Background: Cerebellar ataxia (CA) is a frequent and often disabling condition that impairs motor functioning and impacts\non quality of life (QoL). No medication has yet been proven effective for the symptomatic or even causative treatment of\nhereditary or non-hereditary, non-acquired CA. So far, the only treatment recommendation is physiotherapy. Therefore, new\ntherapeutic options are needed. Based on three observational studies, the primary objective of the acetyl-DL-leucine on\nataxia (ALCAT) trial is to examine the efficacy and tolerability of a symptomatic therapy with acetyl-DL-leucine compared to\nplacebo on motor function measured by the Scale for the Assessment and Rating of Ataxia (SARA) in patients with CA.\nMethods/Design: An investigator-initiated, multicenter, European, randomized, double-blind, placebo-controlled, 2-\ntreatment 2-period crossover phase III trial will be carried out. In total, 108 adult patients who meet the clinical criteria\nof CA of different etiologies (hereditary or non-hereditary, non-acquired) presenting with a SARA total score of at least 3\npoints will be randomly assigned in a 1:1 ratio to one of two different treatment sequences, either acetyl-DL-leucine (up\nto 5 g per day) followed by placebo or vice versa. Each sequence consists of two 6-week treatment periods, separated by\na 4-week wash-out period. A follow-up examination is scheduled 4 weeks after the end of treatment. The primary efficacy\noutcome is the absolute change in the SARA total score. Secondary objectives are to demonstrate that acetyl-DL-leucine\nis effective in improving (1) motor function measured by the Spinocerebellar Ataxia Functional Index (SCAFI) and SARA\nsubscore items and (2) QoL (EuroQoL 5 dimensions and 5 level version, EQ-5D-5 L), depression (Beck Depression Inventory,\nBDI-II) and fatigue (Fatigue Severity Score, FSS). Furthermore, the incidence of adverse events will be investigated.\nDiscussion: The results of this trial will inform whether symptomatic treatment with the modified amino-acid acetyl-DLleucine\nis a worthy candidate for a new drug therapy to relieve ataxia symptoms and to improve patient care. If superiority\nof the experimental drug to placebo can be established it will also be re-purposing of an agent that has been previously\nused for the symptomatic treatment of dizziness.
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