Summary

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• This summary is based on the review of four randomized controlled trial(s). ^[1-4]
• In the Phase III ILLUMINATE Clinical Trials (ILLUMINATE)-B study, infants and children under 6 years old with primary hyperoxaluria type 1 (PH1) showed a 72% reduction in the urinary oxalate ratio at 12 months, with reductions ranging from 68% to 89% across weight subgroups. Plasma oxalate levels decreased by 47% at 12 months.
• The ILLUMINATE-C study in PH1 patients with advanced kidney disease showed plasma oxalate reductions of 33.3% in non-hemodialysis patients and 42.4% in those receiving hemodialysis. All pharmacodynamic secondary endpoints also showed improvements, including urinary oxalate reductions.
• In the ILLUMINATE-B study, mild and transient injection-site reactions were reported in 17% of patients. No severe adverse events were recorded.
• Across the ILLUMINATE-C, single-arm, open-label phase 3, and double-blind phase 3 studies, the most common lumasiran-related adverse events were mild, transient injection-site reactions, affecting 38% of lumasiran-treated patients in the double-blind trial. No patients discontinued treatment due to adverse events.
• There is no population types or subgroups information available in the reviewed studies.