Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products

Sobi

Status: Completed

Conditions: Hemophilia A

Treatments: Drug: Factor VIII; Drug: efmoroctocog alfa

Study type: Observational

Funder types: Industry

Identifiers

NCT02976753

Sobi.Elocta-002

Details and patient eligibility

About

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.

Enrollment: 361 patients

Sex: Male

Volunteers: No Healthy Volunteers

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

Trial design

361 participants in 2 patient groups

Elocta

Description:

Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice

Treatment:

Drug: efmoroctocog alfa

Conventional factor VIII product

Description:

Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice

Treatment:

Drug: Factor VIII