BH29992 - Subcutaneous emicizumab in Haemophilia A paediatric patients
Research type
Research Study
Full title
A single arm, multicentre, open label, phase III clinical trial to evaluate the efficacy, safety and pharmacokinetics of once weekly subcutaneous administration of emicizumab in haemophilia A paediatric patients with inhibitors
IRAS ID
201555
Contact name
Raina Liesner
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2016-000073-21
Duration of Study in the UK
2 years, 6 months, 15 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 presenting 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 children 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 and impact on quality of life.
Approximately 30% of patients develop antibodies (inhibitors) against FVIII which neutralise the activity of endogenous as well as replacement therapy FVIII. Consequently, patients with inhibitors are treated with alternative less effective bypassing agents, and have a higher rate of bleeds, complications, and treatment burden. Inhibitors often develop in childhood (typically within 50 exposures to FVIII), and children with inhibitors have a very high unmet medical need.
Emicizumab is an engineered antibody that mimics the function of FVIII. It is not neutralized by FVIII inhibitors and early findings suggest promising safety and effectiveness in such patients.
Given the tremendous unmet medical need among children with haemophilia A with FVIII inhibitors and positive benefit-risk assessment for emicizumab, initiation of a paediatric Phase III study is appropriate.
The objectives for this study are to investigate the efficacy, safety and pharmacokinetics of prophylactic emicizumab in paediatric patients with haemophilia A with inhibitors.
In the UK inclusion of 7 patients in 1 site is anticipated.REC name
London - City & East Research Ethics Committee
REC reference
16/LO/0735
Date of REC Opinion
25 Jun 2016
REC opinion
Further Information Favourable Opinion