MO39129: Prophylactic Emicizumab in Haemophilia A patients
Research type
Research Study
Full title
A SINGLE-ARM, MULTICENTER PHASE IIIB CLINICAL TRIAL TO EVALUATE THE SAFETY AND TOLERABILITY OF PROPHYLACTIC EMICIZUMAB IN HEMOPHILIA A PATIENTS WITH INHIBITORS
IRAS ID
225586
Contact name
Michael Makris
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2016-004366-25
Duration of Study in the UK
3 years, 0 months, 23 days
Research summary
Haemophilia A is a bleeding disorder resulting from inherited deficiency/absence of blood coagulation factor VIII (FVIII). These patients have a lifelong bleeding tendency manifesting as easy bruising; prolonged bleeding; spontaneous bleeding typically into joints and intracranial haemorrhage. Haemophilia is classified into mild, moderate, and severe based on FVIII activity, and approximately half of patients have severe (<1% normal FVIII activity) form leading to frequent bleeding events with complications such as chronic joint disease, neurocognitive defects, or even death. These disease-related issues have a significant impact on the quality of life of adolescents and adults with haemophilia.
Routine FVIII replacement to prevent bleeds (prophylaxis) markedly reduces bleeding events and complications, and in children it is highly desired to start prophylaxis early in life. Although effective, this treatment is associated with a significant burden of infusion factor 3 times or more weekly (typically in the morning), which in itself impacts on quality of life.
Emicizumab is an engineered antibody that mimics the function of FVIII. It is administered subcutaneously (SC), removing the need for venous access, and the dosing interval is extended to once weekly. This novel compound has the potential to dramatically change the treatment of haemophilia A patients with or without FVIII inhibitors who are in need of safe and effective prophylactic therapy that is not associated with significant burden.
A total of 200 participants will take part in this study globally, designed to evaluate the safety and tolerability of prophylactic emicizumab in patients with haemophilia A with inhibitors.
Safety (including standard blood tests), bleed assessments, quality of life questionnaires and treatment preference will be carried out during the study. In addition to this, more specific tests will determine study drug levels and biomarkers in the blood.
In the UK inclusion of 10 patients across 6 sites is anticipated.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
17/EE/0297
Date of REC Opinion
6 Sep 2017
REC opinion
Further Information Favourable Opinion