Study to Evaluate the Safety and Effectiveness of Ravulizumab in Adults Diagnosed With Atypical Hemolytic Uremic Syndrome aHUS

What this Trial Is About

Researchers are evaluating the platelet count response to ravulizumab in adults clinically diagnosed with atypical hemolytic uremic syndrome aHUS. This post-marketing, open-label, single-arm study aims to assess the efficacy and safety of ravulizumab, focusing on improving platelet counts and kidney function over a 26-week treatment period. The study is sponsored by Alexion Pharmaceuticals, Inc. Participants will receive a weight-based loading dose of ravulizumab administered through intravenous infusion. Following the initial dose, a second weight-based dose is given two weeks later, then maintenance doses are administered every eight weeks via IV infusion. Treatment is planned to last for 26 weeks, with dosing closely monitored according to body weight. During the study, participants will undergo regular assessments of platelet counts, kidney function, thrombotic microangiopathy TMA responses, and other blood markers at baseline and various time points up to week 26. Researchers will track improvements in platelet count and renal function, dialysis dependency, and changes in hemoglobin and lactate dehydrogenase. Safety and effectiveness will be observed throughout the treatment period, which lasts about six months.

Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS