Clincosm LogoSign in Get a demo

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

Sponsor
Octapharma (Industry)
Overall Status
Recruiting
CT.gov ID
NCT03695978
Collaborator
(none)
140
Enrollment
34
Locations
147.5
Anticipated Duration (Months)
4.1
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase

    Detailed 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.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    140 participants
    Observational Model:
    Cohort
    Time Perspective:
    Other
    Official Title:
    Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated & Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment With Nuwiq, Octanate or Wilate - Efficacy & Safety Observational Study-Protect-NOW
    Actual Study Start Date :
    Feb 13, 2018
    Anticipated Primary Completion Date :
    Jun 1, 2030
    Anticipated Study Completion Date :
    Jun 1, 2030

    Arms and Interventions

    Arm Intervention/Treatment
    Nuwiq

    All patients receiving Nuwiq (recombinant FVIII)
    Octanate

    All patients receiving Octanate (plasma derived FVIII)
    Wilate

    All patients receiving Wilate (plasma derived FVIII/von Willebrand factor [VWF])

    Outcome Measures

    Primary Outcome Measures

    1. Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment [100 exposure days]

      Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs

    2. Incidence of Adverse Drug Reactions (ADRs) [100 exposure days]

      Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.

    Secondary Outcome Measures

    1. Dosage of FVIII concentrates [100 exposure days]

      For each individual FVIII injection the dose will be recorded.

    2. Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians [100 exposure days]

      At the end of the postoperative period, treating physicians will assess the effectiveness of surgical prophylaxis using a scale including the four items: 'excellent,' 'good,' moderate,' and 'none'.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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 the study

    • Either

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

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

    • their first ED occurred after 1st January 2015, AND

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

    • they developed an inhibitor during treatment with an Octapharma FVIII concentrate AND continue treatment with THIS Octapharma FVIII concentrate

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

    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Cure 4 The Kids Foundation Children's Specialty Center Las Vegas Nevada United States 89109
    2 Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia Baku Azerbaijan AZ1012
    3 Republican Scientific Center for Radiation Medicine and Human Ecology Gomel Belarus 246040
    4 Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology Minsk Belarus 223053
    5 Hôpital Universitaire des Enfants Reine Fabiola Brussels Belgium 1020
    6 Cliniques Universitaires Saint-Luc Brussels Belgium 1200
    7 McMaster University, Division of Pediatric Hematology/Oncology Room 3N27 Hamilton Ontario Canada 1800NOCLOTS
    8 Department of Hematology Research Research Transition Facility Edmonton Canada T6G 2V2
    9 Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology Tallinn Estonia 13419
    10 Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency Montmorency France 95160
    11 CHU Hotel Dieu, Centre de Traitment de l'Hemophilie Nantes France 44093
    12 CHRU Hopital Nord, Secretariat de pediatre Saint-Priest-en-Jarez France 42270
    13 Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain Berlin Germany 10249
    14 Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR) Bonn Germany 53127
    15 Coagulation Research Centre GmbH Duisburg Germany 47051
    16 Heim Pál National Pediatric Institute Department of Oncology and Hematology Budapest Hungary 1089
    17 University of Debrecen Department of Pediatrics Debrecen Hungary 4032
    18 Policlinico Sant'Orsola Malpighi Bologna Italy 40138
    19 Ospedale San Giacomo Castelfranco Veneto Italy 31033
    20 Azienda Ospedaliero Universitaria Careggi Florence Italy 50134
    21 Ospedale Maggiore Policlinico Milan Italy 920122
    22 Centro Emofilia - AUO di Padova Padova Italy
    23 Policlinico Umberto I Rome Italy 00161
    24 Ospedale Regina Margherita Turin Italy 10126
    25 Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos Vilnius Lithuania LT-08406
    26 Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department" Moscow Russian Federation 119049
    27 Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37" Saint Petersburg Russian Federation 191186
    28 Hospital General Universitario de Alicante Hematología y Hemoterapia Alicante Spain 03010
    29 Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia Barcelona Spain 02035
    30 Istanbul University Faculty of Medicine Fatih Turkey
    31 John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust Headington Oxford United Kingdom OX3 9DU
    32 Birmingham Children's Hospital NHS Foundation Trust Birmingham United Kingdom B46NH
    33 Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre London United Kingdom WC1N 3JH
    34 Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary Newcastle Upon Tyne United Kingdom NE1 4LP

    Sponsors and Collaborators

    • Octapharma

    Investigators

    • Study Director: Sigurd Knaub, PhD, Octapharma

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Octapharma
    ClinicalTrials.gov Identifier:
    NCT03695978
    Other Study ID Numbers:
    • GENA-25
    First Posted:
    Oct 4, 2018
    Last Update Posted:
    Jul 8, 2022
    Last Verified:
    Jul 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Hemophilia A

    Study Results

    No Results Posted as of Jul 8, 2022