Cancer drug shows promise for Parkinson's in small trial
NCT ID NCT02954978
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2 study tested whether low doses of nilotinib, a drug already approved for leukemia, are safe and affect brain markers in 75 people with Parkinson's disease. Participants took either a placebo or one of two low doses of nilotinib daily for 12 months. The goal was to see if the drug could help clear harmful proteins linked to Parkinson's, but the study focused on safety and biological changes, not on improving symptoms.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Nilotinib (Tasigna), a cancer drug used at low doses
- What this could lead to
- If successful, this could point toward a new way to slow Parkinson's progression by clearing toxic proteins in the brain.
- What could go wrong
- This is an early Phase 2 trial with only 75 people, focused on safety and biomarkers, not on curing symptoms. Nilotinib has known side effects, and low doses may not work as hoped.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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75 people
The number who actually took part.
- Started
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Jan 2017
- Finished
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Jul 2020
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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40 to 90 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Written informed consent 2. Capable of providing informed consent and complying with study procedures. Subjects who are unable to provide consent may use a Legally Authorized Representative (LAR). 3. Patients between the age of 40-90 years, medically stable 4. Diagnosis of PD according to the UK Brain Bank Diagnostic Criteria 5. PD subjects with MoCA ≥ 22 6. 2.5 ≥Hoehn and Yahr stage ≤3 7. No mono-amine oxidase (MAO)-B inhibitors (Selegeline or rasagiline) are allowed at least 6 weeks before enrollment 8. Must be medically stable on 800mg Levodopa daily for at least 4 weeks 9. QTc interval 350-460 ms, inclusive 10. Participants must be willing to undergo LP at baseline and 12 months after treatment Exclusion Criteria: 1. Patients with hypokalemia, hypomagnesaemia, or long QT syndrome- QTc≥461 ms 2. Concomitant drugs known to prolong the QTc interval and history of any cardiovascular disease, including myocardial infraction or cardiac failure, angina, arrhythmia 3. History or presence of cardiac conditions including: * Cardiovascular or cerebrovascular event (e.g. myocardial infarction, unstable angina, or stroke) * Congestive heart failure * First, second- or third-degree atrioventricular block, sick sinus syndrome, or other serious cardiac rhythm disturbances * Any history of Torsade de Pointes 4. Treatment with any of the following drugs at the time of screening or the preceding 30 days, and/or planned use over the course of the trial: * Treatment with Class IA or III antiarrhythmic drugs (e.g. quinidine) * Treatment with QT prolonging drugs (www.crediblemeds.org)- excluding Selective Serotonin Reuptake Inhibitors (SSRIs) (e.g. Citalopram, Paxil, Zoloft, Cymbalta, Sertraline, etc...) * Strong CYP3A4 inhibitors (including grapefruit juice). The concomitant use of strong CYP3A4 inhibitors (e.g., ketoconazole, itraconazole, clarithromycin, atazanavir, indinavir, nefazodone, nelfinavir, ritonavir, saquinavir, telithromycin, voriconazole) must be avoided. Grapefruit products may also increase serum concentrations of Nilotinib. Should treatment with any of these agents be required, therapy with Nilotinib should be interrupted. * Anticoagulants, including Coumadin (warfarin), heparin, enoxaparin, daltiparin, xarelto, etc. * St. John's Wort and the concomitant use of strong other CYP3A4 inducers (e.g., dexamethasone, phenytoin, carbamazepine, rifampin, rifabutin, rifapentine, phenobarbital) must be avoided since these agents may reduce the concentration of Nilotinib. 5. Abnormal liver function defined as AST and/or ALT \> 100% the upper limit of the normal 6. Renal insufficiency as defined by a serum creatinine \> 1.5 times the upper limit of normal 7. History of HIV, clinically significant chronic hepatitis, or other active infection 8. Females must not be lactating, pregnant or with possible pregnancy 9. Medical history of liver or pancreatic disease 10. Clinical signs indicating syndromes other than idiopathic PD, including corticobasal degeneration, supranuclear gaze palsy, multiple system atrophy, chronic traumatic encephalopathy, signs of frontal dementia, history of stroke, head injury or encephalitis, cerebellar signs, early severe autonomic involvement, Babinski sign 11. Current evidence or history in past two years of epilepsy, focal brain lesion, head injury with loss of consciousness or DSM-IV criteria for any major psychiatric disorder including psychosis, major depression, bipolar disorder, alcohol or substance abuse 12. Evidence of any significant clinical disorder or laboratory finding that renders the participant unsuitable for receiving an investigational drug including clinically significant or unstable hematologic, hepatic, cardiovascular, pulmonary, gastrointestinal, endocrine, metabolic, renal or other systemic disease or laboratory abnormality 13. Active neoplastic disease, history of cancer five years prior to screening, including breast cancer (history of skin melanoma or stable prostate cancer are not exclusionary) 14. Contraindications to LP: prior lumbosacral spine surgery, severe degenerative joint disease or deformity of the spine, platelets \< 100,000, use of Coumadin/warfarin, or history of a bleeding disorder 15. Must not be on any immunosuppressant medications (e.g. IVig) 16. Must not be enrolled as an active participant in another clinical study 17. Diagnosis of DLB
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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MedStar Georgetown University Hospital
Washington D.C., District of Columbia, 20007, United States
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