Cargando…

Safety and Efficacy of Acetyl-DL-Leucine in Certain Types of Cerebellar Ataxia: The ALCAT Randomized Clinical Crossover Trial

IMPORTANCE: Cerebellar ataxia is a neurodegenerative disease impairing motor function characterized by ataxia of stance, gait, speech, and fine motor disturbances. OBJECTIVE: To investigate the efficacy, safety, and tolerability of the modified essential amino acid acetyl-DL-leucine in treating pati...

Descripción completa

Detalles Bibliográficos
Autores principales:	Feil, Katharina, Adrion, Christine, Boesch, Sylvia, Doss, Sarah, Giordano, Ilaria, Hengel, Holger, Jacobi, Heike, Klockgether, Thomas, Klopstock, Thomas, Nachbauer, Wolfgang, Schöls, Ludger, Steiner, Katharina Marie, Stendel, Claudia, Timmann, Dagmar, Naumann, Ivonne, Mansmann, Ulrich, Strupp, Michael
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Medical Association 2021
Materias:	Original Investigation
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8672236/ https://www.ncbi.nlm.nih.gov/pubmed/34905009 http://dx.doi.org/10.1001/jamanetworkopen.2021.35841

Ejemplares similares

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
por: Feil, Katharina, et al.
Publicado: (2017)

Effects of acetyl-dl-leucine in patients with cerebellar ataxia: a case series
por: Strupp, Michael, et al.
Publicado: (2013)

Acetyl-DL-leucine improves gait variability in patients with cerebellar ataxia—a case series
por: Schniepp, Roman, et al.
Publicado: (2016)

Acetyl-DL-leucine in combination with memantine improves acquired pendular nystagmus caused by multiple sclerosis: a case report
por: Kremmyda, O., et al.
Publicado: (2023)

Aminopyridines and Acetyl-DL-leucine: New Therapies in Cerebellar Disorders
por: Kalla, Roger, et al.
Publicado: (2019)

Acetyl-dl-leucine in cerebellar ataxia ([(18)F]-FDG-PET study): how does a cerebellar disorder influence cortical sensorimotor networks?
por: Becker-Bense, Sandra, et al.
Publicado: (2022)

Mast Syndrome Outside the Amish Community: SPG21 in Europe
por: Amprosi, Matthias, et al.
Publicado: (2022)

Beneficial Effects of Acetyl-DL-Leucine (ADLL) in a Mouse Model of Sandhoff Disease
por: Kaya, Ecem, et al.
Publicado: (2020)

Unexpected differences in the pharmacokinetics of N-acetyl-DL-leucine enantiomers after oral dosing and their clinical relevance
por: Churchill, Grant C., et al.
Publicado: (2020)

Update cerebellum and cognition
por: Jacobi, Heike, et al.
Publicado: (2021)

Longitudinal changes of SARA scale in Friedreich ataxia: Strong influence of baseline score and age at onset
por: Porcu, Luca, et al.
Publicado: (2023)

Prediction of the disease course in Friedreich ataxia
por: Hohenfeld, Christian, et al.
Publicado: (2022)

Results and lessons learnt from a randomized controlled trial: prophylactic treatment of vestibular migraine with metoprolol (PROVEMIG)
por: Bayer, Otmar, et al.
Publicado: (2019)

Cardiolipin remodeling by ALCAT1 links mitochondrial dysfunction to Parkinson’s diseases
por: Song, Chengjie, et al.
Publicado: (2019)

Dual task effect on postural control in patients with degenerative cerebellar disorders
por: Jacobi, Heike, et al.
Publicado: (2015)

ALCAT1 Overexpression Affects Supercomplex Formation and Increases ROS in Respiring Mitochondria
por: Rieger, Bettina, et al.
Publicado: (2019)

Efficacy and safety of betahistine treatment in patients with Meniere’s disease: primary results of a long term, multicentre, double blind, randomised, placebo controlled, dose defining trial (BEMED trial)
por: Adrion, Christine, et al.
Publicado: (2016)

