ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1)

Primary Hyperoxaluria Type 1 (PH1)

Cohort A: Percent change in plasma oxalate from Baseline to Month 6, Cohort B: Percent change in plasma oxalate from Baseline to Month 6

Primary Analysis Period (Baseline to 6 months): Percent change in plasma oxalate AUC between dialysis sessions (Cohort B), Absolute change in plasma oxalate, Change in the following parameters: *Urinary oxalate measured by percent and absolute change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) and spot urinary oxalate:creatinine ratio, when available *QoL assessed by the PedsQL Total Score for patients ≥2 to <18 years of age at consent, and as assessed by KDQOL Burden of Kidney Disease and Effect of Kidney Disease on Daily Life subscales, and SF-12 Physical Component Summary and Mental Component Summary, in patients ≥18, Plasma PK parameters of lumasiran, Percent change in plasma oxalate AUC between dialysis sessions (Cohort B), Percent and absolute change in plasma oxalate, Change in the following parameters: *Nephrocalcinosis as assessed by renal ultrasound; *Frequency and mode of dialysis (Cohort B); *Frequency of renal stone events; *Urinary oxalate measured by 24-hour urinary oxalate excretion corrected for BSA, and spot urinary oxalate:creatinine ratio; *Renal function as assessed by estimated glomerular filtration rate (eGFR) (Cohort A); *Measures of systemic oxalosis in the following systems: -Cardiac -Skeletal -Ocular; *QoL(Quality of life) assessed

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