Treatment of Hemophilia A Patients With FVIII Inhibitors
Research type
Research Study
Full title
MOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – An International Low-Interventional Pragmatic Investigator Initiated Trial
IRAS ID
272829
Contact name
Mary Mathias
Contact email
Sponsor organisation
HZRM – Hämophilie-Zentrum Rhein Main GmbH
Eudract number
2019-003427-38
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
2019-003427-38, EudarCT number
Duration of Study in the UK
8 years, 10 months, 0 days
Research summary
Inhibitor development is the most serious treatment-related complication of replacement coagulation factor VIII (FVIII) therapy for patients with haemophilia A. Immune tolerance induction (ITI), which involves intensive treatment with plasma-derived (pdFVIII) or recombinant FVIII (rFVIII), is the only clinically proven strategy for eradication of inhibitors. The bispecific antibody emicizumab is approved for use in patients with and without inhibitors to prevent bleeding but does not eliminate inhibitors. MOTIVATE aims to capture different approaches to the treatment and management of patients with haemophilia A and inhibitors, document current ITI approaches and evaluate the efficacy and safety of ITI, including ITI with emicizumab prophylaxis. MOTIVATE will include multiple sub-studies to assess long-term impact of treatments on joint and bone health and the risk for thrombosis.
The investigator-initiated MOTIVATE trial – to be initiated in Europe and in North America – will evaluate the management of inhibitors in patients with haemophilia A of any severity who have developed inhibitors to FVIII. All treatment is at the investigator’s discretion. The following treatment approaches will be evaluated: Group 1 – ITI with Nuwiq®, octanate® or wilate® and aPCC/rFVIIa if needed to treat bleeding episodes (BEs) or during surgery or for prophylaxis; Group 2 – ITI with Nuwiq®, octanate® or wilate® and emicizumab prophylaxis and aPCC/rFVIIa if needed to treat BEs or during surgery; Group 3 – routine prophylaxis with emicizumab without ITI and aPCC/rFVIIa if needed to treat BEs or during surgery. Participants are not randomised to any treatment group and may change groups during the study. It is planned to enrol 120 patients who will be followed for up to 5 years.REC name
North of Scotland Research Ethics Committee 2
REC reference
20/NS/0048
Date of REC Opinion
31 Mar 2020
REC opinion
Favourable Opinion