Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

Investigation of an Investigational Medication and Immunosuppressive Therapy for Immune-Mediated Thrombotic Thrombocytopenic Purpura

Recruiting
18 years - 80 years
All
Phase 3
61 participants needed
15 Locations

Study Overview

This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP).

The anticipated study duration per participant without a recurrence while on therapy is maximum 24 weeks (ie, approximately 1 day for screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up). Participants will have daily assessments during hospitalization and weekly visits for assessments during ongoing treatment with caplacizumab and IST. There will be 3 outpatient visits for assessments during the follow-up period. There will be two additional follow-up visits for participants who do not have ADAMTS13 activity levels of ≥50% at the time of caplacizumab discontinuation.

Study Details

The anticipated study duration per participant with the presenting episode therefore is a maximum of about 24 weeks (ie, 1 day of screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up).

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Thrombotic Thrombocytopenic Purpura
  • Age: 18 years - 80 years
  • Gender: All

Inclusion Criteria:

        Participants with a clinical diagnosis of iTTP (initial or recurrent), which includes
        thrombocytopenia, microangiopathic hemolytic anemia (eg, presence of schistocytes in
        peripheral blood smear) and relatively preserved renal function. The iTTP diagnosis should
        be confirmed by ADAMTS13 testing within 48 hours (2 days).
        Participants with a clinical diagnosis of iTTP and a French TMA score of 1 or 2.
        A female participant is eligible to participate if she is not pregnant or breastfeeding,
        and one of the following conditions applies:
          -  Is a woman of nonchildbearing potential (WONCBP), OR
          -  Is a woman of childbearing potential (WOCBP) and agrees to use an acceptable
             contraceptive method during the overall treatment period and for at least 2 months
             after the last study drug administration.
        Male participants with female partners of childbearing potential must agree to follow the
        contraceptive guidance as per protocol during the overall treatment period and for at least
        2 months after last study drug administration.
        Exclusion Criteria:
        Platelet count ≥100 x 10^9/L. Serum creatinine level >2.26 mg/dL (200 µmol/L) in case
        platelet count is >30 x 10^9/L (to exclude possible cases of atypical HUS).
        Known other causes of thrombocytopenia including but not limited to:
          -  Clinical evidence of enteric infection with E. coli 0157 or related organism.
          -  Atypical HUS.
          -  Hematopoietic stem cell, bone marrow or solid organ transplantation-associated
             thrombotic microangiopathy.
          -  Known or suspected sepsis.
          -  Diagnosis of disseminated intravascular coagulation. Congenital TTP (known at the time
             of study entry). Clinically significant active bleeding or known co-morbidities
             associated with high risk of bleeding (excluding thrombocytopenia).
        Inherited or acquired coagulation disorders. Malignant arterial hypertension. Participants
        requiring or expected to require invasive procedures immediately (eg, stroke requiring
        thrombolytic therapy, those who need mechanical ventilation, etc.).
        Those presenting with severe neurological or cardiac disease. Clinical condition other than
        that associated with TTP, with life expectancy <6 months, such as end-stage malignancy.
        Known chronic treatment with anticoagulants and anti-platelet drugs that cannot be stopped
        (interrupted) safely, including but not limited to:
          -  vitamin K antagonists.
          -  direct-acting oral anticoagulants.
          -  heparin or low molecular weight heparin (LMWH).
          -  non-steroidal anti-inflammatory molecules other than acetyl salicylic acid.
             Participants who were previously enrolled in this clinical study (study EFC16521).
        Participants who received an investigational drug, or device, other than caplacizumab,
        within 30 days of anticipated IMP administration or 5 half-lives of the previous
        investigational drug, whichever is longer.
        Positive result on COVID test.
        The above information is not intended to contain all considerations relevant to a potential
        participation in a clinical trial.

Updated on 10 May 2024. Study ID: NCT05468320

This study investigates the use of caplacizumab and immunosuppressive therapy for treating immune-mediated thrombotic thrombocytopenic purpura (iTTP). iTTP is a condition where the immune system mistakenly attacks certain proteins in the blood, leading to low platelet levels. This can cause problems like easy bruising and bleeding. The purpose of this study is to see how effective and safe this treatment is for people aged 18 to 80 years who have iTTP.

Participants in the study will receive daily assessments during their hospital stay and weekly visits during their treatment. They will continue with regular check-ups to monitor their response to the therapy. The study will also include a follow-up period where participants will have outpatient visits to assess their health and any changes in their condition.

  • Who can participate: Adults aged 18 to 80 years with a clinical diagnosis of iTTP confirmed by ADAMTS13 testing can participate. Female participants must not be pregnant or breastfeeding, and all participants must agree to use contraception if applicable.
  • Study details: Participants will receive treatment with an investigational medication and immunosuppressive therapy without the use of therapeutic plasma exchange. A placebo is not used in this study.
  • Study Timelines and Visits: The study will last 24 weeks. The study requires 3 outpatient visits.

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