IB1001 Pivotal Trial Published in NEJM

In this double-blind, placebo-controlled, crossover trial, we randomly assigned patients 4 years of age or older with genetically confirmed Niemann–Pick disease type C in a 1:1 ratio to receive N-acetyl-L-leucine (NALL) for 12 weeks, followed by placebo for 12 weeks, or to receive placebo for 12 weeks, followed by NALL for 12 weeks. NALL met its primary SARA endpoint (least-squares mean difference, −1.28 points; 95% confidence interval, −1.91 to −0.65; P<0.001) and its secondary endpoints. NALL was well-tolerated, with no serious adverse reactions. N Engl J Med 2024; 390:421-431

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