Elucidating the Role of Cholinergic Degeneration in Cognitive Fluctuations in Lewy Body Dementia

NCT ID: NCT07284290

Last Updated: 2025-12-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2026-01-25

Study Completion Date

2029-11-30

Brief Summary

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The proposed study aims to address the critical gaps in understanding the mechanisms of CF (Cognitive Fluctuations) by leveraging recently emerged molecular biomarkers, advanced neuroimaging techniques to assess measures of cholinergic degeneration, and synchronous EEG and assessments of attention. One of the overarching innovations of study is combining all of these assessments into one integrated research plan

Detailed Description

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Aim 1 is a cross-sectional case control study in which cholinergic degeneration in 45 participants with DLB or PDD with CF will be compared to a group of 45 individuals with Lewy Body disease without CF and 30 healthy controls. The first 20 participants who are eligible for Aim 2 and consent will also participate in an 8-week pre-post interventional cohort study immediately following Aim 1 procedures. For Aim 3, Aim 1 participants will complete annual follow-up evaluations for 2 years to understand factors influencing the change in cognitive fluctuations over time.

Conditions

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Dementia With Lewy Bodies Parkinson Disease Dementia Healthy Controls

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

A cross-sectional case control study in which cholinergic degeneration in 45 subjects with DLB or PDD with CF will be compared to a group of 45 subjects with Lewy Body disease without CF and 30 healthy controls. The first 20 participants who are eligible and consent will also participate in an 8-week pre-post interventional cohort study.
Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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Subjects with DLB or PDD

90 subjects with Dementia with Lewy Bodies (DLB) or Parkinson's Disease with Dementia (PDD)

Group Type EXPERIMENTAL

Syn-One skin biopsy

Intervention Type PROCEDURE

Detects phosphorylated alpha synuclein in cutaneous nerve fibrils which has \>95% sensitivity in detecting DLB

Multi modal MRI

Intervention Type DIAGNOSTIC_TEST

Functional MRI used to investigate the network connectivity changes associated with alterations in the cholinergic system. Using this comprehensive methodology to establish cholinergic degeneration's contribution to CF provides a robust framework for future research and therapeutic strategies.

Assessment of dynamic EEG features over 48-hour periods across all study aims

Intervention Type DIAGNOSTIC_TEST

Implementing prolonged EEG monitoring, to capture dynamic changes in neural activity associated with CF. The methods are designed avoid the limitations identified in a systematic review of EEG studies in DLB, as will use quantitative analysis of EEG, uniformly apply diagnostic criteria, and consider the confounding effects of medications.

Concurrent evaluation of PVT performance and EEG This dual assessment will correlate cognitive and neurophysiological dynamics in real-time, providing a more holistic understanding of CF over time and the functional brain activity that underlies them. Temporal integration of PVT with EEG data collection will enable the identity of specific correlates of CF.

Plasma biomarkers

Intervention Type DIAGNOSTIC_TEST

A blood sample will be collected and sent for processing by C2N Diagnostics for the detection of Aβ42/40 and p-tau217 via mass spectrometry, a method shown to be highly accurate for predicting amyloid positivity on PET (AUC=0.94). Exploratory Biomarkers: The additional blood sample will be used for analysis of exploratory biomarkers and future research. A total of up to 20mL of blood will be taken.

Subjects with Lewy Body disease with CF

A subset of 20 subjects with Lewy Body (LB) disease with Cognitive fluctuations (CF)

This arm will be a subset of 20 subjects from Arm #1 (Subjects with DLB or PDD).

Group Type ACTIVE_COMPARATOR

Multi modal MRI

Intervention Type DIAGNOSTIC_TEST

Functional MRI used to investigate the network connectivity changes associated with alterations in the cholinergic system. Using this comprehensive methodology to establish cholinergic degeneration's contribution to CF provides a robust framework for future research and therapeutic strategies.

Assessment of dynamic EEG features over 48-hour periods across all study aims

Intervention Type DIAGNOSTIC_TEST

Implementing prolonged EEG monitoring, to capture dynamic changes in neural activity associated with CF. The methods are designed avoid the limitations identified in a systematic review of EEG studies in DLB, as will use quantitative analysis of EEG, uniformly apply diagnostic criteria, and consider the confounding effects of medications.

Concurrent evaluation of PVT performance and EEG This dual assessment will correlate cognitive and neurophysiological dynamics in real-time, providing a more holistic understanding of CF over time and the functional brain activity that underlies them. Temporal integration of PVT with EEG data collection will enable the identity of specific correlates of CF.

