mTOR Inhibition with Sirolimus in Multiple System Atrophy: A Randomized, Double-Blind, Placebo-Controlled Futility Trial and 1-Year Biomarker Longitudinal Analysis.

Jose Alberto PalmaJose MartinezPatricio Millar VernettiThong MaMiguel A PerezJudy ZhongYingzhi QianSuman DuttaKatherine N MainaIbrar SiddiqueGal BitanBenjamin Ades-AronTimothy M ShepherdUn Jung Kang Horacio Kaufmann

Published in: Movement disorders : official journal of the Movement Disorder Society (2022)

Sirolimus for 48 weeks was futile to slow the progression of MSA and had no effect on biomarkers compared to placebo. One-year change in blood NfL and whole brain atrophy are promising biomarkers of disease progression for future clinical trials. © 2022 International Parkinson and Movement Disorder Society.

Keyphrases

double blind
placebo controlled
clinical trial
phase iii
phase ii
study protocol
open label
cell proliferation
current status
phase ii study
white matter
randomized controlled trial
resting state
cross sectional
blood brain barrier
multiple sclerosis
gestational age