acquired TTP immune-mediated TTP
Conditions
Interventions
Administration of caplacizumab combined with IST, without TPE (standard of care
is inclusive of TPE)
Sponsors
Sanofi BV
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
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
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Proportion of participants achieving Remission without requiring TPE. | — |
Secondary
| Measure | Time frame |
|---|---|
| - Proportion of participants achieving Remission - Proportion of participants who require TPE - The occurrence of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs) - Proportion of participants achieving Clinical Response - Time to platelet count response - Proportion of participants refractory to therapy - Proportion of participants with TTP-related death - Proportion of participants with a clinical exacerbation of iTTP - Proportion of participants with a clinical relapse of iTTP | — |
Countries
Netherlands
Outcome results
None listed