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

  damoun.nourbakht2@sanofi.com

• Sponsor organisation

  Sanofi-Aventis Recherche & Développement

• Eudract number

  2022-001177-31

• Clinicaltrials.gov Identifier

  NCT05468320

• 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

  Lay summary
  "results to be posted: https://www.clinicaltrials.gov/study/NCT05468320?tab=results

  Key information about this study
  • What is caplacizumab? Caplacizumab is a medicine that has been developed to treat people with immune-mediated thrombotic thrombocytopenic purpura (iTTP), also known as acquired thrombotic thrombocytopenic purpura (aTTP). iTTP is a rare blood condition that
  occurs when blood clots form in small blood vessels throughout the body.
  • Reason for this study: Caplacizumab is used in combination with two other treatments, therapeutic plasma exchange (TPE) and immunosuppressive therapy (IST), to treat iTTP. This study wanted to see if caplacizumab worked and was safe for people with iTTP when it
  was used only with IST. The study did not include TPE.
  • What was tested: Caplacizumab was tested in combination with IST.
  • Who took part in this study: 51 people in 11 countries took part. Everyone had iTTP.
  • Results of the study: Caplacizumab worked to treat iTTP in people when given with IST and without TPE. Most people’s iTTP improved.
  • Safety: People experienced side effects from the treatments, but caplacizumab in combination with IST was well tolerated overall."
  

• 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