Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients

Last updated: April 16, 2025
Sponsor: Octapharma
Overall Status: Active - Recruiting

Phase

N/A

Condition

Hemophilia

Treatment

N/A

Clinical Study ID

NCT03695978
GENA-25
  • All Genders

Study Summary

International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male and female patients of any age and ethnicity

  • Severe haemophilia A (FVIII:C<1%)

  • Decision to prescribe Octapharma's FVIII concentrate before enrollment into thestudy

  • Either

  • No previous treatment with FVIII concentrates or other blood products containingFVIII (PUPs) OR

  • Less than 5 Exposure Days (EDs) to FVIII concentrates or other blood productscontaining FVIII (MTPs), if

  • data are available on all previous treatment, AND

  • they did not develop an inhibitor at any time point, OR

  • they developed an inhibitor during treatment with an Octapharma FVIII concentrateAND continue treatment with THIS Octapharma FVIII concentrate (in the presence orabsence of emicizumab).

  • Voluntarily given, fully informed written and signed consent obtained before anystudy-related data documentation is conducted (obtained from the patient'sparent/legal guardian)

Exclusion

Exclusion Criteria:

  • Diagnosis with a coagulation disorder other than haemophilia A

  • Concomitant treatment with any systemic immunosuppressive drug

  • Participation in an interventional clinical trial during the time period evaluated

  • Participation in another non-interventional study of Octapharma

Study Design

Total Participants: 200
Study Start date:
February 13, 2018
Estimated Completion Date:
June 30, 2030

Study Description

Octapharma's FVIII concentrates have been tested in clinical trials and registered for treatment of haemophilia A; however, as haemophilia A is a rare disease, the numbers of patients treated in studies so far are limited. For previously untreated patients (PUPs), who are typically young children, and for minimally treated patients (MTPs), who have been exposed to only minimal FVIII dosages, there is a general interest to increase the body of data on treatment effectiveness and safety, particularly related to inhibitor development. Also, specifically for PUPs, treatment algorithms are not standardized, e.g. with respect to utilisation, dosage, frequency or optimal start age of FVIII prophylaxis. Real world evidence derived from a non-interventional study (NIS) can describe product utilisation and demonstrate value over a product's life cycle and facilitate benefit-risk assessments. The purpose of this study is thus to evaluate product utilisation, effectiveness and safety, including inhibitor development information, in severe haemophilia A PUPs and MTPs, who have been prescribed Octapharma's FVIII concentrates.

Connect with a study center

  • Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia

    Baku, AZ1012
    Azerbaijan

    Site Not Available

  • Republican Scientific Center for Radiation Medicine and Human Ecology

    Gomel, 246040
    Belarus

    Site Not Available

  • Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology

    Minsk, 223053
    Belarus

    Active - Recruiting

  • Cliniques Universitaires Saint-Luc

    Brussels, 1200
    Belgium

    Active - Recruiting

  • Hôpital Universitaire des Enfants Reine Fabiola

    Brussels, 1020
    Belgium

    Active - Recruiting

  • McMaster University, Division of Pediatric Hematology/Oncology Room 3N27

    Hamilton, Ontario 1800NOCLOTS
    Canada

    Site Not Available

  • Department of Hematology Research Research Transition Facility

    Edmonton, T6G 2V2
    Canada

    Completed

  • University Hospital Centre Zagreb

    Croatia,
    Croatia

    Active - Recruiting

  • Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology

    Tallinn, 13419
    Estonia

    Site Not Available

  • CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON

    Besancon,
    France

    Active - Recruiting

  • Centre Régional de Traitement de l'hémophilie

    Le Mans,
    France

    Active - Recruiting

  • Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency

    Montmorency, 95160
    France

    Active - Recruiting

  • CHU Hotel Dieu, Centre de Traitment de l'Hemophilie

    Nantes, 44093
    France

    Active - Recruiting

  • Hôspital Necker Enfants Malades

    Paris,
    France

    Active - Recruiting

  • CHRU Hopital Nord, Secretariat de pediatre

    Saint-Priest-en-Jarez, 42270
    France

    Site Not Available

  • CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3

    Saint-Priest-en-Jarez, 42270
    France

    Active - Recruiting

  • Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain

    Berlin, 10249
    Germany

    Active - Recruiting

  • Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)

    Bonn, 53127
    Germany

    Active - Recruiting

  • Coagulation Research Centre GmbH

    Duisburg, 47051
    Germany

    Active - Recruiting

  • Heim Pál National Pediatric Institute Department of Oncology and Hematology

    Budapest, 1089
    Hungary

    Active - Recruiting

  • University of Debrecen Department of Pediatrics

    Debrecen, 4032
    Hungary

    Active - Recruiting

  • Ospedale Pediatrico "Giovani XXIII"

    Bari,
    Italy

    Active - Recruiting

  • Policlinico Sant'Orsola Malpighi

    Bologna, 40138
    Italy

    Active - Recruiting

  • Ospedale San Giacomo

    Castelfranco Veneto, 31033
    Italy

    Completed

  • Azienda Ospedaliera-Universitaria Policlinico "Vittorio Emanuele", Centro di Riferimento Regionale per la Prevenzione, Diagnosi e Cura delle Malattie Rare della Coagulazione nel Bambino e neel'Adulto. U.O.C Ematologia con trapianto di Midollo Osseo

    Catania, 95123
    Italy

    Active - Recruiting

  • Azienda Ospedaliero Universitaria Careggi

    Florence, 50134
    Italy

    Active - Recruiting

  • Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital

    Milan,
    Italy

    Active - Recruiting

  • Ospedale Maggiore Policlinico

    Milan, 920122
    Italy

    Active - Recruiting

  • Centro Emofilia - AUO di Padova

    Padova,
    Italy

    Site Not Available

  • Policlinico Umberto I

    Rome, 00161
    Italy

    Active - Recruiting

  • C.tro Emofilia A.O. Città della salute e della scienza di Torino, Ospedale Regina Margherita

    Torino,
    Italy

    Active - Recruiting

  • Ospedale Regina Margherita

    Turin, 10126
    Italy

    Site Not Available

  • Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos

    Vilnius, LT-08406
    Lithuania

    Active - Recruiting

  • Hospital Infantil de Morelia Eva Sámano de López Mateos

    Morelia,
    Mexico

    Active - Recruiting

  • Hospital Universitario Dr. José Eleuterio Gonzalez S/N

    Nuevo León,
    Mexico

    Active - Recruiting

  • SMO and Scientific Services S.A.P.P de C.V

    Nuevo León,
    Mexico

    Active - Recruiting

  • Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"

    Moscow, 119049
    Russian Federation

    Site Not Available

  • Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"

    Saint Petersburg, 191186
    Russian Federation

    Site Not Available

  • Hospital General Universitario de Alicante Hematología y Hemoterapia

    Alicante, 03010
    Spain

    Active - Recruiting

  • Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia

    Barcelona, 02035
    Spain

    Active - Recruiting

  • Istanbul University Faculty of Medicine

    Fatih,
    Turkey

    Site Not Available

  • John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust

    Headington, Oxford OX3 9DU
    United Kingdom

    Active - Recruiting

  • Birmingham Children's Hospital NHS Foundation Trust

    Birmingham, B46NH
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre

    London, WC1N 3JH
    United Kingdom

    Active - Recruiting

  • Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary

    Newcastle Upon Tyne, NE1 4LP
    United Kingdom

    Active - Recruiting

  • Cure 4 The Kids Foundation Children's Specialty Center

    Las Vegas, Nevada 89109
    United States

    Site Not Available

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