Galantamine HBr extended-release 8mg capsules (8mg ER).

Intervention Type DRUG

20 participants from the arm 1. Participants will take 1 capsule daily of galantamine 8mg ER for 4 weeks and then increase to 2 capsules daily of galantamine 8mg ER (16mg) for 4 weeks. This titration will mitigate potential for cholinergic side effects. At 2 weeks, 4 weeks and 6 weeks of the treatment period, safety and compliance will be assessed during phone call visits. Any adverse event rated as Grade 2 or greater according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and that is definitely or possibly attributable to the study drug will result in study drug withdrawal or dose reduction.

Healthy control subjects

30 healthy controls

Group Type OTHER

Multi modal MRI

Intervention Type DIAGNOSTIC_TEST

Functional MRI used to investigate the network connectivity changes associated with alterations in the cholinergic system. Using this comprehensive methodology to establish cholinergic degeneration's contribution to CF provides a robust framework for future research and therapeutic strategies.

Assessment of dynamic EEG features over 48-hour periods across all study aims

Intervention Type DIAGNOSTIC_TEST

Implementing prolonged EEG monitoring, to capture dynamic changes in neural activity associated with CF. The methods are designed avoid the limitations identified in a systematic review of EEG studies in DLB, as will use quantitative analysis of EEG, uniformly apply diagnostic criteria, and consider the confounding effects of medications.

Concurrent evaluation of PVT performance and EEG This dual assessment will correlate cognitive and neurophysiological dynamics in real-time, providing a more holistic understanding of CF over time and the functional brain activity that underlies them. Temporal integration of PVT with EEG data collection will enable the identity of specific correlates of CF.

Plasma biomarkers

Intervention Type DIAGNOSTIC_TEST

A blood sample will be collected and sent for processing by C2N Diagnostics for the detection of Aβ42/40 and p-tau217 via mass spectrometry, a method shown to be highly accurate for predicting amyloid positivity on PET (AUC=0.94). Exploratory Biomarkers: The additional blood sample will be used for analysis of exploratory biomarkers and future research. A total of up to 20mL of blood will be taken.

Interventions

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Syn-One skin biopsy

Detects phosphorylated alpha synuclein in cutaneous nerve fibrils which has \>95% sensitivity in detecting DLB

Intervention Type PROCEDURE

Multi modal MRI

Functional MRI used to investigate the network connectivity changes associated with alterations in the cholinergic system. Using this comprehensive methodology to establish cholinergic degeneration's contribution to CF provides a robust framework for future research and therapeutic strategies.

Intervention Type DIAGNOSTIC_TEST

Assessment of dynamic EEG features over 48-hour periods across all study aims

Implementing prolonged EEG monitoring, to capture dynamic changes in neural activity associated with CF. The methods are designed avoid the limitations identified in a systematic review of EEG studies in DLB, as will use quantitative analysis of EEG, uniformly apply diagnostic criteria, and consider the confounding effects of medications.

Concurrent evaluation of PVT performance and EEG This dual assessment will correlate cognitive and neurophysiological dynamics in real-time, providing a more holistic understanding of CF over time and the functional brain activity that underlies them. Temporal integration of PVT with EEG data collection will enable the identity of specific correlates of CF.

Intervention Type DIAGNOSTIC_TEST

Plasma biomarkers

A blood sample will be collected and sent for processing by C2N Diagnostics for the detection of Aβ42/40 and p-tau217 via mass spectrometry, a method shown to be highly accurate for predicting amyloid positivity on PET (AUC=0.94). Exploratory Biomarkers: The additional blood sample will be used for analysis of exploratory biomarkers and future research. A total of up to 20mL of blood will be taken.

Intervention Type DIAGNOSTIC_TEST

Galantamine HBr extended-release 8mg capsules (8mg ER).

