Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial.

Wesley HayesDavid J SasDaniella MagenHadas Shasha-LavskyMini MichaelAnne-Laure Sellier-LeclercJulien HoganTaylor NgoMarianne T SweetserJohn M GansnerTracy L McGregorYaacov Frishberg

Published in: Pediatric nephrology (Berlin, Germany) (2022)

Lumasiran treatment provided sustained reductions in urinary and plasma oxalate through month 12 across all weight subgroups, with an acceptable safety profile, in infants and young children with PH1. A higher resolution version of the Graphical abstract is available as Supplementary information.

Keyphrases

body mass index
clinical trial
study protocol
weight loss
phase ii
weight gain
single molecule
smoking cessation
double blind