biostudies-literatureUnknown9(7)Saland JM<h4>Introduction</h4>Patients with primary hyperoxaluria type 1 (PH1), a genetic disorder associated with hepatic oxalate overproduction, frequently experience recurrent kidney stones and worsening kidney function. Lumasiran is indicated for the treatment of PH1 to lower urinary and plasma oxalate (POx).<h4>Methods</h4>ILLUMINATE-A (NCT03681184) is a phase III trial in patients aged ≥6 years with PH1 and estimated glomerular filtration rate (eGFR) ≥30 ml/min per 1.73 m². A 6-month double-blind placebo-controlled period is followed by an extension period (≤54 months; all patients receive lumasiran). We report interim data through month 36.<h4>Results</h4>Of 39 patients enrolled, 24 of 26 (lumasiran/lumasiran group) and 13 of 13 (placebo/lumasiran group) entered and continue in thKidney international reports2037-2046https://www.ebi.ac.uk/biostudies/studies/S-EPMC11284403biostudies-literatureEfficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial.PMC11284403Magen DLieske JCSaland JMHulton SACoenen MSellier-Leclerc ALHogan JGansner JMSimkova EShasha-Lavsky HWilley RGroothoff JWHayes WMoochhala SHFrishberg YfalseEfficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1: Results from a Phase III Clinical Trial.<h4>Introduction</h4>Patients with primary hyperoxaluria type 1 (PH1), a genetic disorder associated with hepatic oxalate overproduction, frequently experience recurrent kidney stones and worsening kidney function. Lumasiran is indicated for the treatment of PH1 to lower urinary and plasma oxalate (POx).<h4>Methods</h4>ILLUMINATE-A (NCT03681184) is a phase III trial in patients aged ≥6 years with PH1 and estimated glomerular filtration rate (eGFR) ≥30 ml/min per 1.73 m². A 6-month double-blind placebo-controlled period is followed by an extension period (≤54 months; all patients receive lumasiran). We report interim data through month 36.<h4>Results</h4>Of 39 patients enrolled, 24 of 26 (lumasiran/lumasiran group) and 13 of 13 (placebo/lumasiran group) entered and continue in th2024-01-01T00:00:00Z2024 Jul2026-06-02T23:12:20.182Z2025-04-19T14:49:51.803ZS-EPMC112844033908173810.1016/j.ekir.2024.04.048