What side effects are associated with this type of intervention?

Common treatments for dementia, including those similar to Nilotinib, often have side effects that need to be carefully managed. Nilotinib has been shown to be generally well-tolerated, but potential side effects include central nervous system effects such as reduced amyloid burden and changes in cerebrospinal fluid biomarkers. Other common side effects of dementia treatments can include gastrointestinal issues, cardiovascular events, and liver toxicity. It is important to monitor patients closely and adjust dosages as necessary to mitigate these adverse effects.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of dementia, primarily focusing on Alzheimer's disease. These include cholinesterase inhibitors like donepezil, rivastigmine, and galantamine, as well as the NMDA receptor antagonist memantine. While these drugs primarily address symptoms, recent research has explored disease-modifying treatments. Nilotinib, a tyrosine kinase inhibitor, is being studied for its potential to reduce alpha-synuclein accumulation, a mechanism not yet targeted by FDA-approved dementia treatments. This approach is similar to investigational drugs aiming to reduce protein aggregation or neuroinflammation, such as those targeting amyloid-beta or tau proteins in Alzheimer's disease.

What other types of research exist?

Promising, novel alternatives to Nilotinib for dementia patients, particularly those with Dementia with Lewy Bodies, may include investigational drugs targeting alpha-synuclein accumulation or neuroprotective pathways. Examples include MAO-B inhibitors like selegiline and rasagiline, which have shown benefits in related conditions such as Parkinson's disease. Additionally, ongoing research into other neuroprotective agents and therapies targeting alpha-synuclein may provide new options in the near future.

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Tyrosine Kinase Inhibitor

Nilotinib for Lewy Body Dementia

Phase 2

Recruiting

Led By Fernando L Pagan, MD

Research Sponsored by Georgetown University

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 6 months

Summary

This trial tests if Nilotinib, a cancer drug taken as a pill, can help elderly patients with Dementia with Lewy Bodies by clearing harmful brain proteins. Nilotinib is part of a promising combination of medications shown to be effective against Alzheimer's disease.

Who is the study for?

This trial is for adults aged 25-90 with Dementia with Lewy Bodies (DLB), who can consent to study procedures or have a representative do so. They must be medically stable, on ≤800mg Levodopa daily, willing to undergo lumbar puncture, and meet specific clinical criteria for DLB. Excluded are those with renal insufficiency, active infections or cancer history within five years (except certain cases), pregnant/lactating women, liver/pancreatic/cardiac disease sufferers, and those on conflicting medications.

What is being tested?

The trial tests the safety and effects of Nilotinib oral capsules compared to placebo in patients with DLB. It's a phase II study where participants are randomly assigned to receive either the drug or a placebo while researchers monitor its impact on various health markers related to dementia.

What are the potential side effects?

While not specified here, common side effects of Nilotinib may include nausea, rash, fatigue, headache, constipation/diarrhea and muscle/joint pain. As it's used at lower doses than for its original purpose (treating leukemia), different side effects might occur in this context.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Safety and tolerability: occurrence of adverse events (AEs)

Secondary study objectives

The Investigator will determine Nilotinib levels in CSF and plasma.

The Investigators will determine changes in DLB related CSF and plasma biomarkers

The investigators will quantify amyloid burden via Florbetaben PET scan

Other study objectives

Measurement of the effects of Nilotinib on Cognition using the Montreal Cognitive Assessment (MoCA)

Measurement of the effects of Nilotinib on Cognition using the Trail Making Test (TMT)

Measuring the effects of NIlotinib on Cognition using the Alzheimer's Disease Assessment Scale - cognitive (ADAS-cog).

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Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: 200 mg NilotinibActive Control1 Intervention

Sixty (60) participants will be recruited and randomized into 2 arms (1:1). Thirty (30) patients in arm 2 will receive the 200 mg of Nilotinib one (1) capsule orally (without food) once daily for 6 months (180 days).

Group II: PlaceboPlacebo Group1 Intervention

Sixty (60) participants will be recruited and randomized into 2 arms (1:1). Thirty (30) patients in arm 1 will receive the matching placebo ("sugar pill") one (1) capsule orally (without food) once daily for 6 months (180 days).

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 dementia, such as cholinesterase inhibitors (e.g., donepezil, rivastigmine) and NMDA receptor antagonists (e.g., memantine), work by enhancing cholinergic function and regulating glutamate activity, respectively, to improve cognitive function and slow symptom progression. Nilotinib, a Tyrosine Kinase Inhibitor, is being studied for its potential to reduce alpha-synuclein accumulation, which is significant for conditions like Dementia with Lewy Bodies and Parkinson's Disease Dementia. These treatments are crucial as they target different pathological pathways, aiming to alleviate symptoms, improve quality of life, and potentially modify disease progression.

Nilotinib Effects on Safety, Tolerability, and Biomarkers in Alzheimer's Disease.Drugs in Alzheimer's disease Dementia: An overview of current pharmacological management and future directions.Utilizing combination therapy in the treatment of Alzheimer's disease.