Ambroxol as a Treatment for Parkinson's Disease Dementia
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ClinicalTrials.gov Identifier: NCT02914366 |
Recruitment Status : Unknown
Verified June 2020 by Stephen Pasternak, University of Western Ontario, Canada.
Recruitment status was: Recruiting
First Posted : September 26, 2016
Last Update Posted : June 22, 2020
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Condition or disease | Intervention/treatment | Phase |
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Parkinson's Disease Dementia | Drug: Ambroxol Other: Placebo | Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 75 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Ambroxol as a Novel Disease Modifying Treatment for Parkinson's Disease Dementia |
Study Start Date : | November 2015 |
Estimated Primary Completion Date : | July 2021 |
Estimated Study Completion Date : | December 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Ambroxol high dose (1050 mg)
Participants randomized to the 1050 mg/day group will begin with a dose of 225mg (3 mg/kg/day), increasing bi-weekly by ~3mg/kg to a dose of 1050 mg/day (~l5 mg/kg/day).
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Drug: Ambroxol
Other Name: Mucosolvon |
Placebo Comparator: Placebo
Participants receive capsules visually identical to the experimental groups but without active ingredients.
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Other: Placebo |
- Changes in the Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-cog) [ Time Frame: baseline, week 26, and week 52 ]This 70-point test examines language, recall, word finding, comprehension, naming, drawing, praxis, orientation, and word recognition. Although designed for Alzheimer's disease where it is considered a gold standard, the ADAS-Cog has been used effectively in many clinical trials of PDD including large randomized trials. This scale has been recommended for the assessment of Parkinson's dementia in "Diagnostic Procedures for Parkinson's Disease Dementia: Recommendations from the Movement Disorder Society Task Force"
- Changes in the ADCS-Clinician's Global Impression of Change (CGIC) [ Time Frame: baseline, week 26, and week 52 ]This is a 7-point scale for rating patient function in cognition behavior and activities of daily living, and this test is standard in clinical trials in Alzheimer's disease and has been useful in trials with PDD.
- Changes in the Montreal Cognitive Assessment [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Clinical Dementia Rating Scale (CDR) [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Trail Making Test (TRAILS) [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Parkinson's Disease-Cognitive Rating Scale (PD-CRS) [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Stroop Test [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Unified Parkinson's disease Rating Scale motor subsection (UPDRS-III) [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Purdue Pegboard [ Time Frame: baseline, week 26, and week 52 ]
- Changes in the Timed Up and Go [ Time Frame: baseline, week 26, and week 52 ]
- Change in Quantitative Movement Testing [ Time Frame: baseline, week 26, and week 52 ]gait assessment on electronic mat (Zeno Walkway System)
- Changes in Cerebrospinal Fluid (CSF) biomarkers [ Time Frame: baseline, week 12, and week 52 ]levels of α-synuclein (pg/ml), Tau (pg/ml), phospho-Tau (pg/ml) and beta amyloid-42 (pg/ml)
- Changes in Magnetic Resonance Imaging (MRI) [ Time Frame: baseline and week 52 ]brain ventricle volume (cm3) and hippocampal atrophy (cm3)
- Changes in the Mini-Mental State Examination [ Time Frame: screening, baseline, week 4, week 6, week 12, week 18, week 26, week 34, week 42, week 52 ]
- Changes in GCAse in lymphocytes [ Time Frame: baseline, week 2, week 4, week 6, week 8, week 12, week 18, week 26, week 34, week 42, week 52 ]from blood sample
- Changes in Plasma Ambroxol levels [ Time Frame: baseline, week 2, week 4, week 6, week 8, week 12, week 18, week 26, week 34, week 42, week 52 ]from blood sample
- Mayo Fluctuation Questionnaire [ Time Frame: baseline, week 26, and week 52 ]

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Ages Eligible for Study: | 50 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age >50,
- Mild to Moderate Dementia (established by an upper cut off of a Montreal Cognitive Assessment score of 24 or below, and the lower bound by a Mini Mental State Exam of 16 or greater),
- Parkinson's Disease (Hoehn & Yahr stage 2 - 3.5) clearly established more than 1 year before the onset of dementia
- Patients must have a responsible caregiver = 4 days/wk
- Must be on stable doses of medications for Parkinson's disease mood and cognition (Cholinesterase Inhibitor) for at least 3 months prior to the study.
Exclusion Criteria:
- Evidence of clinically significant stroke or other neurological condition
- Any other serious underlying condition (i.e. cancer or unstable cardiac disease etc.
- Concurrent treatment with oral anticoagulants (including Vitamin K agonists and Novel Oral Anticoagulants (NOACs)) within 4 weeks of screening or anticipated during the 52 week double-blind and open label periods. Specifically, Apixaban, Dabigatran, Edoxaban, Fondaparinux, Rivaroxaban, and Warfarin are prohibited concomitant medications.
3.1 Exceptions: antiplatelet agents such as Aspirin, Clopidogrel, and Aggrenox are allowed.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02914366
Contact: Stephen Pasternak, M.D., Ph.D. | +1 519-646-6000 ext 66032 | spasternak@robarts.ca | |
Contact: Carolina Silveira, Ph.D | +1 519-656-6100 ext 42367 | Carolina.Silveira@sjhc.london.ca |
Canada, Ontario | |
Parkwood Institute | Recruiting |
London, Ontario, Canada, N6C 0A7 | |
Contact: Carolina Silveira, Ph.D 519-646-6100 ext 42367 Carolina.Silveira@sjhc.london.on.ca | |
Contact: Tommy Li, MSc 519-646-6100 ext 42711 Zhonghan.Li@sjhc.london.on.ca |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Stephen Pasternak, M.D., Ph.D., University of Western Ontario, Canada |
ClinicalTrials.gov Identifier: | NCT02914366 |
Other Study ID Numbers: |
R15-006 105234 ( Other Identifier: Western University Health Science Research Ethics Board (HSREB) ) 181033 ( Other Identifier: Health Canada ) |
First Posted: | September 26, 2016 Key Record Dates |
Last Update Posted: | June 22, 2020 |
Last Verified: | June 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Peer reviewed publication | Presentation |
Ambroxol Dementia Parkinson's disease |
Disease modifying treatment Pharmacological Chaperone GBA1 |
Parkinson Disease Dementia Parkinsonian Disorders Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Movement Disorders |
Synucleinopathies Neurodegenerative Diseases Neurocognitive Disorders Mental Disorders Ambroxol Expectorants Respiratory System Agents |