MOTIVATE (MOdern Treatment of Inhibitor-positiVe pATiEnts with haemophilia A) Begins Patient Recruitment

ATLANTA and MÖRFELDEN-WALLDORF, Germany, June 17, 2020 /PRNewswire/ -- Dr Carmen Escuriola-Ettingshausen and Dr Robert Sidonio are pleased to announce that MOTIVATE (MOdern Treatment of Inhibitor-positiVe pATiEnts with haemophilia A) has been approved in the United States and Europe and is now recruiting participants.

MOTIVATE is a multicentre, investigator-initiated study to capture different approaches to the management of patients with haemophilia A and inhibitors. MOTIVATE is registered as a non-interventional study in the US (NCT04023019 [https://clinicaltrials.gov/ct2/show/NCT04023019]) and as a low-interventional, pragmatic trial in Europe (EudraCT No. 2019-003427-38 [https://www.clinicaltrialsregister.eu/ctr-search/search?query=2019-003427-38]).

Inhibitor development is the most serious treatment-related complication of replacement factor VIII (FVIII) therapy for patients with haemophilia A as it renders FVIII ineffective at preventing and controlling bleeds. Immune tolerance induction (ITI) with prolonged replacement FVIII treatment is the only proven strategy for eradication of inhibitors. Although the bispecific monoclonal antibody emicizumab offers an alternative to bypassing agents for controlling bleeds in patients with inhibitors, neither emicizumab nor bypassing agents eradicate inhibitors.

The goal of MOTIVATE is to document established and current ITI approaches in order to evaluate the efficacy and safety of ITI, including with emicizumab prophylaxis. The study will also analyse the impact of different treatment approaches on prevention of bleeding in patients with inhibitors. Three patient groups will be evaluated based on their treatment approach:


-- ITI (with the human cell line-derived recombinant FVIII Nuwiq(®) or the
plasma-derived FVIII products octanate(®) or wilate(®))
-- ITI (with Nuwiq(®), octanate(®) or wilate(®)) and emicizumab
prophylaxis
-- Routine prophylaxis with emicizumab, aPCC or rFVIIa without ITI
All three approaches may include bypassing agents (aPCC or rFVIIa) if needed to treat bleeding episodes or during surgery. Patients will be followed for up to five years and may switch to another group if their treatment is changed at the discretion of the local physician.

MOTIVATE includes several optional sub-studies, including analysis of bone and joint health biomarkers, thrombotic risk assessment and F8 gene variant analysis, to better understand factors associated with treatment outcomes and the impact of different treatment regimens on patient outcomes.

MOTIVATE is led by two coordinating investigators, Dr Carmen Escuriola-Ettingshausen, Director of the Hämophilie-Zentrum Rhein Main in Mörfelden-Walldorf, Germany, and Dr Robert Sidonio Jr, Assistant Professor of Pediatrics and Director of Clinical Operations at the Hemophilia of Georgia Center for Bleeding and Clotting Disorders, Children's Healthcare of Atlanta, Emory University, Atlanta, Georgia, US.

Dr Sidonio commented that "It is very exciting that patient recruitment for MOTIVATE has begun. We expect MOTIVATE to provide valuable insights from real-world experience of haemophilia A patients who develop inhibitors to FVIII". Dr Escuriola-Ettingshausen added "We hope that data from MOTIVATE will inform treatment decisions to help us to optimise patient management. The sub-studies also promise to greatly increase our understanding of inhibitors and how we can impact patient outcomes".

MOTIVATE aims to enrol a total of 120 patients of any age and with haemophilia A of any severity who have developed inhibitors to any FVIII product. MOTIVATE is supported by funding from Octapharma AG (Lachen, Switzerland).

For further information on MOTIVATE and study participation, please visit www.motivate-study.com [http://www.motivate-study.com/].

Contact: Dr Robert Sidonio (US and Canada) robert.sidonio.jr@emory.edu [mailto:robert.sidonio.jr@emory.edu] and Dr Carmen Escuriola-Ettingshausen (Europe and rest of world) carmen.escuriola@hzrm.de [mailto:carmen.escuriola@hzrm.de]

CONTACT: Dr Robert Sidonio: +1 404 785 1637. Dr Carmen Escuriola-Ettingshausen, +49 6105 96 389 09

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