Transplant-associated Thrombotic Microangiopathy (TA TMA) research hub

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)

This study is looking at a new medicine called Pegcetacoplan for adults who have developed a rare but serious condition called Transplant-associated Thrombotic Microangiopathy (TA-TMA) after a stem cell transplant. It aims to see how the medicine works in the body, if it's safe, and if it helps people get better.