Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
- Conditions
- Haemophilia A
- Registration Number
- NCT03695978
- Lead Sponsor
- Octapharma
- 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.
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 200
- 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
- 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 concentrate AND continue treatment with THIS Octapharma FVIII concentrate (in the presence or absence of emicizumab).
- 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)
- 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
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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
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
Name Time Method Dosage of FVIII concentrates 100 exposure days For each individual FVIII injection the dose will be recorded.
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'.
Trial Locations
- Locations (45)
Hospital Infantil de Morelia Eva Sámano de López Mateos
🇲🇽Morelia, Mexico
Hospital Universitario Dr. José Eleuterio Gonzalez S/N
🇲🇽Nuevo León, Mexico
SMO and Scientific Services S.A.P.P de C.V
🇲🇽Nuevo León, Mexico
Cure 4 The Kids Foundation Children's Specialty Center
🇺🇸Las Vegas, Nevada, United States
Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia
🇦🇿Baku, Azerbaijan
Republican Scientific Center for Radiation Medicine and Human Ecology
🇧🇾Gomel, Belarus
Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
🇧🇾Minsk, Belarus
Hôpital Universitaire des Enfants Reine Fabiola
🇧🇪Brussels, Belgium
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium
McMaster University, Division of Pediatric Hematology/Oncology Room 3N27
🇨🇦Hamilton, Ontario, Canada
Department of Hematology Research Research Transition Facility
🇨🇦Edmonton, Canada
University Hospital Centre Zagreb
🇭🇷Croatia, Croatia
Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology
🇪🇪Tallinn, Estonia
CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON
🇫🇷Besancon, France
Centre Régional de Traitement de l'hémophilie
🇫🇷Le Mans, France
Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency
🇫🇷Montmorency, France
CHU Hotel Dieu, Centre de Traitment de l'Hemophilie
🇫🇷Nantes, France
Hôspital Necker Enfants Malades
🇫🇷Paris, France
CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3
🇫🇷Saint-Priest-en-Jarez, France
Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain
🇩🇪Berlin, Germany
Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)
🇩🇪Bonn, Germany
Coagulation Research Centre GmbH
🇩🇪Duisburg, Germany
Heim Pál National Pediatric Institute Department of Oncology and Hematology
🇭🇺Budapest, Hungary
University of Debrecen Department of Pediatrics
🇭🇺Debrecen, Hungary
Ospedale Pediatrico "Giovani XXIII"
🇮🇹Bari, Italy
Policlinico Sant'Orsola Malpighi
🇮🇹Bologna, Italy
Ospedale San Giacomo
🇮🇹Castelfranco Veneto, Italy
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, Italy
Azienda Ospedaliero Universitaria Careggi
🇮🇹Florence, Italy
Ospedale Maggiore Policlinico
🇮🇹Milan, Italy
Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital
🇮🇹Milan, Italy
Centro Emofilia - AUO di Padova
🇮🇹Padova, Italy
Policlinico Umberto I
🇮🇹Rome, Italy
C.tro Emofilia A.O. Città della salute e della scienza di Torino, Ospedale Regina Margherita
🇮🇹Torino, Italy
Ospedale Regina Margherita
🇮🇹Turin, Italy
Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos
🇱🇹Vilnius, Lithuania
Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"
🇷🇺Moscow, Russian Federation
Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"
🇷🇺Saint Petersburg, Russian Federation
Hospital General Universitario de Alicante Hematología y Hemoterapia
🇪🇸Alicante, Spain
Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia
🇪🇸Barcelona, Spain
Istanbul University Faculty of Medicine
🇹🇷Fatih, Turkey
John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust
🇬🇧Headington, Oxford, United Kingdom
Birmingham Children's Hospital NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre
🇬🇧London, United Kingdom
Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary
🇬🇧Newcastle Upon Tyne, United Kingdom