TY  - JOUR
AU  - Feil, Katharina
AU  - Adrion, Christine
AU  - Boesch, Sylvia
AU  - Doss, Sarah
AU  - Giordano, Ilaria
AU  - Hengel, Holger
AU  - Jacobi, Heike
AU  - Klockgether, Thomas
AU  - Klopstock, Thomas
AU  - Nachbauer, Wolfgang
AU  - Schöls, Ludger
AU  - Steiner, Katharina Marie
AU  - Stendel, Claudia
AU  - Timmann, Dagmar
AU  - Naumann, Ivonne
AU  - Mansmann, Ulrich
AU  - Strupp, Michael
TI  - Safety and Efficacy of Acetyl-DL-Leucine in Certain Types of Cerebellar Ataxia: The ALCAT Randomized Clinical Crossover Trial.
JO  - JAMA network open
VL  - 4
IS  - 12
SN  - 2574-3805
CY  - Chicago, Ill.
PB  - American Medical Association
M1  - DZNE-2022-00151
SP  - e2135841
PY  - 2021
N1  - (CC BY)
AB  - 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
KW  - Administration, Oral
KW  - Adult
KW  - Aged
KW  - Cerebellar Ataxia: classification
KW  - Cerebellar Ataxia: drug therapy
KW  - Cross-Over Studies
KW  - Double-Blind Method
KW  - Drug Administration Schedule
KW  - Female
KW  - Humans
KW  - Leucine: administration & dosage
KW  - Leucine: analogs & derivatives
KW  - Male
KW  - Middle Aged
KW  - Treatment Outcome
KW  - Leucine (NLM Chemicals)
KW  - acetylleucine (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:34905009
C2  - pmc:PMC8672236
DO  - DOI:10.1001/jamanetworkopen.2021.35841
UR  - https://pub.dzne.de/record/163389
ER  -