Ambroxol for Lewy Body Dementia

Overseen By Carolina Silveira, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: London Health Sciences Centre Research Institute and Lawson Research Institute of St. Joseph's

Must be taking: Levodopa, Cholinesterase inhibitors

Must not be taking: Oral anticoagulants

Disqualifiers: Stroke, Cancer, Cardiac disease, others

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

15 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stay on a stable dose of your current medications for parkinsonism, cognition, and psychiatric conditions for at least 3 months before starting. However, you cannot take certain blood thinners like Warfarin during the trial.

What data supports the effectiveness of the drug Ambroxol Hydrochloride for Lewy Body Dementia?

Research suggests that Ambroxol may help increase the activity of an enzyme called GCase, which is involved in breaking down harmful proteins in the brain. This could potentially help manage symptoms of Lewy Body Dementia by improving the brain's ability to clear these proteins. ^{1 2 3 4 5}

Is Ambroxol safe for use in humans?

Ambroxol has been used as a cough medicine for many years and is generally considered safe. In studies involving patients with Gaucher disease and Parkinson's disease, some minor side effects like bowel discomfort, cough, and dizziness were reported, but no severe adverse effects were noted. ^{1 2 5 6 7}

How is the drug Ambroxol Hydrochloride unique for treating Lewy Body Dementia?

Ambroxol Hydrochloride is unique because it may enhance the breakdown of harmful proteins in the brain by increasing the activity of an enzyme called glucocerebrosidase, which is linked to the disease process in Lewy Body Dementia. Unlike other treatments, it is being explored as a potential disease-modifying drug, rather than just managing symptoms. ^{1 2 3 8 9}

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Inclusion Criteria

Montreal Cognitive Assessment (MoCA) score: 24-18

I am over 50 years old.

I have someone to take care of me for at least 4 days a week.

Exclusion Criteria

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

I am not taking certain blood thinners but may use specific antiplatelet medications.

Depression that is, in the opinion of the investigator, significant enough to interfere with neuropsychology and safety assessments

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Ambroxol or placebo for 52 weeks to assess safety and effectiveness in treating Lewy Body Dementia

52 weeks

Regular visits for clinical, neuropsychological, and neuroimaging assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Ambroxol Hydrochloride
• Placebo

Trial Overview 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.

Participant Groups

Group I: Ambroxol Experimental Treatment 1 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: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

London Health Sciences Centre Research Institute and Lawson Research Institute of St. Joseph's

Lead Sponsor

Trials

686

Recruited

427,000+

London Health Sciences Centre OR Lawson Research Institute of St. Joseph's

Lead Sponsor

Trials

668

Recruited

424,000+

London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

Lead Sponsor

Trials

678

Recruited

421,000+

Lawson Health Research Institute

Lead Sponsor

Trials

684

Recruited

432,000+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

The ANeED study - ambroxol in new and early dementia with Lewy bodies (DLB): protocol for a phase IIa multicentre, randomised, double-blinded and placebo-controlled trial. [2023]

2. England pubmed.ncbi.nlm.nih.gov

RENEWAL: REpurposing study to find NEW compounds with Activity for Lewy body dementia-an international Delphi consensus. [2023]

3. Germany pubmed.ncbi.nlm.nih.gov

[Dementia with Lewy bodies]. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Dementia with lewy bodies: findings from an international multicentre study. [2019]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Aripiprazole for the treatment of psychotic symptoms in patients with dementia with Lewy bodies: a case series. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial. [2020]

8. England pubmed.ncbi.nlm.nih.gov

Dementia with Lewy bodies. [2021]

9. England pubmed.ncbi.nlm.nih.gov

Increased dosage of donepezil for the management of behavioural and psychological symptoms of dementia in dementia with Lewy bodies. [2022]