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Study on Caplacizumab and Immunosuppressive Therapy for Adults with Immune-Mediated Thrombotic Thrombocytopenic Purpura

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare blood disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP). This condition involves the formation of small blood clots throughout the body, which can lead to a low platelet count and damage to organs. The study will explore the effectiveness and safety of a treatment using a medication called caplacizumab, in combination with immunosuppressive therapy. Immunosuppressive therapy is a treatment that helps reduce the activity of the immune system. The study will not use the usual first-line treatment known as therapeutic plasma exchange, which is a procedure that removes and replaces blood plasma.

The purpose of the study is to evaluate how well the combination of caplacizumab and immunosuppressive therapy works in treating adults with iTTP. Participants in the study will receive caplacizumab, which is given as an injection either into a vein (intravenous) or under the skin (subcutaneous). The study will monitor participants over a period of time to see if they achieve remission, which means the symptoms of iTTP improve or disappear without needing therapeutic plasma exchange. The study will also look at the occurrence of any side effects or adverse events during the treatment period.

Participants will be observed for their response to the treatment, including how quickly their platelet count returns to normal and whether they experience any relapses or worsening of their condition. The study aims to provide valuable information on the potential benefits of using caplacizumab and immunosuppressive therapy for treating iTTP, offering insights into alternative treatment options for this serious condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, a clinical diagnosis of immune-mediated thrombotic thrombocytopenic purpura (iTTP) is confirmed. This includes checking for low platelet count, specific blood cell damage, and normal kidney function.

    The diagnosis is confirmed through a test called ADAMTS13 within 48 hours.

  2. Step 2

    Treatment initiation

    The treatment involves the administration of caplacizumab, a medication provided as a 10 mg powder and solvent for solution for injection.

    The medication is administered either intravenously (IV) or subcutaneously (SC), depending on the specific requirements of the treatment plan.

  3. Step 3

    Treatment duration

    The treatment period is divided into two phases: the on-treatment period from day 1 to day 84, and the overall study period from day 1 to day 168.

    During this time, the effectiveness of the treatment is monitored, focusing on achieving remission without the need for therapeutic plasma exchange (TPE).

  4. Step 4

    Monitoring and evaluation

    Throughout the study, the response to treatment is evaluated by monitoring platelet count and other health indicators.

    The occurrence of any adverse events, including serious ones, is recorded to ensure safety.

  5. Step 5

    Completion of study

    The study is estimated to conclude by December 13, 2024.

    Upon completion, the results will be analyzed to determine the proportion of participants achieving remission and other health outcomes.

Who can join the trial?

4 criteria

  • Participants must have a clinical diagnosis of immune mediated thrombotic thrombocytopenic purpura (iTTP). This includes having low platelet counts, a type of anemia where red blood cells are broken down, and relatively normal kidney function. The diagnosis should be confirmed by a specific test called ADAMTS13 within 48 hours (2 days).
  • Participants must have a clinical diagnosis of iTTP and a French TMA score of 1 or 2. This score helps assess the severity of the condition.
  • Female participants must not be pregnant or breastfeeding. They must either be women who cannot have children or agree to use an acceptable method of birth control during the treatment period and for at least 2 months after the last dose of the study drug.
  • Male participants with female partners who can have children must agree to follow the birth control guidance during the treatment period and for at least 2 months after the last dose of the study drug.

Who cannot join the trial?

8 criteria

  • Patients who are not adults cannot participate. This means you must be 18 years or older.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have a history of allergic reactions to the study medication or similar medications cannot participate.
  • Patients who are currently participating in another clinical trial cannot participate.
  • Patients who have a condition that affects their ability to understand or follow the study instructions cannot participate.
  • Patients who have a history of drug or alcohol abuse that might interfere with the study cannot participate.
  • Patients who have received certain treatments recently that might interfere with the study cannot participate.
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Investigated drugs

  • Caplacizumab

    is a medication used in this study to help treat a condition called immune-mediated thrombotic thrombocytopenic purpura (iTTP). It works by preventing the formation of blood clots, which can help reduce the risk of complications associated with this condition.

  • Immunosuppressive Therapy (IST)

    involves using medications that suppress or reduce the strength of the body's immune system. In this trial, IST is used alongside caplacizumab to help manage iTTP by preventing the immune system from attacking the body's own cells, which can lead to the symptoms of this condition.

What is already known about the treatment

Caplacizumab – Caplacizumab is administered via injection and is used in combination with immunosuppressive therapy to treat adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP). It is currently being studied in clinical trials to assess its effectiveness and safety without the need for first-line therapeutic plasma exchange. The medication works by targeting a specific protein involved in blood clot formation, helping to prevent the formation of harmful clots. Caplacizumab is classified as an anti-thrombotic agent.

Investigated diseases

Thrombotic Thrombocytopenic Purpura – This is a rare blood disorder characterized by the formation of small blood clots throughout the body. These clots can lead to a low platelet count, which is essential for normal blood clotting, and can cause bleeding issues. The disease often begins suddenly and can progress rapidly, affecting various organs due to restricted blood flow. Symptoms may include fatigue, fever, and neurological changes such as confusion or headaches. The condition is immune-mediated, meaning the body's immune system mistakenly attacks its own cells, contributing to the disease's progression. It requires careful management to prevent complications related to blood clotting and bleeding.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-513262-19-00Protocol codeEFC16521Estimated enrolment73 patientsSponsorSanofi-Aventis Recherche & Developpement

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