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000163389 1001_ $$aFeil, Katharina$$b0
000163389 245__ $$aSafety and Efficacy of Acetyl-DL-Leucine in Certain Types of Cerebellar Ataxia: The ALCAT Randomized Clinical Crossover Trial.
000163389 260__ $$aChicago, Ill.$$bAmerican Medical Association$$c2021
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000163389 520__ $$aCerebellar 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%) were female; the mean (SD) age was 54.8 (14.4) years; and the mean (SD) SARA total score was 13.33 (5.57) points. The full analysis set included 105 patients (80 patients with hereditary, 25 with nonhereditary or unknown cerebellar ataxia). There was no evidence of a difference in the mean absolute change from baseline to week 6 in SARA total scores between both treatments (mean treatment difference: 0.23 points [95% CI, -0.40 to 0.85 points]).In this large multicenter, double-blind, randomized, placebo-controlled clinical crossover trial, acetyl-DL-leucine in the investigated dosage and treatment duration was not superior to placebo for the symptomatic treatment of certain types of ataxia. The drug was well tolerated; and ALCAT yielded valuable information about the duration of treatment periods and the role of placebo response in cerebellar ataxia. These findings suggest that further symptom-oriented trials are needed for evaluating the long-term effects of acetyl-DL-leucine for well-defined subgroups of cerebellar ataxia.EudraCT 2015-000460-34.
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000163389 650_7 $$0GMW67QNF9C$$2NLM Chemicals$$aLeucine
000163389 650_7 $$0K76S41V71X$$2NLM Chemicals$$aacetylleucine
000163389 650_2 $$2MeSH$$aAdministration, Oral
000163389 650_2 $$2MeSH$$aAdult
000163389 650_2 $$2MeSH$$aAged
000163389 650_2 $$2MeSH$$aCerebellar Ataxia: classification
000163389 650_2 $$2MeSH$$aCerebellar Ataxia: drug therapy
000163389 650_2 $$2MeSH$$aCross-Over Studies
000163389 650_2 $$2MeSH$$aDouble-Blind Method
000163389 650_2 $$2MeSH$$aDrug Administration Schedule
000163389 650_2 $$2MeSH$$aFemale
000163389 650_2 $$2MeSH$$aHumans
000163389 650_2 $$2MeSH$$aLeucine: administration & dosage
000163389 650_2 $$2MeSH$$aLeucine: analogs & derivatives
000163389 650_2 $$2MeSH$$aMale
000163389 650_2 $$2MeSH$$aMiddle Aged
000163389 650_2 $$2MeSH$$aTreatment Outcome
000163389 7001_ $$aAdrion, Christine$$b1
000163389 7001_ $$aBoesch, Sylvia$$b2
000163389 7001_ $$aDoss, Sarah$$b3
000163389 7001_ $$0P:(DE-2719)2811662$$aGiordano, Ilaria$$b4$$udzne
000163389 7001_ $$0P:(DE-2719)2811940$$aHengel, Holger$$b5$$udzne
000163389 7001_ $$0P:(DE-2719)2811564$$aJacobi, Heike$$b6$$udzne
000163389 7001_ $$0P:(DE-2719)2810314$$aKlockgether, Thomas$$b7$$udzne
000163389 7001_ $$0P:(DE-2719)2810704$$aKlopstock, Thomas$$b8$$udzne
000163389 7001_ $$aNachbauer, Wolfgang$$b9
000163389 7001_ $$0P:(DE-2719)2810795$$aSchöls, Ludger$$b10$$udzne
000163389 7001_ $$aSteiner, Katharina Marie$$b11
000163389 7001_ $$0P:(DE-2719)2812141$$aStendel, Claudia$$b12$$udzne
000163389 7001_ $$aTimmann, Dagmar$$b13
000163389 7001_ $$aNaumann, Ivonne$$b14
000163389 7001_ $$aMansmann, Ulrich$$b15
000163389 7001_ $$aStrupp, Michael$$b16
000163389 7001_ $$aGroup, ALCAT Study$$b17$$eCollaboration Author
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