You have accessJournal of UrologyCME1 May 2022PD05-03 EFFICACY AND SAFETY OF LUMASIRAN IN PATIENTS WITH PRIMARY HYPEROXALURIA TYPE 1: 24-MONTH ANALYSIS OF THE ILLUMINATE-A TRIAL John Lieske, Jaap Groothoff, Yaacov Frishberg, Anne-Laure Sellier-Leclerc, Hadas Shasha-Lavsky, Jeffrey Saland, Wesley Hayes, Daniella Magen, Shabbir Moochhala, Martin Coenen, Eva Simkova, Taylor Ngo, John Gansner, and Sally-Anne Hulton John LieskeJohn Lieske More articles by this author , Jaap GroothoffJaap Groothoff More articles by this author , Yaacov FrishbergYaacov Frishberg More articles by this author , Anne-Laure Sellier-LeclercAnne-Laure Sellier-Leclerc More articles by this author , Hadas Shasha-LavskyHadas Shasha-Lavsky More articles by this author , Jeffrey SalandJeffrey Saland More articles by this author , Wesley HayesWesley Hayes More articles by this author , Daniella MagenDaniella Magen More articles by this author , Shabbir MoochhalaShabbir Moochhala More articles by this author , Martin CoenenMartin Coenen More articles by this author , Eva SimkovaEva Simkova More articles by this author , Taylor NgoTaylor Ngo More articles by this author , John GansnerJohn Gansner More articles by this author , and Sally-Anne HultonSally-Anne Hulton More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000002524.03AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: We report data from the 24-month (M) analysis of ILLUMINATE-A, a Phase 3 trial of lumasiran, an RNAi therapeutic to lower urinary oxalate (UOx) excretion in patients with primary hyperoxaluria type 1 (PH1). METHODS: ILLUMINATE-A is an ongoing Phase 3, randomized, placebo-controlled trial in patients ≥6 years old with genetically confirmed PH1 and eGFR ≥30 mL/min/1.73m2, with a 6M primary analysis period followed by an extension period (EP) of up to 54M where all patients receive lumasiran. RESULTS: Of 39 patients enrolled, 24/26 in the lumasiran/lumasiran (L/L) group and 13/13 in the placebo/lumasiran (P/L) group entered the EP. Mean 24h UOx reduction at M24 relative to baseline was 58% in the L/L group and 49% in the P/L group. The proportion of patients achieving 24h UOx excretion ≤1.5x ULN at M24 was 83% in the L/L group and 62% in the P/L group; mean reductions from baseline in plasma oxalate at M24 were 56% and 61%, respectively. eGFR remained stable in both groups. Kidney stone event rates decreased from 3.19/person-year during the 12 months prior to consent to 0.80 in the L/L group and from 0.54/person-year to 0.28 in the P/L group. The most common lumasiran-related adverse events were mild, transient injection-site reactions. CONCLUSIONS: Long-term lumasiran treatment resulted in sustained reduction in UOx through M24 with acceptable safety in patients with PH1 and encouraging results on clinical outcomes. Source of Funding: Alnylam Pharmaceuticals © 2022 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 207Issue Supplement 5May 2022Page: e87 Advertisement Copyright & Permissions© 2022 by American Urological Association Education and Research, Inc.MetricsAuthor Information John Lieske More articles by this author Jaap Groothoff More articles by this author Yaacov Frishberg More articles by this author Anne-Laure Sellier-Leclerc More articles by this author Hadas Shasha-Lavsky More articles by this author Jeffrey Saland More articles by this author Wesley Hayes More articles by this author Daniella Magen More articles by this author Shabbir Moochhala More articles by this author Martin Coenen More articles by this author Eva Simkova More articles by this author Taylor Ngo More articles by this author John Gansner More articles by this author Sally-Anne Hulton More articles by this author Expand All Advertisement PDF DownloadLoading ...