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
- Conditions
- acquired TTPimmune-mediated TTP10035534
- Registration Number
- NL-OMON53928
- Lead Sponsor
- Sanofi BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 2
- 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
- Platelet count >=100 × 109/L.
- Serum creatinine level >2.26 mg/dL (200 µmol/L) in case platelet count is >30
× 109/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 severe 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>- Proportion of participants achieving Remission without requiring TPE.</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Proportion of participants achieving Remission<br /><br>- Proportion of participants who require TPE<br /><br>- The occurrence of adverse events (AEs), serious adverse events (SAEs), and<br /><br>adverse events of special interest (AESIs)<br /><br>- Proportion of participants achieving Clinical Response<br /><br>- Time to platelet count response<br /><br>- Proportion of participants refractory to therapy<br /><br>- Proportion of participants with TTP-related death<br /><br>- Proportion of participants with a clinical exacerbation of iTTP<br /><br>- Proportion of participants with a clinical relapse of iTTP</p><br>