What is the mechanism of action of this treatment?

Common treatments for Lewy Body Disease (LBD) include cholinesterase inhibitors and memantine, which manage cognitive and neuropsychiatric symptoms by enhancing neurotransmitter function. However, these treatments do not address the underlying cause of LBD, which involves the accumulation of misfolded proteins in the brain. Ambroxol, a disease-modifying treatment currently under study, aims to reduce or prevent this protein accumulation. This approach is significant for LBD patients as it has the potential to slow or halt disease progression, offering a more sustainable improvement in their condition compared to symptomatic treatments alone.

What side effects are associated with this type of intervention?

Common treatments for Lewy Body Disease (LBD) primarily target symptom management and include cholinesterase inhibitors, antipsychotics, and medications for Parkinsonian symptoms. Cholinesterase inhibitors, such as donepezil and rivastigmine, can cause gastrointestinal issues like nausea, vomiting, and diarrhea. Antipsychotics, particularly atypical ones like quetiapine and clozapine, may lead to sedation, weight gain, and increased risk of stroke. Medications for Parkinsonian symptoms, such as levodopa, can cause dyskinesia, orthostatic hypotension, and hallucinations. While Ambroxol is being studied for its potential to modify disease progression by reducing protein accumulation, its long-term safety and side effect profile are still under investigation.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically for Lewy Body Disease that target the underlying cause, such as reducing or preventing protein accumulation in the brain. Most treatments for LBD focus on managing symptoms rather than modifying the disease process. Ambroxol is being studied for its potential to address the underlying protein accumulation, which is a novel approach compared to existing therapies.

What other types of research exist?

Currently, there are no specific alternatives to Ambroxol mentioned in the provided context for Lewy Body Disease. However, treatments like pramipexole, which has shown efficacy in improving motor function and reducing depressive symptoms in Parkinson's disease, might be considered for symptom management in LBD. Further research and clinical trials are needed to identify and confirm novel disease-modifying treatments for LBD.

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Mucolytic Agent

Ambroxol for Lewy Body Dementia

Phase 1 & 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

Age greater than 50 years old

Must be on a stable dose of medications for parkinsonism (levodopa, dopaminergic agonist) and cognition (cholinesterase inhibitors) and psychiatric (i.e. antidepressants, antipsychotic) for at least 3 months prior to the study

Must not have

Contraindication to MRI e.g. presence of metal fragments in head or eye, implanted electrical devices or conductive implants or devices (pacemakers, neurostimulators)

Any other serious underlying condition or brain disorder that can account in part or in full for the clinical presentation (i.e. cancer or unstable cardiac disease etc.)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

Awards & highlights

All Individual Drugs Already Approved

Summary

This trial tests if Ambroxol, a common mucus-clearing medicine, is safe and effective for treating Lewy Body Dementia (LBD). LBD patients are targeted because current treatments only manage symptoms and do not address the underlying protein buildup in the brain. Ambroxol increases an enzyme that helps break down these harmful proteins, potentially improving brain function.

Who is the study for?

This trial is for people over 50 with Lewy Body Dementia, who have a caregiver at least 4 days a week and are on stable medication for parkinsonism, cognition, and psychiatric symptoms. Pregnant women or those planning to conceive during the study can't participate. People with metal in their body that affects MRI scans or taking certain blood thinners also can't join.

What is being tested?

The study tests Ambroxol Hydrochloride's safety and effectiveness against Lewy Body Dementia compared to a placebo. It's randomized (participants are randomly assigned treatment), double-blind (neither researchers nor participants know who gets what), lasting one year with assessments throughout.

What are the potential side effects?

While specific side effects of Ambroxol in this context aren't detailed here, common ones may include allergic reactions, gastrointestinal discomforts like nausea or vomiting, headache, dizziness, dry mouth/throat.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am over 50 years old.

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I have been on a stable dose of my Parkinson's and mental health medications for at least 3 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I cannot have an MRI due to metal in my body or implanted devices.

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I do not have any serious conditions like cancer or heart disease that explain my symptoms.

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I have had a stroke or another neurological condition.

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I am not pregnant, breastfeeding, nor planning to conceive during the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 4, week 10, 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

Change from baseline in cerebrospinal fluid (CSF) concentrations of Ambroxol at specified time points

Change from baseline in enzyme β-Glucocerebrosidase (GCase) concentration levels in CSF

Change from baseline in enzyme β-Glucocerebrosidase (GCase) concentration levels in white blood cells

+9 more

Secondary study objectives

Change in Cerebrospinal Fluid (CSF) biomarkers

Change in global brain magnetic resonance imaging atrophy measures

Change in plasma biomarkers

+10 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: AmbroxolExperimental Treatment1 Intervention

Participants randomized to the 1350 mg/day group will begin with a dose of 450 mg, increasing bi-weekly to a dose of 1350 mg/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

0 / 6Eligibility criteria met

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 Lewy Body Disease (LBD) include cholinesterase inhibitors and memantine, which manage cognitive and neuropsychiatric symptoms by enhancing neurotransmitter function. However, these treatments do not address the underlying cause of LBD, which involves the accumulation of misfolded proteins in the brain. Ambroxol, a disease-modifying treatment currently under study, aims to reduce or prevent this protein accumulation. This approach is significant for LBD patients as it has the potential to slow or halt disease progression, offering a more sustainable improvement in their condition compared to symptomatic treatments alone.

Bromocriptine lessens the incidence of mortality in L-dopa-treated parkinsonian patients: prado-study discontinued.Istradefylline - a first generation adenosine A2A antagonist for the treatment of Parkinson's disease.Memantine for Lewy body disorders: systematic review and meta-analysis.