Control and Data Acquisition System for Lower Hybrid Current Drive in Alcator C-Mod
por: Basse, N P, et al.
Publicado: (2003)

Construction, Calibration and Testing of a Multi-Waveguide Array for the Application of LHCD on Alcator C-MOD
por: Wilson, J R, et al.
Publicado: (2003)

Optimized AT Target Plasma Studies in Alcator C-Mod with I$_{p}$-Ramp and Intense ICRH
por: Porkolab, M, et al.
Publicado: (2003)

The Roles of FGF21 and ALCAT1 in Aerobic Exercise-Induced Cardioprotection of Postmyocardial Infarction Mice
por: Bo, Wenyan, et al.
Publicado: (2021)

Onset features and time to diagnosis in Friedreich’s Ataxia
por: Indelicato, Elisabetta, et al.
Publicado: (2020)

Bayesian model selection techniques as decision support for shaping a statistical analysis plan of a clinical trial: An example from a vertigo phase III study with longitudinal count data as primary endpoint
por: Adrion, Christine, et al.
Publicado: (2012)

Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app
por: Buchholz, Maresa, et al.
Publicado: (2023)

Pharmacological inhibition of ALCAT1 mitigates amyotrophic lateral sclerosis by attenuating SOD1 protein aggregation
por: Liu, Xueling, et al.
Publicado: (2022)

4-Aminopyridine Improves Lower Urinary Tract Symptoms in a Patient With Benign Prostatic Hyperplasia and Downbeat Nystagmus Syndrome
por: Strupp, Michael, et al.
Publicado: (2014)

Cardioprotection Attributed to Aerobic Exercise-Mediated Inhibition of ALCAT1 and Oxidative Stress-Induced Apoptosis in MI Rats
por: Liu, Niu, et al.
Publicado: (2022)

Update on the pharmacotherapy of cerebellar and central vestibular disorders
por: Kalla, Roger, et al.
Publicado: (2016)

Conjugate Eye Deviation in Unilateral Lateral Medullary Infarction
por: Teufel, Julian, et al.
Publicado: (2019)

The gait disorder in primary orthostatic tremor
por: Möhwald, Ken, et al.
Publicado: (2020)

The frequency of non-motor symptoms in SCA3 and their association with disease severity and lifestyle factors
por: Hengel, Holger, et al.
Publicado: (2022)

Predictive Capability of an iPad-Based Medical Device (medx) for the Diagnosis of Vertigo and Dizziness
por: Feil, Katharina, et al.
Publicado: (2018)

An algorithm as a diagnostic tool for central ocular motor disorders, also to diagnose rare disorders
por: Kraus, Ludwig, et al.
Publicado: (2019)

Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA)
por: Reetz, Kathrin, et al.
Publicado: (2019)

Examination of betahistine bioavailability in combination with the monoamine oxidase B inhibitor, selegiline, in humans—a non-randomized, single-sequence, two-period titration, open label single-center phase 1 study (PK-BeST)
por: Strupp, Michael, et al.
Publicado: (2023)

Parental mosaicism in another case of Dravet syndrome caused by a novel SCN1A deletion: a case report
por: Sharkia, Rajech, et al.
Publicado: (2016)

Acetylation turns leucine into a drug by membrane transporter switching
por: Churchill, Grant C., et al.
Publicado: (2021)

A longitudinal VBM study monitoring treatment with erythropoietin in patients with Friedreich ataxia
por: Santner, Wolfram, et al.
Publicado: (2014)

PolyQ-expanded ataxin-3 protein levels in peripheral blood mononuclear cells correlate with clinical parameters in SCA3: a pilot study
por: Gonsior, Kathrin, et al.
Publicado: (2020)

No Evidence of a Contribution of the Vestibular System to Frequent Falls in Progressive Supranuclear Palsy
por: Goldschagg, Nicolina, et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_pmsglqq1oq4ggeohlrje5q1g3e