A Phase III double-blind, randomized, parallel group, multicenter placebo-controlled trial to study the efficacy and safety of caplacizumab in patients with acquired thrombotic thrombocytopenic purpura.
- Conditions
- Thrombotic Thrombocytopenic Purpura10038158
- Registration Number
- NL-OMON43776
- Lead Sponsor
- Ablynx NV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 4
1.Adult male or female * 18 years of age at the time of signing the informed consent form (ICF)
2.Clinical diagnosis of acquired TTP (initial or recurrent), which includes thrombocytopenia and microscopic evidence of red blood cell fragmentation (e.g. schistocytes)
3.Requires initiation of daily PE treatment and has received 1 PE treatment prior to randomization .
* Platelet count *100×109/L
* Serum creatinine level >200 *mol/L in case platelet count is > 30×109/L (to exclude possible cases of atypical Hemolytic Uremic Syndrome [aHUS])
* 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 organ transplantation-associated thrombotic microangiopathy
- Known or suspected sepsis
- Diagnosis of disseminated intravascular coagulation
* Congenital TTP (known at the time of study entry)
* Pregnancy or breast-feeding
* Clinically significant active bleeding or high risk of bleeding (excluding thrombocytopenia)
* Known chronic treatment with anticoagulant treatment that cannot be stopped (interrupted) safely, including but not limited to:
- vitamin K antagonists
- heparin or low molecular weight heparin (LMWH)
- non-acetyl salicylic acid non-steroidal anti-inflammatory molecules
* Malignant arterial hypertension
* Clinical condition other than that associated with TTP, with life expectancy < 6 months, such as end-stage malignancy
* Subjects who were previously enrolled in a clinical study with caplacizumab and received caplacizumab or for whom the assigned treatment arm is unknown.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>To evaluate efficacy of caplacizumab in more rapidly restoring normal platelet<br /><br>counts as measure of prevention of further microvascular thrombosis</p><br>
- Secondary Outcome Measures
Name Time Method <p>- to evaluate the effect of study drug on a composite endpoint consisting of<br /><br>TTP-related mortality, recurrence of TTP and major thromboembolic events during<br /><br>study drug treatment<br /><br>- to evaluate the effect of study drug on prevention of recurrence of TTP over<br /><br>the entire study period<br /><br>- to evaluate the effect of study drug on refractoriness to treatment<br /><br>- to evaluate the effect of study drug on biomarkers of organ damage: lactate<br /><br>dehydrogenase (LDH), cardiac troponin I (cTnI), and serum creatinine<br /><br>- to evaluate the effect of study drug on PE parameters (days of PE and<br /><br>volume), days in intensive care unit (ICU), days in hospital<br /><br>- adverse events (AEs)<br /><br>- pharmacodynamic (PD) markers: von Willebrand factor (vWF), coagulation factor<br /><br>VIII (FVIII), ristocetin cofactor activity (RICO)<br /><br>- pharmacokinetic (PK) parameters<br /><br>- immunogenicity (anti-drug antibodies [ADA])</p><br>