BH29884 - Prophylactic RO5534262 vs no prophylaxis in Haemophilia A.
Research type
Research Study
Full title
A RANDOMISED, MULTICENTER, OPEN-LABEL, PHASE III CLINICAL TRIAL TO EVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF PROPHYLACTIC RO5534262 VERSUS NO PROPHYLAXIS IN HAEMOPHILIA A PATIENTS WITH INHIBITORS
IRAS ID
192702
Contact name
Gerard Dolan
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2015-002866-21
Duration of Study in the UK
2 years, 1 months, 0 days
Research summary
Haemophilia A is an X-linked recessive bleeding disorder that occurs in approximately 1/5000 live male births. These patients have a deficiency/absence of blood coagulation factor VIII(FVIII) which leads to a lifelong bleeding tendency. Signs include easy bruising; prolonged bleeding; spontaneous bleeding and intracranial haemorrhage.
Approximately 68% haemophilia A patients have moderate(25%) or severe(43%) forms leading to frequent bleeding events with complications such as arthropathy, haemorrhagic shock, neurocognitive defects, or even death. These disease-related issues can have a significant impact on the health-related quality of life(HRQoL) of adult and adolescent patients.
Prophylactic FVIII replacement therapy has been proven to minimise bleeding events and complications. Prophylactic regimens use infusion therapy 3X or more weekly (including frequent morning infusions) and compliance with these demanding regimens to achieve adequate haemostatic coverage during periods of highest activity makes them less effective and compromises their cost-benefit ratio.
Development of inhibitory alloantibodies(inhibitors) occurs in approximately 20%−30% of patients with severe disease and 3%−13% of those with moderate-mild disease. Inhibitors neutralise the activity of endogenous as well as replacement therapy FVIII.
RO5534262 is a recombinant, humanised, immunoglobulinG4 monoclonal-antibody that binds to activated factor IX(FIXa) and factor X(FX), mimicking the co-factor function of FVIII.
RO5534262 offers the possibility of subcutaneous-administration(SC), removing the need for venous access. Also because of the expected pharmacokinetic properties of this antibody, extending the dosing interval to once weekly or even less frequently, this novel compound has potential to dramatically change the treatment of haemophilia A patients with/without FVIII inhibitors who are in need of effective, safe and convenient prophylactic therapy.
The primary efficacy objective for this study is to evaluate the efficacy of prophylactic RO5534262 compared with no prophylaxis in patients with haemophilia A with inhibitors.
In the UK it is anticipated there will be 8 patients across 4 sites.REC name
London - City & East Research Ethics Committee
REC reference
16/LO/0034
Date of REC Opinion
6 Feb 2016
REC opinion
Further Information Favourable Opinion