%0 Journal Article
%A Feil, Katharina
%A Adrion, Christine
%A Boesch, Sylvia
%A Doss, Sarah
%A Giordano, Ilaria
%A Hengel, Holger
%A Jacobi, Heike
%A Klockgether, Thomas
%A Klopstock, Thomas
%A Nachbauer, Wolfgang
%A Schöls, Ludger
%A Steiner, Katharina Marie
%A Stendel, Claudia
%A Timmann, Dagmar
%A Naumann, Ivonne
%A Mansmann, Ulrich
%A Strupp, Michael
%T Safety and Efficacy of Acetyl-DL-Leucine in Certain Types of Cerebellar Ataxia: The ALCAT Randomized Clinical Crossover Trial.
%J JAMA network open
%V 4
%N 12
%@ 2574-3805
%C Chicago, Ill.
%I American Medical Association
%M DZNE-2022-00151
%P e2135841
%D 2021
%Z (CC BY)
%X Cerebellar ataxia is a neurodegenerative disease impairing motor function characterized by ataxia of stance, gait, speech, and fine motor disturbances.To investigate the efficacy, safety, and tolerability of the modified essential amino acid acetyl-DL-leucine in treating patients who have cerebellar ataxia.The Acetyl-DL-leucine on Cerebellar Ataxia (ALCAT) trial was an investigator-initiated, multicenter, double-blind, randomized, placebo-controlled, clinical crossover trial. The study was conducted at 7 university hospitals in Germany and Austria between January 25, 2016, and February 17, 2017. Patients were aged at least 18 years and diagnosed with cerebellar ataxia of hereditary (suspected or genetically confirmed) or nonhereditary or unknown type presenting with a total score of at least 3 points on the Scale for the Assessment and Rating of Ataxia (SARA). Statistical analysis was performed from April 2018 to June 2018 and January 2020 to March 2020.Patients were randomly assigned (1:1) to receive acetyl-DL-leucine orally (5 g per day after 2 weeks up-titration) followed by a matched placebo, each for 6 weeks, separated by a 4-week washout, or vice versa. The randomization was done via a web-based, permuted block-wise randomization list (block size, 2) that was stratified by disease subtype (hereditary vs nonhereditary or unknown) and site.Primary efficacy outcome was the absolute change of SARA total score from (period-dependent) baseline to week 6.Among 108 patients who were randomly assigned to sequence groups (54 patients each), 55 (50.9
%K Administration, Oral
%K Adult
%K Aged
%K Cerebellar Ataxia: classification
%K Cerebellar Ataxia: drug therapy
%K Cross-Over Studies
%K Double-Blind Method
%K Drug Administration Schedule
%K Female
%K Humans
%K Leucine: administration & dosage
%K Leucine: analogs & derivatives
%K Male
%K Middle Aged
%K Treatment Outcome
%K Leucine (NLM Chemicals)
%K acetylleucine (NLM Chemicals)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:34905009
%2 pmc:PMC8672236
%R 10.1001/jamanetworkopen.2021.35841
%U https://pub.dzne.de/record/163389