Research Summary

Lumasiran Shows Efficacy, Plasma Oxalate Reductions for Patients With Primary Hyperoxaluria, Severe Renal Impairment at 24 Months

Anthony Calabro, MA

Follow-up data at 24 months from the ongoing phase 3 ILLUMINATE-C trial showed reduced plasma oxalate levels among patients with advanced primary hyperoxaluria type 1 (PH1) and severe renal impairment. Researchers presented their findings at the 2023 American Society of Nephrology (ASN) Kidney Week in Philadelphia, PA.

The ongoing, single arm, open-label, phase 3 study had an initial 6-month primary analysis period, followed by an extended 54-month dosing period. This was done to evaluate the safety and efficacy of lumasiran in patients with PH1 and severe renal impairment (defined as an estimated glomerular filtration rate of 45 mL/min/1.73m2 or less and a plasma oxalate of 20 umol/L or less).

For the study, researchers assigned patients to cohorts based on hemodialysis (HD) treatment status. Cohort A included patients who did not receive HD (n = 5). Cohort B included those who received HD (n = 12).

>> Research Summary: New Oral Manifestation of Primary Hyperoxaluria

At 24 months, researchers found reduced plasma oxalate levels compared with baseline levels among patients in both cohorts. The mean reduction was 60.5% for Cohort A and 30.6% for Cohort B. Although four patients underwent a kidney transplant (two patients underwent a kidney only transplant whereas the other two underwent a combined liver-kidney transplant), all four patients maintained their treatment with a functional kidney graft.

“Plasma oxalate reductions with lumasiran were maintained at 24 months with an acceptable safety profile in patients with PH1 and chronic kidney disease 3b–5, including those on HD, and in patients with an isolated kidney transplant,” the researchers concluded.

The 2024 ASN Kidney Week will be held on October 23 through October 27, 2024, in San Diego, CA.

Lieske JC, Magen D, Sellier-Leclerc, A-L, et al. Lumasiran for primary hyperoxaluria type 1 and impaired kidney function: 24-month analysis of the phase 3 ILLUMINATE-C trial. Poster presented at: 2023 American Society of Nephrology Kidney Week; November 2-5, 2023; Philadelphia, PA. Accessed May 14, 2024.