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ASN leads the fight to prevent, treat, and cure kidney diseases throughout the world by educating health professionals and scientists, advancing research and innovation, communicating new knowledge, and advocating for the highest quality care for patients.

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Abstract: INFO14-SA

ILLUMINATE-C: A Phase 3 Single-Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

Session Information

  • Informational Posters - III
    November 09, 2019 | Location: Exhibit Hall, Walter E. Washington Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Genetic Diseases of the Kidneys


  • Frishberg, Yaacov, Shaare Zedek Medical Center, Jerusalem, Israel
  • Cochat, Pierre, Université ClaudeBernard Lyon1-, Bron, France
  • Talamudupula, Sandeep, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Sama, Reddy, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • McGregor, Tracy, Alnylam Pharmaceuticals, Cambridge, Massachusetts, United States
  • Tamimi, Nihad, Medicopharma Solutions Limited, Canterbury, United Kingdom

Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder characterized by persistent hepatic overproduction of oxalate. Oxalate crystalizes with calcium leading to recurrent kidney stones, nephrocalcinosis, progressive renal failure, and multiorgan damage from systemic oxalosis. Lumasiran is a subcutaneously-administered investigational RNAi therapeutic specifically designed to decrease oxalate production. In the Phase 1/2 study, lumasiran demonstrated an acceptable safety profile and clinically significant urinary oxalate (UOx) lowering in patients with PH1. A Phase 3 Program, ILLUMINATE, consisting of 3 studies, is evaluating the efficacy and safety of lumasiran in patients with PH1; ILLUMINATE-C is the third study within this program.

ILLUMINATE-C, a multicenter, single arm study, is designed to evaluate the efficacy, safety, PK and PD of lumasiran in 16 patients with PH1 who have advanced renal disease (eGFR ≤45 ml/min/1.73 m2 or serum creatinine elevated for age, in patients <12 months of age) including those on dialysis. All eligible patients will be administered open-label lumasiran. This study will include two cohorts: Cohort A - patients who do not yet require dialysis and Cohort B - patients who are on dialysis. The primary endpoint of percent change in plasma oxalate from baseline will be evaluated after 6 months of treatment. Key secondary endpoints include percent change in plasma oxalate AUC between dialysis sessions, changes in quality of life, and plasma pharmacokinetic parameters of lumasiran.


  • Alnylam Pharmaceuticals