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 (42)
CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3
馃嚝馃嚪Saint-Priest-en-Jarez, France
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
馃嚠馃嚬Catania, Italy
Cure 4 The Kids Foundation Children's Specialty Center
馃嚭馃嚫Las Vegas, Nevada, United States
Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia
馃嚘馃嚳Baku, Azerbaijan
Republican Scientific Center for Radiation Medicine and Human Ecology
馃嚙馃嚲Gomel, Belarus
Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
馃嚙馃嚲Minsk, Belarus
H么pital Universitaire des Enfants Reine Fabiola
馃嚙馃嚜Brussels, Belgium
Cliniques Universitaires Saint-Luc
馃嚙馃嚜Brussels, Belgium
McMaster University, Division of Pediatric Hematology/Oncology Room 3N27
馃嚚馃嚘Hamilton, Ontario, Canada
Department of Hematology Research Research Transition Facility
馃嚚馃嚘Edmonton, Canada
Tallinn Children艣 Hospital Clinic of Paediatric Department of Haematology and Oncology
馃嚜馃嚜Tallinn, Estonia
Centre R茅gional de Traitement de l'h茅mophilie
馃嚝馃嚪Le Mans, France
Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency
馃嚝馃嚪Montmorency, France
CHU Hotel Dieu, Centre de Traitment de l'Hemophilie
馃嚝馃嚪Nantes, France
H么spital Necker Enfants Malades
馃嚝馃嚪Paris, France
Vivantes - Netzwerk f眉r Gesundheit GmbH Klinikum im Friedrichshain
馃嚛馃嚜Berlin, Germany
Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (A枚R)
馃嚛馃嚜Bonn, Germany
Coagulation Research Centre GmbH
馃嚛馃嚜Duisburg, Germany
Heim P谩l National Pediatric Institute Department of Oncology and Hematology
馃嚟馃嚭Budapest, Hungary
University of Debrecen Department of Pediatrics
馃嚟馃嚭Debrecen, Hungary
Ospedale Pediatrico "Giovani XXIII"
馃嚠馃嚬Bari, Italy
Policlinico Sant'Orsola Malpighi
馃嚠馃嚬Bologna, Italy
Ospedale San Giacomo
馃嚠馃嚬Castelfranco Veneto, Italy
Azienda Ospedaliero Universitaria Careggi
馃嚠馃嚬Florence, Italy
Ospedale Maggiore Policlinico
馃嚠馃嚬Milan, Italy
Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital
馃嚠馃嚬Milan, Italy
Centro Emofilia - AUO di Padova
馃嚠馃嚬Padova, Italy
Policlinico Umberto I
馃嚠馃嚬Rome, Italy
Ospedale Regina Margherita
馃嚠馃嚬Turin, Italy
Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos
馃嚤馃嚬Vilnius, Lithuania
Hospital Infantil de Morelia Eva S谩mano de L贸pez Mateos
馃嚥馃嚱Morelia, Mexico
Hospital Universitario Dr. Jos茅 Eleuterio Gonzalez S/N
馃嚥馃嚱Nuevo Le贸n, Mexico
SMO and Scientific Services S.A.P.P de C.V
馃嚥馃嚱Nuevo Le贸n, Mexico
Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"
馃嚪馃嚭Moscow, Russian Federation
Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"
馃嚪馃嚭Saint Petersburg, Russian Federation
Hospital General Universitario de Alicante Hematolog铆a y Hemoterapia
馃嚜馃嚫Alicante, Spain
Hospital Universitari Vall D'Hebr贸n, Unitat d'Hemofilia
馃嚜馃嚫Barcelona, Spain
Istanbul University Faculty of Medicine
馃嚬馃嚪Fatih, Turkey
John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust
馃嚞馃嚙Headington, Oxford, United Kingdom
Birmingham Children's Hospital NHS Foundation Trust
馃嚞馃嚙Birmingham, United Kingdom
Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre
馃嚞馃嚙London, United Kingdom
Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary
馃嚞馃嚙Newcastle Upon Tyne, United Kingdom