20 participants from the arm 1. Participants will take 1 capsule daily of galantamine 8mg ER for 4 weeks and then increase to 2 capsules daily of galantamine 8mg ER (16mg) for 4 weeks. This titration will mitigate potential for cholinergic side effects. At 2 weeks, 4 weeks and 6 weeks of the treatment period, safety and compliance will be assessed during phone call visits. Any adverse event rated as Grade 2 or greater according to the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 and that is definitely or possibly attributable to the study drug will result in study drug withdrawal or dose reduction.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Arm 1:

* Age range: 50 ≤ age \< 90.
* Diagnosis of dementia with Lewy bodies (DLB), Parkinson disease dementia (PDD), Parkinson disease with Mild Cognitive Impairment (PD-MCI), Mild Cognitive Impairment with Lewy bodies (MCI-LB).
* DLB participants must fulfill criteria for clinically probable DLB based on the 2017 4th consensus report of the DLB consortium.
* PDD participants must meet criteria for clinically probable PD according to the MDS Clinical Diagnostic Criteria for Parkinson's Disease and must also meet criteria for probable PDD based on the 2007 Movement Disorders Society clinical diagnostic criteria.
* PD-MCI participants must meet criteria for clinically probable PD according to the MDS Clinical Diagnostic Criteria for Parkinson's Disease and meet criteria for Mild Cognitive Impairment on cognitive testing at screening.
* MCI-LB participants with must meet established research criteria.
* Capacity to provide informed consent or, if unable, availability of a legally authorized representative or guardian who can provide informed consent.
* Availability of informant (for participants meeting criteria for dementia).
* Ability and willingness to comply with the study-related procedures.
* Fluent in spoken and written English (due to cognitive testing)



* Completed Aim 1.
* Clinical diagnosis of LBD (DLB or PDD) with CF.
* Not taking a cholinesterase inhibitor and has not taken a cholinesterase inhibitor in the previous 90 days.
* Ability and willingness to comply with the ChEI Cohort procedures (including galantamine administration), or a caregiver willing and able to ensure compliance.


Arm 3 (Healthy Controls)

* Age range: 50 ≤ age \< 90.
* Healthy controls should not have any known neurologic conditions that could interfere with study procedures or results.
* Capacity to provide informed consent or, if unable, availability of a legally authorized representative or guardian who can provide informed consent.
* Availability of informant (for participants meeting criteria for dementia).
* Ability and willingness to comply with the study-related procedures.
* Fluent in spoken and written English (due to cognitive testing).

Exclusion Criteria

Arm 1

* History of cognitive disorder or psychiatric disorder other than that related to dementia with Lewy bodies or Parkinson disease dementia.
* History of deep brain stimulation or any neurosurgical procedure.
* History of structural brain disease or known significant cerebrovascular disease.
* History of seizures or epilepsy and/or use of sodium channel blockers, i.e. carbamazepine, oxcarbazepine, phenytoin, topiramate, lamotrigine, felbamate, zonisamide, rufinamide, lacosamide, eslicarbazepine, and valproate.
* Greater than two alcoholic drinks per day for men and one per day for women.
* Regular use of benzodiazepines or barbiturates. (If benzodiazepines are taken as needed only, these medications cannot be taken within 5 half-lives of screening visit or between screening visit and EEG.)
* Severe dementia (based on PI assessment of subject dependence level for instrumental activities of daily living)
* Any contraindication to brain MRI.
* Any medical condition that would interfere with ability to complete all study procedures.
* Participants must not be pregnant, planning to become pregnant, or father a child for the duration of the study



* Severe hepatic impairment.
* Renal failure.
* Significant bradycardia (\<50 bpm) at screening or history of AV block.
* Any contraindication to galantamine administration based on PI discretion.


Arm 3 (Healthy Controls)

* No History of cognitive disorder or psychiatric disorder other than that related to dementia with Lewy bodies or Parkinson disease dementia.
* No History of deep brain stimulation or any neurosurgical procedure.
* No History of structural brain disease or known significant cerebrovascular disease.
* No History of seizures or epilepsy and/or use of sodium channel blockers, i.e. carbamazepine, oxcarbazepine, phenytoin, topiramate, lamotrigine, felbamate, zonisamide, rufinamide, lacosamide, eslicarbazepine, and valproate.
* Any medical condition that would interfere with ability to complete all study procedures.
* Participants must not be pregnant, planning to become pregnant, or father a child for the duration of the study
Minimum Eligible Age

50 Years

Maximum Eligible Age

89 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role collaborator

Virginia Commonwealth University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Matthew Barrett

Role: PRINCIPAL_INVESTIGATOR

Virginia Commonwealth University

Locations

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Virginia Commonwealth University

Richmond, Virginia, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Madison Clemons

Role: CONTACT

(804) 827-1754

Kara McHaney

Role: CONTACT

Facility Contacts

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Madison Clemons

Role: primary

804-827-1754

Kara McHaney

Role: backup

Other Identifiers

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R01NS142622

Identifier Type: NIH

Identifier Source: secondary_id

View Link

HM20033370

Identifier Type: -

Identifier Source: org_study_id