Clinical Trial: Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: 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 T

Brief Summary: The study is a phase III, double blind, placebo-controlled, randomized study to evaluate the efficacy and safety of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis

Detailed Summary:
Sponsor: Ablynx

Current Primary Outcome: Time to platelet count response [ Time Frame: For maximum 6 months ]

Initial platelet count ≥ 150×10E9/L with subsequent stop of daily plasma exchange within 5 days.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Proportion of subjects with TTP-related death, a recurrence of TTP, or at least one treatment-emergent major thromboembolic event during the study drug treatment period (including extensions) [ Time Frame: From Day 1 until last dosing ]
  • Proportion of subjects with a recurrence of TTP in the overall study period [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • Proportion of subjects with refactory TTP, defined as absence of platelet count doubling after 4 days of standard treatment, and LDH > ULN [ Time Frame: Day 5 ]
  • Time to normalization of all 3 of the following organ damage marker levels [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
    Time to normalization of all 3 of the following organ damage marker levels: Time to LDH equal or lower than 1 time the upper limit of normal (ULN) and cardiac Troponin I (cTnI) equal or lower than 1 time ULN and serum creatinine equal or lower than 1 time ULN
  • (Serious) adverse events [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]


Original Secondary Outcome:

  • Proportion of subjects with an exacerbation and/or relapse of TTP as well as the number of such events [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • Proportion of subjects with treatment-emergent clinically significant TTP-related events as well as the number of such events [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • Area under the curve (AUC) of platelet count [ Time Frame: Day 5 ]
  • Time to lactate dehydrogenase (LDH) ≤ 2 x upper limit of normal (ULN) [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • Time to cardiac Troponin I (cTnI) ≤ 1 x ULN [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • Time to serum creatinine ≤ 1 x ULN [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • Mortality rate [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]
  • (Serious) adverse events [ Time Frame: From Day 1 until last follow-up visit (28 days after last dosing) ]


Information By: Ablynx

Dates:
Date Received: September 14, 2015
Date Started: September 2015
Date Completion: October 2017
Last Updated: March 10, 2017
Last Verified: March 2017