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Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial

Silveira, C R A ; MacKinley, J ; Coleman, K ; Li, Z ; Finger, E ; Bartha, R ; Morrow, S A ; Wells, J ; Borrie, M ; Tirona, R G ; Rupar, C A ; Zou, G ; Hegele, R A ; Mahuran, D ; MacDonald, P ; Jenkins, M E ; Jog, M ; Pasternak, S H

BMC neurology, 2019-02, Vol.19 (1), p.20-20, Article 20 [Periódico revisado por pares]

England: BioMed Central Ltd

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Título:
Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial
Autor: Silveira, C R A ; MacKinley, J ; Coleman, K ; Li, Z ; Finger, E ; Bartha, R ; Morrow, S A ; Wells, J ; Borrie, M ; Tirona, R G ; Rupar, C A ; Zou, G ; Hegele, R A ; Mahuran, D ; MacDonald, P ; Jenkins, M E ; Jog, M ; Pasternak, S H
Assuntos: Alzheimer's disease ; Ambroxol ; Analysis ; Animal models ; Brain ; Cell culture ; Cerebrospinal fluid ; Clinical trials ; Cognition ; Cognition & reasoning ; Cognitive ability ; Cortex ; Dementia ; Dementia disorders ; Development and progression ; Diagnostic imaging ; Double-blind studies ; Drug dosages ; Drug therapy ; Enzymes ; Expectorants ; Gait ; Genes ; Genetic research ; Glucocerebrosidase ; Glucosylceramidase ; Kinematics ; Lymphocytes ; Magnetic resonance imaging ; Medical screening ; Movement disorders ; Mutation ; Neurodegeneration ; Neurodegenerative diseases ; Neuroimaging ; Parkinson disease ; Parkinson's disease ; Parkinson’s disease dementia ; Pharmacodynamics ; Pharmacokinetics ; Plasma levels ; Proteins ; Psychiatry ; Quality of life ; Risk factors ; Studies ; Study Protocol ; Synuclein ; Titration ; α-Synuclein
É parte de: BMC neurology, 2019-02, Vol.19 (1), p.20-20, Article 20
Notas: ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-1
ObjectType-News-3
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Descrição: Currently there are no disease-modifying treatments for Parkinson's disease dementia (PDD), a condition linked to aggregation of the protein α-synuclein in subcortical and cortical brain areas. One of the leading genetic risk factors for Parkinson's disease is being a carrier in the gene for β-Glucocerebrosidase (GCase; gene name GBA1). Studies in cell culture and animal models have shown that raising the levels of GCase can decrease levels of α-synuclein. Ambroxol is a pharmacological chaperone for GCase and is able to raise the levels of GCase and could therefore be a disease-modifying treatment for PDD. The aims of this trial are to determine if Ambroxol is safe and well-tolerated by individuals with PDD and if Ambroxol affects cognitive, biochemical, and neuroimaging measures. This is a phase II, single-centre, double-blind, randomized placebo-controlled trial involving 75 individuals with mild to moderate PDD. Participants will be randomized into Ambroxol high-dose (1050 mg/day), low-dose (525 mg/day), or placebo treatment arms. Assessments will be undertaken at baseline, 6-months, and 12-months follow up times. Primary outcome measures will be the Alzheimer's disease Assessment Scale-cognitive subscale (ADAS-Cog) and the ADCS Clinician's Global Impression of Change (CGIC). Secondary measures will include the Parkinson's disease Cognitive Rating Scale, Clinical Dementia Rating, Trail Making Test, Stroop Test, Unified Parkinson's disease Rating Scale, Purdue Pegboard, Timed Up and Go, and gait kinematics. Markers of neurodegeneration will include MRI and CSF measures. Pharmacokinetics and pharmacodynamics of Ambroxol will be examined through plasma levels during dose titration phase and evaluation of GCase activity in lymphocytes. If found effective and safe, Ambroxol will be one of the first disease-modifying treatments for PDD. ClinicalTrials.gov NCT02914366, 26 Sep 2016/retrospectively registered.
Editor: England: BioMed Central Ltd
Idioma: Inglês

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Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial

Silveira, C R A ; MacKinley, J ; Coleman, K ; Li, Z ; Finger, E ; Bartha, R ; Morrow, S A ; Wells, J ; Borrie, M ; Tirona, R G ; Rupar, C A ; Zou, G ; Hegele, R A ; Mahuran, D ; MacDonald, P ; Jenkins, M E ; Jog, M ; Pasternak, S H

England: BioMed Central Ltd

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