Study of Iptacopan Treatment Switching from Anti-C5 Antibody in Adults With Atypical Hemolytic Uremic Syndrome

What this Trial Is About

Researchers are evaluating the safety and effectiveness of switching from anti-C5 antibody treatment to iptacopan in adults diagnosed with atypical hemolytic uremic syndrome aHUS. This Phase 3, multicenter, single-arm, open-label study aims to provide important data on how iptacopan works in patients who have already responded to anti-C5 antibody treatment. The study is sponsored by Novartis Pharmaceuticals and focuses on key measures related to aHUS symptoms and kidney function. Participants will receive iptacopan at a dose of 200 mg twice daily during the treatment periods. The study includes an initial screening period lasting up to 14 weeks, followed by a 12-month core treatment phase and then a 12-month extension treatment phase. This design allows researchers to observe the effects of iptacopan over a full two years after switching from previous therapy. Throughout the study, participants will undergo regular assessments including blood tests for platelet count, LDH, hemoglobin, kidney function markers such as serum creatinine and eGFR, and urine protein levels. Researchers will monitor for signs of thrombotic microangiopathy TMA and kidney disease progression, as well as track the need for dialysis. Safety and clinical responses will be evaluated at multiple time points to understand the treatment impact and participant well-being.

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)