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Abstract: FR-PO592

Lumasiran for Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 24-Month Analysis of the Phase 3 ILLUMINATE-C Trial

Session Information

Category: Genetic Diseases of the Kidneys

  • 1202 Genetic Diseases of the Kidneys: Non-Cystic


  • Lieske, John C., Mayo Clinic Division of Nephrology and Hypertension, Rochester, Minnesota, United States
  • Magen, Daniella, Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa, Israel
  • Sellier-Leclerc, Anne-Laure All, Hôpital Femme Mère Enfant en Centre d’Investigation Clinique INSERM, Hospices Civils de Lyon, ERKnet, Bron, France
  • Shasha-Lavsky, Hadas, Pediatric Nephrology Unit, Galilee Medical Center, Nahariya, Israel
  • Simkova, Eva, Nephrology - Medical Affairs, Al Jalila Children’s Specialty Hospital, Dubai, United Arab Emirates
  • Devresse, Arnaud, Division of Nephrology, Cliniques Universitaires Saint-Luc, Brussels, Belgium
  • Michael, Mini, Texas Children’s Hospital/Baylor College of Medicine, Houston, Texas, United States
  • Frishberg, Yaacov, Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem, Israel
  • Bakkaloglu, Sevcan A., Department of Pediatric Nephrology, Faculty of Medicine, Gazi University, Ankara, Turkey
  • Mourani, Chebl, Pediatrics, Hôtel-Dieu de France Hospital (HDF), Beirut, Lebanon
  • Saqan, Rola, Pharmaceutical Research Center - Jordan University of Science and Technology, Irbid, Jordan
  • Singer, Richard F., Senior Staff Specialist, Renal Service, Canberra Health Services, Garran, Australian Capital Territory, Australia
  • Guzzo, Isabella, Ospedale Pediatrico Bambino Gesu, Rome, Italy
  • Makarova, Nune, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Willey, Richard G., Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Kaspar, Cristin, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Gansner, John M., Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Groothoff, Jaap, Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands

Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder in which hepatic oxalate overproduction can lead to chronic kidney disease (CKD). Plasma oxalate (POx) increases with declining kidney function, leading to systemic oxalosis. Lumasiran, an RNA interference therapeutic that reduces hepatic oxalate production, administered to patients with PH1 and CKD 3b–5 in the ILLUMINATE-C trial (NCT04152200), resulted in decreased POx at month (M) 6 and 12 with acceptable safety. Here we present M24 results.


ILLUMINATE-C is an ongoing, single-arm study evaluating lumasiran in patients of all ages with PH1, eGFR ≤45 mL/min/1.73m2 including hemodialysis (HD), and POx ≥20 μmol/L. The 6M primary analysis period is followed by a 54M extension period.


Of 21 patients who entered the study, 5 of 6 (83%) assigned at study start to Cohort A (no HD) and 12 of 15 (80%) assigned to Cohort B (on HD) completed the M24 visit. Mean reduction from baseline in POx at M24 was 60.5% (Cohort A) and 30.6% (Cohort B). Two Cohort A patients with low baseline eGFR (8.6, 16 mL/min/1.73m2) initiated HD. Two Cohort B patients who underwent kidney-only transplantation remained in the study with a functional graft after 16 and 21 months and comparable POx levels to 2 patients who underwent combined liver-kidney transplant. In patients with an ejection fraction (EF) <55% at baseline, 2 of 3 had improvement of ≥5% at M24; none met criteria for EF worsening. Mild injection site reactions were the most common lumasiran-related AEs, occurring in 5 of 21 (24%) patients overall. No deaths or lumasiran-related serious/severe AEs, discontinuations, or withdrawals occurred.


POx reductions with lumasiran were maintained at M24 with an acceptable safety profile in PH1 patients with CKD 3b–5, including those on HD, and in patients with an isolated kidney transplant. Noninvasive indicators of systemic oxalosis including EF appear stable or possibly improving after 2 years of treatment in this study population.


  • Commercial Support – Alnylam Pharmaceuticals