Pritesh J. Gandhi, PharmD, Former Vice President and General Manager, Lumasiran Program at Alnylam Pharmaceuticals, discusses the results of the Illuminate-A study, a Phase 3 randomized, double-blind, placebo-controlled study that evaluated the efficacy and safety of lumasiran in children (age 6 or older) and adults with primary hyperoxaluria type 1 (PH1).
PH1 is a rare genetic disease in which excessive oxalate production leads to painful and recurrent kidney stones. These recurrent stones increase the chance of hematuria, urinary tract infections, and end stage renal disease.
The results of the Illuminate-A trial have been published in the New England Journal of Medicine.
As Dr. Gandhi explains, relative to placebo, treatment with lumasiran resulted in a clinically significant (53.5%) reduction in 24-hour urinary oxalate excretion from baseline to month 6 – the primary endpoint of the study. The results demonstrated patient improvements in a number of secondary endpoints, including the proportion of patients achieving normal or near-normal levels of urinary oxalate, with 84 percent of lumasiran-treated patients meeting this endpoint compared with no patients (0%) on placebo. Administration of lumasiran was associated with an encouraging safety and tolerability profile, with no serious or severe adverse events.
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