Research Project

Project Title:	Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy (APPELHUS)
Project Type:	Clinical Trial, Enrolment ongoing
Disease group(s):	Thrombotic microangiopathies
Project Summary:	The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatment naive to complement inhibitor therapy (including anti-C5 antibody). The study will enroll approximately 50 participants and assess the effects of iptacopan on a range of efficacy assessments relevant to aHUS including hematological and kidney parameters, dialysis requirement, changes in chronic kidney disease (CKD) stage, as well as patient reported outcomes (PRO) for fatigue and quality of life.
Lead principal investigator(s):	Smaragdi Marinaki, Athens
Co-investigator(s):	Ioannis Boletis, Athens Stathis Tsiakas, Athens Chrysanthi Skalioti, Athens
Project Period:	01/2022 - 01/2026