MAYARI
Research type
Research Study
Full title
An open-label, single-arm, multicenter study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy without first line therapeutic plasma exchange in adults with immune-mediated thrombotic thrombocytopenic purpura
IRAS ID
1005801
Contact name
Damoun Nourbakht
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Eudract number
2022-001177-31
Clinicaltrials.gov Identifier
Research summary
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a condition that causes very small blood clots to form in blood vessels. This can cause problems with most of the body’s major organs and can increase the chance of heart attacks and strokes. Caplacizumab is currently approved for the treatment of this condition, in combination with a procedure called Therapeutic Plasma Exchange (TPE) and Immunosuppressive Therapy (IST). TPE is a process that removes and replaces a patient's blood plasma to treat the disease and the IST helps prevent the immune system from fighting against itself, which plays a part in the blood clots forming.
TPE itself is a burdensome and invasive procedure for patients, and it is associated with significant complications and risks. Based on how iTTP arises and how the drug, caplacizumab, works, it is thought that caplacizumab and IST without initial TPE may be safe and effective as first-line treatment for iTTP. This idea is supported by both pre-clinical data as well as emerging, real-world clinical evidence, whereby patients have still been able to be treated for iTTP, but without TPE.
As a result, the sponsor is looking to investigate how safe and effective it is to treat patients with iTTP with just caplacizumab and IST alone. If required, TPE can still be given whilst on the study. The study will be ran in multiple hospital sites within the UK, as well as worldwide and participants can be on treatment for a maximum of 12 weeks.
A successful study will establish a new treatment paradigm and a new standard of care for treatment of iTTP for the use of caplacizumab and immunosuppression without first-line TPE.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
22/LO/0531
Date of REC Opinion
21 Sep 2022
REC opinion
Further Information Favourable Opinion