Sobi.Elocta-002 _ A-SURE
Research type
Research Study
Full title
24-month prospective, multicentre, non-interventional study to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A (A-SURE)
IRAS ID
213250
Contact name
Charles HAY
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AB
Clinicaltrials.gov Identifier
16/CBS/6830, Proportionate review reference
Duration of Study in the UK
4 years, 0 months, 0 days
Research summary
This is a prospective, non-interventional, multicentre study designed to evaluate the effectiveness of prophylactic treatment of patients with haemophilia A with Elocta compared to conventional factor products. Retrospective data will also be collected.
Haemophilia A is a rare genetic disorder estimated to occur in one in 10,000 live births characterised by a deficiency in coagulation Factor VIII causing impaired haemostasis and prolonged bleeding episodes. Severe haemophilia A occurs in approximately 45% of haemophilia A patients, and results in frequent and spontaneous bleeds into muscles and joints, commonly the elbows, knees, and ankles. Bleeding into joints can cause acute pain and swelling and can result in reduced joint range of motion, long-term cartilage damage and debilitating haemophilic arthropathy. As a result of bleeding episodes and consequent progressive joint damage, haemophilia A patients experience decreased physical functioning, pain, and poor health-related quality of life.
All eligible patients who are prescribed prophylactic factor treatment according to usual clinical practice will be enrolled at research sites. The study plans to enroll 350 patients with a ratio of 1:1 of patients treated with Elocta versus conventional factor products. Patients will be prescribed Elocta or a conventional factor product, according to usual clinical practice and the dosage guide in the respective SmPC and choice of treatment will not be dictated by the study protocol. Thus, switching between factor products during the 24-month prospective study period is also allowed.REC name
London - London Bridge Research Ethics Committee
REC reference
16/LO/1863
Date of REC Opinion
15 Nov 2016
REC opinion
Further Information Favourable Opinion