What side effects are associated with this type of intervention?

Common treatments for Parkinson's Disease, such as cholinesterase inhibitors (e.g., rivastigmine and donepezil), can cause side effects like nausea, vomiting, and worsened tremor. Dopaminergic therapies, including levodopa and dopamine agonists, may lead to nausea, dizziness, somnolence, hallucinations, and dyskinesia. Adjunctive treatments like MAO B inhibitors and amantadine can also cause side effects such as insomnia, livedo reticularis, and confusion. While Ambroxol is being studied for its potential benefits in PD by raising beta-glucocerebrosidase levels and lowering alpha-synuclein levels, its safety profile in this context is still under investigation.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically targeting the mechanisms of raising beta-glucocerebrosidase levels and lowering alpha-synuclein levels in Parkinson's Disease. Most FDA-approved treatments for Parkinson's Disease focus on symptomatic relief and include dopaminergic therapies such as carbidopa-levodopa, nonergot dopamine agonists (e.g., pramipexole, ropinirole), and MAO-B inhibitors (e.g., rasagiline, selegiline). These treatments aim to manage motor symptoms but do not modify the underlying disease process.

What other types of research exist?

Promising novel alternatives to Ambroxol for Parkinson's Disease patients include: 1) Pramipexole combined with levodopa, which has shown efficacy in relieving PD symptoms and improving quality of life by potentially suppressing serum TNF-α levels. 2) Nonergot dopamine agonists like ropinirole and transdermal rotigotine, which are effective in reducing motor symptoms and have a lower risk of dyskinesia compared to levodopa. 3) Cholinesterase inhibitors such as rivastigmine and donepezil, which have shown mild to moderate benefits in cognitive symptoms of PD dementia. 4) CX-8998, a calcium channel modulator, currently being evaluated for PD tremor. These alternatives offer different mechanisms and potential benefits for PD patients.

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Mucolytic Agent

Ambroxol for Parkinson's Disease Dementia

Phase 2

Waitlist Available

Research Sponsored by Lawson Health Research Institute

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 2, week 4, week 6, week 8, week 12, week 18, week 26, week 34, week 42, week 52

Awards & highlights

All Individual Drugs Already Approved

Summary

This trial tests if Ambroxol, a safe over-the-counter medication, can help people with Parkinson's Disease Dementia by increasing an enzyme that reduces harmful proteins in the brain. The goal is to see if it improves memory and movement problems. Ambroxol has been shown to be safe and well tolerated in previous studies.

Who is the study for?

This trial is for individuals over 50 with Parkinson's Disease Dementia (PDD), who have mild to moderate dementia and a caregiver available at least 4 days a week. Participants must be on stable Parkinson's medication for three months, without serious conditions like significant strokes or cancer, and not on oral anticoagulants.

What is being tested?

The study tests if Ambroxol can improve cognitive and motor symptoms in PDD by increasing beta-glucocerebrosidase levels, potentially lowering alpha-synuclein protein. This year-long trial involves clinical assessments, neuropsychological testing, and neuroimaging to monitor changes.

What are the potential side effects?

While the side effects of Ambroxol in this context are being studied, it may include gastrointestinal discomforts such as nausea or diarrhea, headache, dizziness or allergic reactions. The severity of these side effects will be closely monitored throughout the trial.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 2, week 4, week 6, week 8, week 12, week 18, week 26, week 34, week 42, week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 2, week 4, week 6, week 8, week 12, week 18, week 26, week 34, week 42, week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 2, week 4, week 6, week 8, week 12, week 18, week 26, week 34, week 42, week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Changes in the ADCS-Clinician's Global Impression of Change (CGIC)

Changes in the Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-cog)

Secondary study objectives

Change in Quantitative Movement Testing

Changes in Cerebrospinal Fluid (CSF) biomarkers

Changes in GCAse in lymphocytes

+2 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Ambroxol high dose (1050 mg)Experimental Treatment1 Intervention

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).

Group II: PlaceboPlacebo Group1 Intervention

Participants receive capsules visually identical to the experimental groups but without active ingredients.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ambroxol

FDA approved

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Parkinson's Disease (PD) aim to manage symptoms and improve quality of life. Ambroxol, currently under study, raises beta-glucocerebrosidase levels and lowers alpha-synuclein levels, which may improve cognitive and motor symptoms by reducing protein aggregation and enhancing cellular function. Other treatments include levodopa, which replenishes dopamine levels to improve motor control, and cholinesterase inhibitors like rivastigmine, which enhance cognitive function by increasing acetylcholine levels. These treatments are crucial as they target different aspects of PD pathology, offering a multifaceted approach to symptom management and potentially slowing disease progression.

Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial.