Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
Sponsor:
Octapharma
Collaborators:
Information provided by (Responsible Party):
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| Tracking Information | |||
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| First Submitted Date ICMJE | October 3, 2018 | ||
| First Posted Date ICMJE | October 4, 2018 | ||
| Last Update Posted Date | October 4, 2018 | ||
| Actual Study Start Date ICMJE | February 13, 2018 | ||
| Estimated Primary Completion Date | June 2022 (Final data collection date for primary outcome measure) | ||
| Current Primary Outcome Measures ICMJE |
Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment[ Time Frame: 100 exposure days ] Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs Incidence of Adverse Drug Reactions (ADRs)[ Time Frame: 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. |
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| Original Primary Outcome Measures ICMJE | Same as current | ||
| Current Secondary Outcome Measures ICMJE |
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| Descriptive Information | |||
| Brief Title ICMJE | Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients |
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| Official Title ICMJE | 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 |
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| 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. |
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| 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 Type ICMJE | Observational | ||
| Study Phase | |||
| Study Design ICMJE | Allocation: Intervention Model: Intervention Model Description: Masking: Observational Masking Description: Primary Purpose: |
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| Condition ICMJE | |||
| Intervention ICMJE |
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| Study Arms |
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| Recruitment Information | |||
| Recruitment Status ICMJE | Recruiting | ||
| Estimated Enrollment ICMJE |
140 | ||
| Original Estimated Enrollment ICMJE | Same as current | ||
| Estimated Study Completion Date | June 2022 | ||
| Estimated Primary Completion Date | June 2022 (Final data collection date for primary outcome measure) | ||
| Eligibility Criteria ICMJE | 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 | ||
| Sex/Gender |
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| Ages | N/A and older (Adult, Older Adult) | ||
| Accepts Healthy Volunteers | No | ||
| Listed Location Countries ICMJE | Canada|Germany|Lithuania|Russian Federation|Spain|United Kingdom|United States | ||
| Removed Location Countries | |||
| Administrative Information | Has Data Monitoring Committee | No | |
| U.S. FDA-regulated Product |
Studies a U.S. FDA-regulated Drug Product: No Studies a U.S. FDA-regulated Device Product: No |
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| IPD Sharing Statement |
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| Responsible Party | ， | ||
| Study Sponsor ICMJE | Octapharma | ||
| Collaborators ICMJE | |||
| Investigators ICMJE |
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| PRS Account | |||
| Verification Date | October 2018 | ||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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