Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
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
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.
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.
Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment
Dosage of FVIII concentrates
Study Arms / Comparison Groups
Description: All patients receiving Nuwiq (recombinant FVIII)
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
February 13, 2018
Primary Completion Date
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
N/A - N/A
Accepts Healthy Volunteers
Sigurd Knaub, PhD, +41 554512141, [email protected]
Sigurd Knaub, PhD, Study Director, Octapharma