Trust patient is first participant in Europe to join study of new treatment for rare bleeding disorder
A patient at Imperial College Healthcare NHS Trust is the first to be recruited in Europe to a study looking at a new treatment for people who have a rare autoimmune bleeding condition known as immune thrombocytopenic purpura (ITP).
This is the first phase 3 study in Europe for mezatigamab, a new medicine for patients with ITP, known as a monoclonal antibody treatment that targets the CD38 protein. Phase 3 trials typically compare a new treatment to the standard treatment for a condition or to a placebo drug.
Supported by the NIHR Research Delivery Network, the new study welcomed its first participant in Europe at Hammersmith Hospital in July.
Patients with this rare disorder – immune thrombocytopenia (ITP) - have low levels of platelets in their blood. These are cells that help stop bleeding after an injury or damage in the body by clumping together to form a clot. This leads to bruising or bleeding in these patients because they don't have enough platelets in the blood. The condition is usually caused by the immune system mistakenly destroying platelets.
The aim of this study is to learn how effective the new treatment, mezagitamab, is at keeping the platelet count of adults with ITP stable when compared to a placebo drug.
Professor Nichola Cooper, consultant haematologist at Imperial College Healthcare, lead for the immune haematology service, and the principal investigator leading the study, said: “We are delighted to have recruited the first patient in Europe to this study. ITP has a significant effect on the quality of life of patients, increasing bleeding risk, and impacting work, schooling and social life. This promising new treatment targets a different immune pathway and is an exciting new development for these patients.”
Imperial College Healthcare is one of several sites across Europe taking part in the trial sponsored by Takeda Pharmaceuticals.
Donna Copeland, research manager at Imperial College Healthcare, said: “Being the first hospital in Europe to offer this trial to patients is a fantastic achievement, showcasing our strength in opening trials quickly. It would not have been possible without the dedication, enthusiasm, and expertise of our multidisciplinary team, made up of our doctors, nurses, practitioners, administrative staff and supporting services including pharmacy, imaging and labs.
“Opening first increases the number of our patients that get that opportunity to take part, and crucially, it gives our patients access to study treatment early, which is especially important in areas where options are limited. We are looking forward to progressing with the trial and recruiting more patients.”
Imperial College Healthcare has a longstanding partnership with Takeda through Paddington Life Sciences. Takeda, who has an office in Paddington, is one of the founding members of Paddington Life Sciences Partners. This partnership brings together the NHS, industry, community and academic organisations with a shared commitment to improving health outcome and reduce inequalities in north west London and beyond.
