Follow-up Study for Patients who Completed Study ALX0681-C301
Research type
Research Study
Full title
Prospective Follow-up Study for Patients who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)
IRAS ID
201997
Contact name
Marie Scully
Contact email
Sponsor organisation
Ablynx NV
Eudract number
2016-001503-23
Clinicaltrials.gov Identifier
31446, CPMS ID:
Duration of Study in the UK
4 years, 6 months, 17 days
Research summary
Plasma Exchange (PE) is the principal therapy for acquired thrombotic thrombocytopenic purpura (TTP) and commonly associated with immunosuppressive therapy to eliminate the antibody responsible for hampering the thrombotic process (coagulation) leading to the reduction of circulating platelet count and vascular clot formation (thrombosis). This subsequently result in tissue and organ damage (brain, heart, kidney). In spite of PE therapy, the high level of patient relapse and mortality remain a major concern. The TITAN phase II revealed that the study drug caplacizumab in conjunction with the PE standard of care could benefit to TTP patients by restoring faster platelet level to normal and demonstrating potential protective effect. The current overall risk benefit balance is therefore considered to be positive.
The Phase III study, ALX0681-C301 is currently ongoing in the development of caplacizumab in order to evaluate the efficacy and safety of caplacizumab treatment in reducing more rapidly microvascular thrombosis and preventing recurrent TTP episode. The subject who completed study ALX0681-C301 will be given the option to participate in the follow-up study ALX0681-C302.
The aim of the follow-up study is to evaluate the long-term efficacy and safety of caplacizumab as well as efficacy and safety of the repeated use of the study drug, and long-term impact of TTP. Subjects will attend twice yearly visits for 3 years. Subjects experiencing a recurrence of TTP and who require re-initiation of PE will receive daily administration of the study drug with PE at the hospital until the end of the standard of care. The subjects will then receive at home for 30 days caplacizumab treatment, which can be extended for further 30 days.
The study will last for 3 years. All subjects enrolled in study ALX0681-C301 are eligible to participate in study ALX0681-C302.
REC name
London - South East Research Ethics Committee
REC reference
16/LO/1612
Date of REC Opinion
15 Nov 2016
REC opinion
Further Information Favourable Opinion