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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 CRA, MacKinley J, Coleman K, Li Z, Finger E, Bartha R, Morrow SA, Wells J et al.. BMC neurology, 2019-02-09
Topic: Amyloid, tau and blood-test diagnosis
Background 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.
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APA 7th
Silveira, C. R. A., MacKinley, J., Coleman, K., Li, Z., Finger, E., Bartha, R., et al. (2019). Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial. BMC neurology. https://doi.org/10.1186/s12883-019-1252-3
Vancouver
Silveira CRA, MacKinley J, Coleman K, Li Z, Finger E, Bartha R, et al. Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial. BMC neurology. 2019. doi:10.1186/s12883-019-1252-3. PMID: 30738426.
BibTeX
@article{silveira2019-30738426,
author = {Silveira, C. R. A. and MacKinley, J. and Coleman, K. and Li, Z. and Finger, E. and Bartha, R. and Morrow, S. A. and Wells, J. and others},
title = {{Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial}},
journal = {BMC neurology},
year = {2019},
doi = {10.1186/s12883-019-1252-3},
pmid = {30738426},
url = {https://doi.org/10.1186/s12883-019-1252-3}
}
Built from the record alzhub holds for this paper, not looked up. Volume, issue and page numbers are not held in the record. The record's author list ends in “et al.”, so not every author is named. Check the citation against the original before you use it.
Key findings
- 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.
Why it matters
This is a protocol for a trial testing whether Ambroxol is safe and well tolerated in Parkinson's disease dementia, based on cell and animal studies showing it can raise GCase levels and lower α-synuclein. It is a planned trial, so no results on treating, curing or preventing dementia are yet available.
Study details
- Participants75
- Evidence strengthHigh
How this study scored 55/100
Published in a peer-reviewed journal. Scored zero for study design and effect reported because the record does not state it.
| Component | Points | Why |
|---|---|---|
| Journal tier | 7 / 10 | Tier 3 — peer-reviewed journal (BMC neurology) |
| Journal's citation record | 1 / 4 | BMC Neurology: journal h-index 122 (OpenAlex, 2026-10-29) |
| Study design | 0 / 12 | No study design recorded — scores zero, not guessed (not stated in the record — scored 0) |
| Strength of evidence | 5 / 5 | Rated “high” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 2 / 5 | 2019 — within 10 year(s) of the newest study here |
| Sample size | 1 / 4 | 75 participants |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 3 / 3 | Studied in people (participant count stated) |
| Randomised / blinded | 2 / 2 | Randomised and blinded or placebo-controlled (as stated in the record) |
| Times cited | 5 / 7 | Cited 137 times (OpenAlex, 2026-10-10) |
| Citations per year | 3 / 5 | 15.2 citations a year over 9 year(s) since 2019 |
| Impact for its field and year | 4 / 6 | Cited 11.5× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 1 / 6 | Cited by 1 other studies in this library |
| Still being cited | 2 / 4 | Cited 37 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 2 / 4 | Last-listed author Stephen Pasternak: h-index 48 (OpenAlex, whole career) |
| Institution's track record | 1 / 3 | Western University: institution h-index 899 (OpenAlex) |
| Multi-centre collaboration | 2 / 3 | OpenAlex lists 5 institution(s) in 1 country |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 30738426) |
| Free to read | 2 / 2 | Free to read |
| Total | 55 / 100 | — |
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