For people with haemophilia A, factor VIII (FVIII) replacement therapy is used to prevent and treat bleeding. The most serious complication of FVIII replacement therapy is the development of inhibitors against FVIII.

Inhibitors prevent FVIII concentrate from controlling bleeding. As a result, people with inhibitors have increased risk of bleeding and joint problems, poorer quality of life and higher treatment costs.

Immune tolerance induction (ITI) is the only clinically proven strategy to eliminate inhibitors so that people can continue effective FVIII replacement therapy. ITI involves frequent administration of FVIII, often for many months.

The non-factor therapy emicizumab is now available for use in patients with and without inhibitors. Emicizumab provides effective bleed protection but does not eliminate inhibitors.

Given the increasing number of treatment options for people with inhibitors, there is a need to better understand the impact of different approaches and whether there are benefits of using FVIII ITI and emicizumab.

MOTIVATE is studying different approaches to treating people with inhibitors, including FVIII ITI with emicizumab prophylaxis, to help us to better understand how best to care for these patients.

Anyone with haemophilia A who has inhibitors to any FVIII product may participate in MOTIVATE, including patients who have previously failed ITI.

MOTIVATE seeks to answer the following questions on different treatment strategies for patients with inhibitors

Will treatment with FVIII ITI and emicizumab prophylaxis

  • Lead to improved rates of ITI success and a shorter time to ITI success?
  • Improve bleeding protection in patients with inhibitors?
  • Be safe and well tolerated?
  • Have an impact on the risk of venous thrombosis?

What is the role of FVIII dose on efficacy and safety of the treatments?

What is the impact of different treatment approaches on joint and bone health?