Table 2.

Key study assessments

Assessment category	Assessment
Key efficacy assessments	Complete TMA responsea
	Hematologic parameters
	eGFR and CKD stage
	Patient-reported outcomes (FACIT-fatigue)
Key safety assessments	Vital signs
	Laboratory evaluations in blood and urine
	Electrocardiogram
	Pregnancy and assessments of fertility
	Selected biomarkers related to disease progression
	Safety and efficacy parameters in patients with different aHUS genetic mutations
Other assessments	Health care resource utilization
	The need for hemodialysis or peritoneal dialysis will be monitored by the investigator during the study

aHUS, atypical hemolytic uremic syndrome; CKD, chronic kidney disease; eGFR, estimated glomerular filtration rate; FACIT-Fatigue, Functional Assessment of Chronic Illness Therapy-Fatigue; TMA, thrombotic microangiopathy.

^aComplete TMA response is defined as: (1) hematological normalization in platelet count (platelet count ≥150 × 10⁹/l) and lactate dehydrogenase (below upper limit of normal); and (2) improvement in kidney function (≥25% serum creatinine reduction from baseline), maintained for 2 measurements obtained at least 4 weeks apart, and any measurement in between.