Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
- Registration Number
- NCT02976753
- Lead Sponsor
- Swedish Orphan Biovitrum
- Brief Summary
The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 361
Inclusion Criteria
- Male patients with a diagnosis of haemophilia A
- Received prophylactic treatment with a factor product for management of haemophilia A in the 12 months prior to enrolment
- At enrolment prescribed prophylactic treatment with Elocta or conventional factor product regardless of participation in the study
- Having at least 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and bleeding episodes prior to the baseline visit
- Signed and dated informed consent provided by the patient or the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
Exclusion Criteria
- Enrolment in a concurrent clinical interventional study involving intake of an investigational medicinal product (IMP), within 1 year prior to enrolment
- Previously treated with commercially available extended half-life products other than Elocta
- Presence of factor VIII inhibitors (≥0.60 Bethesda Unit [BU]/mL ) at the latest available inhibitor test using the Nijmegen modified Bethesda assay
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Conventional factor VIII product Factor VIII Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice Elocta efmoroctocog alfa Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice
- Primary Outcome Measures
Name Time Method Annualised factor consumption (International Unit [IU]) 24 months Annualised bleeding rate (ABR) 24 months Annualised injection frequency 24 months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (4)
Swedish Orphan Biovitrum Research Site
🇮🇹Padova, Italy
Swedish Orphan Biovitrum Reserach site
🇬🇧London, United Kingdom
Swedish Orphan Biovitrum research site
🇪🇸Alicante, Spain
Swedish Orphan Biovitrum Research site
🇬🇧Southampton, United Kingdom