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Abstract: TH-PO721

Safety and Efficacy Study of Lumasiran (ALN-GO1), an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Primary Hyperoxaluria Type 1

Session Information

Category: Genetic Diseases of the Kidney

  • 1002 Genetic Diseases of the Kidney: Non-Cystic


  • Hulton, Sally, Birmingham Childrens'' Hospital, Birmingham, United Kingdom
  • Frishberg, Yaacov, Shaare Zedek Medical Center, Jerusalem, Israel
  • Deschênes, Georges, Hospital Robert Debre/Pediatric Nephrology, Paris, France
  • Cochat, Pierre, Université ClaudeBernard Lyon1-, Bron, France
  • Magen, Daniella, Rambam Health Care Campus, Haifa, Israel
  • Groothoff, Jaap Willem, Emma Children's Hospital AMC, Amsterdam, Netherlands
  • Harambat, Jerome, Bordeaux University Hospital, Bordeaux, France
  • van't Hoff, William, Great Ormond Street Hospital, Lond, United Kingdom
  • Hoppe, Bernd, University Hospital Bonn, Bonn, Germany
  • Lieske, John C., Mayo Clinic , Rochester, Minnesota, United States
  • McGregor, Tracy, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Haslett, Patrick, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Erbe, David V., Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Milliner, Dawn S., Mayo Clinic , Rochester, Minnesota, United States

In Primary Hyperoxaluria Type 1 (PH1), defective alanine:glyoxylate aminotransferase leads to excessive hepatic oxalate production, leading to progressive renal impairment and multi-organ damage from systemic oxalosis. Lumasiran, an investigational RNAi therapeutic suppresses hepatic glycolate oxidase, decreases the conversion of glycolate to glyoxylate, and results in reduced oxalate production.


ALN-GO1-001 is a randomized, placebo-controlled, single-blind, multicenter trial, evaluating lumasiran in patients with PH1 ≥6 years of age with urinary oxalate (UOx) ≥0.7 mmol/1.73m2/day and eGFR >45 mL/min/1.73m2. One of four patients in each dosing cohort was randomized to placebo prior to subcutaneous lumasiran. Cohorts 1 & 2 received 3 monthly doses of 1 mg/kg or 3 mg/kg, respectively; cohort 3 received 2 quarterly doses of 3 mg/kg lumasiran. An additional 4 patients received lumasiran in expansions of each of the first 2 cohorts. The primary endpoint is safety; secondary endpoints include change in 24-hour UOx from baseline. Eligible patients may continue dosing in the open-label extension (OLE) study.


Patients in cohorts 1-3 had a mean age of 13.1 years (range 6-43), 7 (58%) female, and baseline UOx was 1.58 mmol/1.73m2/day (range 0.63-2.37). Lumasiran has demonstrated acceptable preliminary safety and tolerability with no treatment related serious adverse events or discontinuations; majority of adverse events were mild/moderate and unrelated to study drug. All patients treated with lumasiran in cohorts 1-3 experienced UOx lowering below 0.7 mmol/1.73m2/day, with a mean maximal decrease of 65%. Data available 85 days after initial dosing in the first 3 cohorts (n=9) showed a mean UOx reduction of 63% (range 49-73%). Data from patients in all cohorts (n=20), including quarterly dosing, expansions and OLE will be presented.


Preliminary results demonstrate acceptable safety data and lowering of UOx in patients with PH1 supporting the continued development of lumasiran as a potential therapeutic to alleviate pathologic overproduction and consequences of excess oxalate in this devastating disease.


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