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Not yet recruitingPhase 1 / PHASE2

Ambroxol as a Novel Disease Modifying Treatment for Lewy Body Dementia

Lead sponsor

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

Asset

Ambroxol

Listed sites

Recruiting sites

Enrollment

estimated

Study population

Lewy body dementia

Key I/E criteria

•MoCA 18-24•Study partner/caregiver required•AD symptomatic therapy: stable•MRI contraindications excluded

Primary endpoints

•Mini-Mental State Examination (MMSE)•The incidence, nature and severity of AE's and SAE's•The number of participants with treatment discontinuations

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

NCT IDNCT04405596

Org study IDREB:115252

Timeline

Milestones

Study first posted2020-05-28actual

Last update posted2023-12-13actual

Study start2025-01estimated (month precision)

Primary completion2026-01estimated (month precision)

Study completion2027-01estimated (month precision)

Assets

Drug assets

Ambroxol

Study populations

Who this study enrolls

Lewy body dementia

Eligibility

Who can enroll

Minimum age50 Years

SexAll

Healthy volunteersNot accepted

Inclusion criteria

1. Probable diagnosis of Lewy Body Dementia

2. Age greater than 50 years old

3. Montreal Cognitive Assessment (MoCA) score: 24-18

4. Patients must have a responsible caregiver = 4days/week

5. 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

Exclusion criteria

1. Evidence of stroke or other neurological condition

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

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

4. Unable to undergo DAT-scan

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

6. Females who are pregnant or breastfeeding, or planning to conceive within the study period

7. 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. Exceptions: antiplatelet agents such as Aspirin, Clopidogrel, and Aggrenox.

Endpoints (25)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Safety / tolerability / PK

Global cognition

Other (unclassified)

Behavior / neuropsychiatric

Amyloid biomarkers

Neuroimaging

Fluid / digital biomarkers

Other clinical outcomes

Global cognition

5 endpoints

Primary/protocol endpoint

Change in Mini Mental State Examination score from baseline over time

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

Mini-Mental State Examination (MMSE)

change from baseline, improvement

Secondary/protocol endpoint

Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)

Time frame:Baseline, week 26, and week 52

categorical status, descriptive

Secondary/protocol endpoint

Montreal Cognitive Assessment (MoCA)

Time frame:Baseline, week 26, and week 52

Montreal Cognitive Assessment (MoCA)

descriptive

Secondary/protocol endpoint

Trail making test A and B to assess cognitive function

Time frame:Baseline, week 26, and week 52

descriptive

Secondary/protocol endpoint

Parkinson's disease - Cognitive rating scale to assess cognitive function

Time frame:Baseline, week 26, and week 52

descriptive

Behavior / neuropsychiatric

2 endpoints

Secondary/protocol endpoint

Neuropsychological Inventory (NPI)

Time frame:Baseline, week 26, and week 52

Neuropsychiatric Inventory (NPI)

event count, event

Secondary/protocol endpoint

Geriatric Depression Scale

Time frame:Baseline, week 26, and week 52

descriptive

Amyloid biomarkers

2 endpoints

Secondary/protocol endpoint

Change in plasma biomarkers

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, improvement

Secondary/protocol endpoint

Change in Cerebrospinal Fluid (CSF) biomarkers

Time frame:Baseline, week 10, week 52

change from baseline, improvement

Neuroimaging

2 endpoints

Secondary/protocol endpoint

Change in regional brain magnetic resonance imaging atrophy measures

Time frame:Baseline, week 52

change from baseline, improvement

Secondary/protocol endpoint

Change in global brain magnetic resonance imaging atrophy measures

Time frame:Baseline, week 52

change from baseline, improvement

Fluid / digital biomarkers

2 endpoints

Primary/protocol endpoint

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

Time frame:Baseline, week 10, week 52

change from baseline, improvement

Primary/protocol endpoint

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

Time frame:Baseline, week 10, week 52

change from baseline, improvement

Safety / tolerability / PK

7 endpoints

Primary/protocol endpoint

Change in the incidence, nature and severity of AE's and SAE's from baseline

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Primary/protocol endpoint

Change in the number of participants with treatment discontinuations and study discontinuation due to AEs from baseline

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Primary/protocol endpoint

Change in the number of participants with electrocardiogram (ECG) abnormalities

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Primary/protocol endpoint

Change from baseline the number of participants with abnormal changes in hemodynamic values while seated

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Primary/protocol endpoint

Change from baseline the number of participants with abnormal changes in hemodynamic values while standing

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Primary/protocol endpoint

Change in blood analyses from baseline over time

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Primary/protocol endpoint

Change in urine analyses from baseline over time

Time frame:Baseline, week 4, week 10, week 18, week 26, week 34, week 42, week 52

change from baseline, event

Other clinical outcomes

1 endpoint

Secondary/protocol endpoint

Timed Up and Go

Time frame:Baseline, week 26, and week 52

descriptive

Other (unclassified)

4 endpoints

Primary/protocol endpoint/low confidence

Change from baseline in plasma concentrations of Ambroxol from blood sample

Time frame:Baseline, week 4, week 10, week 26, week 52

change from baseline, improvement

Primary/protocol endpoint/low confidence

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

Time frame:Baseline, week 4, week 10, week 26, week 52

change from baseline, improvement

Secondary/protocol endpoint/low confidence

Clinician's Global Impression of Change (CGIC)

Time frame:Baseline, week 26, and week 52

descriptive

Secondary/protocol endpoint/low confidence

The Motor subscale of Unified Parkinson's Disease Rating Scale (UPDRS-III)

Time frame:Baseline, week 26, and week 52

descriptive

Publications (20)

Bibliography

Records linked to this trial through ClinicalTrials.gov references, PubMed NCT search, and curated study seeds. 'Canonical' marks design/result papers; others are registry references or candidates.

Registry references + supporting bibliography

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableno registry results posted yet

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.