediatries & Blood

    Tokyo, 167-0035, Japan

  • Ordination Prof. Zwiauer

    Sankt Pölten, A 3100, Austria

  • Penn State MS Hershey Med Ctr

    Hershey, Pennsylvania, 17033, United States

  • Radboudumc

    Nijmegen, 6525 GA, Netherlands

  • Ramathibodi Hospital_Paediatrics

    Bangkok, 10400, Thailand

  • Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Royal Hospital for Children

    Glasgow, G51 4TF, United Kingdom

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • SI Institute of Urgent and Recovery Surgery - Haematology

    Donetsk, 83045, Ukraine

  • Saitama Children's Med Centre_Hematology-Oncology

    Saitama, 330-8777, Japan

  • Sanatorio Mayo Privado S.A

    Córdoba, X5000FAL, Argentina

  • Shands Hospital at the University of Florida

    Gainesville, Florida, 32610, United States

  • Sheba MC - The Israeli National Hemophilia Center

    Tel Litwinsky, 52621, Israel

  • Shizuoka Children's Hospital, Hematology-Oncology

    Shizuoka, 420-8660, Japan

  • St Christopher Hosp for Child

    Philadelphia, Pennsylvania, 19134, United States

  • St Thomas' Hospital - Haemostasis and Thrombosis Centre

    London, SE1 7EH, United Kingdom

  • St. Luke's Mountain States Tumor Institute

    Boise, Idaho, 83712, United States

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • ULS São João, E.P.E.

    Porto, 4200-319, Portugal

  • UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

    Plovdiv, 4002, Bulgaria

  • Univ Hosp Cleveland Med Ctr

    Cleveland, Ohio, 44106, United States

  • Univ of NE Med Center_Omaha

    Omaha, Nebraska, 68198-6828, United States

  • Univ of Utah Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • University Children's Hospital Tirsova

    Belgrade, 11000, Serbia

  • University Hospital Saadna Abdenour of Setif

    Sétif, 19000, Algeria

  • University Of Iowa

    Iowa City, Iowa, 52242, United States

  • University of North Carlolina-Chapel Hill

    Chapel Hill, North Carolina, 27514, United States

  • University of Virginia Children's Hospital

    Charlottesville, Virginia, 22903, United States

  • Universitätsklinik Kinder-Jugendheilkunde Innsbruck

    Innsbruck, 6020, Austria

  • Universitätsklinik für Kinder- und Jugendheilkunde

    Vienna, A 1090, Austria

  • Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

    Bonn, 53127, Germany

  • Vanderbilt Hemostasis Thrombosis Clinic

    Nashville, Tennessee, 37232, United States

  • Werlhof-Institut

    Hanover, 30159, Germany

Conditions

Explore the condition pages connected to this study.