Source:Pediatric Neurology
Author(s): Daniel G. Glaze, Jeffrey. L. Neul, Alan Percy, Tim Feyma, Arthur Beisang, Alex Yaroshinsky, George Stoms, David Zuchero, Joseph Horrigan, Larry Glass, Nancy E. Jones
ObjectiveTo determine the safety and tolerability of trofinetide and to evaluate efficacy measures in adolescent and adult females with Rett syndrome, a serious and debilitating neurodevelopmental condition for which no therapies are available for its core features.MethodsThis was an exploratory, Phase 2, multicenter, double-blind, placebo-controlled, dose-escalation study of the safety and tolerability of trofinetide in 56 adolescent and adult females with Rett syndrome. Subjects were randomly assigned in a 2:1 ratio to 35 mg/kg b.i.d. of trofinetide or placebo for 14 days, 35 mg/kg b.i.d. or placebo for 28 days or 70 mg/kg b.i.d. or placebo for 28 days.Safety assessments included adverse events, clinical laboratory tests, vital signs, electrocardiograms, physical examinations and concomitant medications. Efficacy measurements were categorized into 4 efficacy domains, which related to clinically relevant, phenotypic dimensions of impairment associated with Rett syndrome.ResultsBoth 35 mg/kg and 70 mg/kg dose levels of trofinetide were well tolerated and generally safe. Trofinetide at 70 mg/kg demonstrated efficacy compared with placebo based on pre-specified criteria.InterpretationTrofinetide was well tolerated in adolescent and adult females with Rett syndrome. Although this was a relatively short duration study in a small number of subjects with an advanced stage of disease, consistent efficacy trends at the higher dose were observed in several outcome measures that assess important dimensions of Rett syndrome. These results represented clinically meaningful improvement from the perspective of the clinicians as well as the caregivers.
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