Transplant-Associated Thrombotic Microangiopathy Clinical Trial
Official title:
An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients With Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)
The purpose of the study is to assess PK, Pharmacodynamics (PD), Efficacy and safety of pegcetacoplan in patients with TA-TMA after HSCT.
This is a pilot study and the sample size is based on practical rather than statistical aspects. A total of 12 patients will be included and treated in the study. With 12 patients included, it is estimated that 9 patients will complete at least 4 weeks of treatment, which is deemed sufficient to characterize the PK of pegcetacoplan in patients with TA-TMA to an appropriate precision. In addition, 12 patients will provide a 72 % probability to observe a response rate of at least 8 responders of the 12 patients recruited (assuming the true response rates is 70 %). ;