Table 2.

Summary of study assessments

Assessment category	Assessment
Efficacy	Proteinuria (UPCR) eGFR Composite renal end point (1) a stable or improved eGFR compared with baseline visit (≤15% reduction in eGFR); and (2) a ≥50% reduction in UPCR compared with baseline visit] Patient-reported outcome – (FACIT-Fatigue) score
Key safety	Adverse event monitoring Laboratory evaluations (blood and urine) Electrocardiogram Cardiovascular surveillance (adolescents only)
Other	Complement pathway and renal injury biomarkers Patient-reported outcomes – SF-36, EQ-5D-5L and PGIS Iptacopan levels at trough (PK assessment)

eGFR, estimated glomerular filtration rate; EQ-5D-5L, EuroQol-5 dimensions-5 levels; FACIT-Fatigue, functional assessment of chronic illness therapy-fatigue; PGIS, patient global impression of severity; PK, pharmacokinetic; SF-36, Short-Form 36; UPCR, urine protein–creatinine ratio.