ATLANTA, May 28, 2021 /PRNewswire/ -- Dr Robert Sidonio and Dr Carmen Escuriola-Ettingshausen have announced that MOTIVATE (MOdern Treatment of Inhibitor-positiVe pATiEnts with haemophilia A) has commenced patient enrollment in the US.
MOTIVATE is a multicenter, investigator-initiated study aiming to capture different approaches used for the management of patients with hemophilia A and inhibitors. It is registered as a non-interventional study in the US (NCT04023019) and as a low-interventional, pragmatic trial in Europe (EudraCT No. 2019-003427-38).
Dr Sidonio, Coordinating Investigator of the study for the US and Canada, commented that "We are very excited that patient enrollment in MOTIVATE has begun in the US. The data being collected will be invaluable in progressing our understanding of the efficacy and safety of different therapeutic regimens, bringing us closer to our goal of improving the standard of care for hemophilia A patients with inhibitors".
Development of inhibitors remains the biggest challenge associated with factor VIII (FVIII) replacement therapy in the treatment of hemophilia A, rendering FVIII ineffective for bleeding control. Availability of non-factor replacement therapies, such as the bispecific monoclonal antibody emicizumab, offers an alternative to bypassing agents for bleed protection. However, neither emicizumab nor bypassing agents eradicate inhibitors and immune tolerance eradication (ITI) remains the only proven method of inhibitor eradication.
MOTIVATE provides an exciting opportunity to collect real-world clinical data on modern approaches to treating patients with hemophilia A and inhibitors, and to compare the efficacy and safety of different treatment approaches. The study is investigating ITI with and without emicizumab prophylaxis.
Six optional sub-studies are being conducted within MOTIVATE, including analysis of bone and joint health, thrombotic risk assessment and F8 gene variant analysis. These sub-studies will provide valuable insight into factors associated with treatment outcomes and how different therapeutic regimens might impact patient outcomes.
Dr Escuriola-Ettingshausen, Coordinating Investigator for the rest of the world, explained "Real-world data from MOTIVATE and its sub-studies will help us greatly to understand how to optimize treatment for hemophilia A patients with inhibitors to improve patient outcomes".
For further information on MOTIVATE and study participation, please visit www.motivate-study.com.
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.
MOTIVATE will include the following three treatment groups:
- ITI alone (with the human cell line-derived recombinant FVIII Nuwiq® or the plasma-derived FVIII products octanate® or wilate®)
- ITI (with Nuwiq®, octanate® or wilate®) combined with emicizumab prophylaxis
- Non-ITI regimens (routine prophylaxis with emicizumab, activated prothrombin complex concentrate [aPCC] or rFVIIa)
Patients from all groups are permitted to receive bypassing agents (aPCC or rFVIIa) if required for treatment of bleeding episodes or during surgery. Patients will be followed for up to five years and may switch to another treatment group if their treatment is changed by their physician. MOTIVATE aims to enroll 120 patients of any age and with hemophilia of any severity who have developed inhibitors to any FVIII product.
MOTIVATE is supported by funding from Octapharma AG (Lachen, Switzerland). Full logistical support from a contract research organisation (CRO) is available to all centers.
SOURCE The MOTIVATE Study