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
| Autor: | Wolfgang Nachbauer, Ilaria Giordano, Ingrid Berger, Jens Claassen, Christine Adrion, Ivonne Naumann, Otmar Bayer, Thomas Klopstock, Ulrich Mansmann, Bart P.C. van de Warrenburg, Thomas Klockgether, Claudia Stendel, Katharina Feil, Heike Jacobi, Julian Teufel, Michael Strupp, Sylvia Bösch, Hans-Helge Müller, Ludger Schöls, Holger Hengel, Ellen Uslar |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
30.260 Neurodegeneration Medizin Patient questionnaires law.invention 20.140 Qualitative research Study Protocol 0302 clinical medicine Randomized controlled trial law Ataxia rating scales Cerebellar ataxia Cross-Over Studies Symptomatic therapy General Medicine Disorders of movement Donders Center for Medical Neuroscience [Radboudumc 3] Tolerability Crossover design Spinocerebellar ataxia Amino acids analogs & derivatives [Leucine] therapeutic use [Leucine] 30.040 Ataxia medicine.symptom Adult Quality of life medicine.medical_specialty Ataxia Cerebellar Ataxia 20.060 Clinical trial Medizinische Fakultät » Universitätsklinikum Essen » Klinik für Neurologie Clinical Neurology Placebo drug therapy [Cerebellar Ataxia] 03 medical and health sciences Double-Blind Method Leucine Internal medicine medicine ddc:61 Humans Spinocerebellar Ataxias 10.070 Movement disorders ddc:610 Psychiatric Status Rating Scales business.industry acetylleucine Beck Depression Inventory Acetyl-DL-leucine medicine.disease Crossover study drug therapy [Spinocerebellar Ataxias] 030104 developmental biology Physical therapy Quality of Life Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | BMC Neurology BMC Neurology, 17, 7 BMC neurology 17(1), 7 (2017). doi:10.1186/s12883-016-0786-x BMC Neurology, 17, 1, pp. 7 |
| ISSN: | 1471-2377 0000-9733 |
| DOI: | 10.1186/s12883-016-0786-x |
| Popis: | 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. An investigator-initiated, multicenter, European, randomized, double-blind, placebo-controlled, 2-treatment 2-period crossover phase III trial will be carried out. In total, 108 adult patients who meet the clinical criteria of CA of different etiologies (hereditary or non-hereditary, non-acquired) presenting with a SARA total score of at least 3 points will be randomly assigned in a 1:1 ratio to one of two different treatment sequences, either acetyl-DL-leucine (up to 5 g per day) followed by placebo or vice versa. Each sequence consists of two 6-week treatment periods, separated by a 4-week wash-out period. A follow-up examination is scheduled 4 weeks after the end of treatment. The primary efficacy outcome is the absolute change in the SARA total score. Secondary objectives are to demonstrate that acetyl-DL-leucine is effective in improving (1) motor function measured by the Spinocerebellar Ataxia Functional Index (SCAFI) and SARA subscore items and (2) QoL (EuroQoL 5 dimensions and 5 level version, EQ-5D-5 L), depression (Beck Depression Inventory, BDI-II) and fatigue (Fatigue Severity Score, FSS). Furthermore, the incidence of adverse events will be investigated. 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. The trial was prospectively registered at www.clinicaltrialsregister.eu (EudraCT no. 2015–000460–34) and at https://www.germanctr.de (DRKS-ID: DRKS00009733 ). |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|
Full text from SpringerLink