Table 3.

Key study objectives and related endpoints

Assessment	Objective	End point
Primary objective	To assess the proportion of patients treated with iptacopan achieving complete TMA responsea during 26 weeks of study treatment	Complete TMA responsea without the use of PE/PI or anti-C5 antibody therapy during 26 weeks of study treatment
Key secondary objectives	To assess the effect of iptacopan on time to complete TMA response	Time to achieve TMA response during 26 weeks of study treatment
	To assess the proportion of patients achieving an increase of ≥2 g/dl from baseline in hemoglobin levels	An increase in hemoglobin of ≥2 g/dl from baseline during 26 weeks of study treatment
	To assess the following at week 26: •The effect of iptacopan on hematological parameters (platelets, lactate dehydrogenase, hemoglobin) •The effect of iptacopan on eGFR, CKD stage, patient-reported overall fatigue severity, and health-related quality of life	Change from baseline in relevant parameters at week 26
	To assess the effect of iptacopan on dialysis requirement status	Proportion of patients on dialysis who no longer require it through 26 weeks of study treatment
	To assess the safety and tolerability of iptacopan	Adverse events/serious adverse events, laboratory parameters, and vital signs

CKD, chronic kidney disease; eGFR, estimated glomerular filtration rate; PE/PI, plasma exchange/plasma infusion; TMA, thrombotic microangiopathy.

^aComplete TMA response: see Table 2 for definition.