Start: August 2019
End: April 30, 2023
What Is This Study About?
Nilotinib is a drug approved by the U.S. Food and Drug Administration to treat certain types of leukemia. In this Phase 2 trial, researchers are investigating the drug for its potential to break down abnormal alpha-synuclein proteins associated with dementia with Lewy bodies. Participants will be randomly assigned to take 200 mg of nilotinib or placebo for six months and monitored over a seven-month period.
Do I Qualify To Participate in This Study?
- Diagnosis of dementia with Lewy bodies
- Both dementia Montreal Cognitive Assessment of 18 and parkinsonism defined as bradykinesia (slowness of movement) in combination with rest tremor, rigidity, or both
- Unified Parkinson Disease Rating Scale (UPDRS) I-III less than 50 and UPDRS-III of 15 to 40
- Dementia and parkinsonism must be present with at least one other symptom such as fluctuation, visual hallucinations, or REM sleep behavioral disorder
- Hoehn and Yahr stage 2.5 to 3
- Abnormal DaTScan, a brain imaging test used to help diagnosis parkinsonian syndromes
- Stable medical and/or psychiatric illnesses
- Stable on mono-amine oxidase-B inhibitors (selegeline or rasagiline) for at least 4 weeks before enrollment and during treatment
- Stable on less than or equal to 800 mg levodopa daily for at least 4 weeks
- QTc interval 350 to 460 ms
- Willing to undergo lumbar puncture at baseline and 6 months after treatment
Must NOT have:
- Hypokalemia (high potassium levels), hypomagnesaemia (low levels of magnesium), or long QT syndrome
- Drugs known to prolong the QTc interval and history of any heart disease, including heart attack, heart failure, angina, and arrhythmia
- History or presence of heart conditions, including cardiovascular or cerebrovascular event (e.g., heart attack, unstable angina, or stroke); congestive heart failure; serious rhythmic disturbances; or history of torsade de pointes
- Prohibited medications: Class IA or III antiarrhythmic drugs such as quinidine; QT prolonging drugs, excluding SSRIs (e.g., citalopram, paroxetine, sertraline, duloxetine); strong CYP3A4 inhibitors, including grapefruit juice, ketoconazole, itraconazole, clarithromycin, atazanavir, indinavir, nefazodone, nelfinavir, ritonavir, saquinavir, telithromycin, and voriconazole; anticoagulants, including warfarin, heparin, enoxaparin, daltiparin, and xarelto; St. John's Wort and other CYP3A4 inducers (e.g., dexamethasone, phenytoin, carbamazepine, rifampin, rifabutin, rifapentine, phenobarbital)
- Abnormal liver function defined as aspartate aminotransferase and/or alanine transaminase greater than 100 percent the upper limit of normal
- Kidney insufficiency as defined by serum creatinine levels greater than 1.5 times the upper limit of normal
- History of HIV, clinically significant chronic hepatitis, or other active infection
- Lactating, pregnant, or with possible pregnancy
- Medical history of liver or pancreatic disease
- Clinical signs indicating syndromes other than dementia with Lewy bodies, including Parkinson's disease, Parkinson's disease with dementia, corticobasal degeneration, supranuclear gaze palsy, multiple system atrophy, chronic traumatic encephalopathy, frontotemporal dementia, stroke, head injury or encephalitis, cerebellar signs, early severe autonomic involvement, or Babinski sign
- Current evidence or history in past 2 years of epilepsy, focal brain lesion, or head injury with loss of consciousness, or any major psychiatric disorder including psychosis, major depression, bipolar disorder, or alcohol or substance abuse
- Any significant clinical disorder or laboratory finding, including blood, liver, heart, lung, gastrointestinal, endocrine, metabolic, kidney, or other systemic disease or abnormality
- Active neoplastic disease or history of cancer in past 5 years (history of skin melanoma or stable prostate cancer are permitted)
- Inability to undergo lumbar puncture, including prior lower back spine surgery, severe degenerative joint disease or deformity of the spine, low platelets, use of warfarin, or history of a bleeding disorder
- Taking immunosuppressant medications or intravenous immune globulin
- Enrolled as an active participant in another clinical study
If I Qualify, Who Do I Contact?
Contact study personnel listed either under the general study contact or the location nearest you.
Myrna J Arellano, RN
Sara Matar, BS
Contact NIA’s Alzheimer’s and related Dementias Education and Referral (ADEAR) Center at 800-438-4380 or email ADEAR.
Where Is This Study Located?
Lead: Georgetown University
National Institutes of Health (NIH)
Source: ClinicalTrials.gov ID: NCT04002674