Effects of Acetyl-DL-Leucine on Cerebellar Ataxia (Study Protocol)

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Effects of Acetyl-DL-Leucine on Cerebellar Ataxia (Study Protocol)

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Create DateJuly 3, 2018
Last UpdatedJuly 16, 2018

Effects of acetyl-DL-leucine on cerebellar ataxia (ALCAT trial): study protocol for a multicenter, multinational, randomized, double-blind, placebo-controlled, crossover phase III trial

Katharina Feil1,2,11* , Christine Adrion3, Julian Teufel1,2, Sylvia Bösch8, Jens Claassen5, Ilaria Giordano7, Holger Hengel6, Heike Jacobi7, Thomas Klockgether7, Thomas Klopstock1,4,12, Wolfgang Nachbauer8, Ludger Schöls6, Claudia Stendel1,4, Ellen Uslar5, Bart van de Warrenburg9, Ingrid Berger2, Ivonne Naumann2, Otmar Bayer2, Hans-Helge Müller10, Ulrich Mansmann3 and Michael Strupp1,2

Background: Cerebellar ataxia (CA) is a frequent and often disabling condition that impairs motor functioning and impacts on quality of life (QoL). No medication has yet been proven effective for the symptomatic or even causative treatment of hereditary or non-hereditary, non-acquired CA. So far, the only treatment recommendation is physiotherapy. Therefore, new therapeutic options are needed. Based on three observational studies, the primary objective of the acetyl-DL-leucine on ataxia (ALCAT) trial is to examine the efficacy and tolerability of a symptomatic therapy with acetyl-DL-leucine compared to placebo on motor function measured by the Scale for the Assessment and Rating of Ataxia (SARA) in patients with CA.

The results of this trial will inform whether symptomatic treatment with the modified amino-acid acetyl-DL- leucine is a worthy candidate for a new drug therapy to relieve ataxia symptoms and to improve patient care. If superiority of the experimental drug to placebo can be established it will also be re-purposing of an agent that has been previously used for the symptomatic treatment of dizziness.