A Phase III trial with caplacizumab in patients with acquired thrombotic thrombocytopenic purpura.

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.

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-001098-42-BE

Enrollment

Registered

2015-07-13

Start date

2015-08-27

Completion date

Unknown

Last updated

2017-11-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acquired Thrombotic thrombocytopenic purpura MedDRA version: 18.0 Level: PT Classification code 10043648 Term: Thrombotic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders

Interventions

Product Name: Caplacizumab (an anti-von Willebrand Factor Nanobody) Product Code: ALX-0081 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: Caplacizumab CAS Numb

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 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 PE treatment prior to randomization . Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 89 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3

Exclusion criteria

Exclusion criteria: 1.Platelet count =100×109/L 2.Serum creatinine level >200 µmol/L in case platelet count is > 30×109/L 3.Known other causes of thrombocytopenia 4.Congenital TTP (known at the time of study entry) 5.Pregnancy or breast-feeding.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate efficacy of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis.;Secondary Objective: -To evaluate prevention of recurrence of presenting TTP episode after cessation of daily PE, including prolongation of caplacizumab treatment beyond 30 days post-daily PE treatment based on clinical assessment of underlying disease and ADAMTS13 activity -To evaluate mortality rate -To evaluate the effect of caplacizumab on biomarkers for organ damage: LDH, cTnI, and serum creatinine.;Primary end point(s): Time to platelet count response defined as initial platelet count = 150×109/L with subsequent stop of daily PE within 5 days.;Timepoint(s) of evaluation of this end point: daily PE period	—

Measure

Time frame

Main Objective: To evaluate efficacy of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis.;Secondary Objective: -To evaluate prevention of recurrence of presenting TTP episode after cessation of daily PE, including prolongation of caplacizumab treatment beyond 30 days post-daily PE treatment based on clinical assessment of underlying disease and ADAMTS13 activity -To evaluate mortality rate -To evaluate the effect of caplacizumab on biomarkers for organ damage: LDH, cTnI, and serum creatinine.;Primary end point(s): Time to platelet count response defined as initial platelet count = 150×109/L with subsequent stop of daily PE within 5 days.;Timepoint(s) of evaluation of this end point: daily PE period

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Secondary

Measure	Time frame
Secondary end point(s): 1-Proportion of subjects with an exacerbation and/or relapse of TTP as well as the number of such events 2-Proportion of subjects with treatment-emergent clinically significant TTP-related events as well as the number of such events. 3-Area under the curve (AUC) of platelet count 4-Time to lactate dehydrogenase (LDH) = 2 x upper limit of normal (ULN) 5-Time to cardiac Troponin I (cTnI) = 1 x ULN 6-Time to serum creatinine = 1 x ULN 7-Mortality Rate 8-(Serious)adverse events;Timepoint(s) of evaluation of this end point: 1-During study drug treatment and during follow-up (FU) 2, 4, 5 &6- During the overall study period. 3- until Day 5 7-Initial daily PE period, full study drug treatment period, FU period and overall study period 8-During the overall study period	—

Measure

Time frame

Secondary end point(s): 1-Proportion of subjects with an exacerbation and/or relapse of TTP as well as the number of such events 2-Proportion of subjects with treatment-emergent clinically significant TTP-related events as well as the number of such events. 3-Area under the curve (AUC) of platelet count 4-Time to lactate dehydrogenase (LDH) = 2 x upper limit of normal (ULN) 5-Time to cardiac Troponin I (cTnI) = 1 x ULN 6-Time to serum creatinine = 1 x ULN 7-Mortality Rate 8-(Serious)adverse events;Timepoint(s) of evaluation of this end point: 1-During study drug treatment and during follow-up (FU) 2, 4, 5 &6- During the overall study period. 3- until Day 5 7-Initial daily PE period, full study drug treatment period, FU period and overall study period 8-During the overall study period

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Countries

Australia, Austria, Belgium, Canada, Czech Republic, France, Germany, Hungary, Israel, Italy, Netherlands, New Zealand, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

Public ContactAblynx Clinical Operations

Ablynx NV

clinicaltrials@ablynx.com32(0)9 262 00 00

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026