Parkinson's DiseasePhase 1NCT02281474
Nilotinib in Cognitively Impaired Parkinson Disease Patients 001
A Phase 1 study testing Nilotinib in people with Parkinson's Disease and Parkinson's Disease Dementia, run by Georgetown University at 1 site in United States.
Trial summary
This pilot study will test Nilotinib's ability to alter the abnormal protein build up in Parkinson disease and Diffuse Lewey Body Disease patients . Patients will receive Nilotinib at different doses for 6 months. Read more about this study
- Who is it for?
- Ages 40 to 90 yearsHealthy volunteers can take part
- Conditions the study is for
- Parkinson's Disease, Parkinson's Disease Dementia, Diffuse Lewy Body Disease
- What is being tested?
- Nilotinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2014to May 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 90 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 1. Patients aged 40 to 90 with Idiopathic Parkinson's Disease (Significant Sinemet response) on a stable medication drug regimen L-dopa and/or Dopamine agonist (at least 1 month before enrollment with no new medication change) and with moderate to severe cognitive impairment (MOCA ≤24).
- Inclusions criteria:
- Written informed consent
- Capability and willingness to comply with the study related criteria
- Patients between the age of 40-90 y
- Diagnosis of PD according to the UK Brain Bank Diagnostic Criteria
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- Early PD subjects with MMSE between 23-30.
- Hoehn and Yahr stage <2
- Stable treatment (>4 weeks) with MAO-B inhibitor (Selegeline up to 10mg/d or rasagiline up to 1 mg/d) allowable
- Patients not needing dopamine agonist or levodopa therapy presently or at least for the next 6 months
- Idiopathic PD with NO genetic mutations (autosomal recessive or dominant)
- Detectable levels of CSF for blood and CSF Alpha-Synuclein
Who cannot join
- Patients with a known genetic form of PD that does not involve alpha-synuclein.
- Unwillingness to undergo lumbar punctures
- Immeasurable CSF α-synuclein.
- Presence of dementia or severe cognitive impairment that would not permit the patient to give adequate feedback for potential side effects.
- Unwilling to be in an off state for UPDRS assessment.
- Pre-menopausal women
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- Patients with autosomal recessive (PARKIN, PINK1 or DJ1) or dominant mutations (LRRK2)
- Patients with hypokalemia, hypomagnesaemia, or long QT syndrome.
- Concomitant drugs known to prolong the QT interval
- Strong CYP3A4 inhibitors
- Any drugs or foods that may interact with Nilotinib as stated in the Package Insert (PI).
- Medical history of liver and pancreatic diseases.
- Clinical signs indicating syndromes other than idiopathic PD, including supranucelar gaze palsy, signs of frontal dementia, history of stroke, head injury or encephalitis, cerebellar sings, early severe autonomic involvement, Babinski's signs.
- History of any cardiovascular disease, including hypertension, myocardial infraction or cardiac failure, angina, arrhythmia.
Trial locations and contacts
1 site in 1 country.
United States1 site
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, 20007
About this study
Official title: Open Label Dose Escalation of Nilotinib in Cognitively Impaired Parkinson Disease Patients With Elevated Cerebrospinal Fluid and Blood α-Synuclein
This pilot study will test Nilotinib's ability to alter the abnormal protein build up in Parkinson disease and Diffuse Lewey Body Disease patients . Patients will receive Nilotinib at different doses for 6 months. Patients will then be tested to see if there is change in three areas: 1) has the disease symptoms changed. 2) has levels of a specific misfolded protein changed in the fluid around their brain and spine. 3) Have inflammatory markers changed in the patient's blood and fluid around their brain and spine. If successful, this drug could be used to slow down or stop the progression of disorders that involve abnormal collection of misfolded proteins. However, the main purpose of this pilot study is to check for the safety of using this medication at this level.
- Being tested
- Nilotinib
- Medical subject headings
- Parkinson Disease, Lewy Body Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NilotinibDrug
Also called Tasigna
| Study group | Role | What they receive |
|---|---|---|
| 150mg dosing | Active comparator | This arm will take 150mg of Nilotinib by mouth daily for the 6 month drug period to establish a safe and efficacious dose. Receives: Drug: Nilotinib |
| 300mg dosing | Active comparator | This arm will take 300mg of Nilotinib by mouth daily for the 6 month drug period to establish a safe and efficacious dose. Receives: Drug: Nilotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in α-synuclein and Tau concentrations in the CSF and serum of patients
Working Hypothesis: PD patients have been shown to have elevated levels of α-synuclein in their CSF. Nilotinib has been shown to reduce α-synuclein and Tau in the gastrointestinal tract and central nervous system in animal models, and similarly, we propose will show changes in CSF and serum α-synuclein concentrations in nilotinib treated PD patients.
Measured: 6 months
Secondary outcomes (2)
Determine nilotinib's efficacy by improvement in motor and non-motor symptoms
Working Hypothesis: By following strict safety guidelines, monitoring patients through physical examinations, self-examinations, laboratory and neurological examinations, nilotinib will be a safe drug to use in patients with PD and PD related patients.
Determine if any clinical benefit is observed in this small, short, limited clinical trial.
Working Hypothesis: In cell culture and animal models of PD, dopaminergic neurons have shown increased cell death with accumulating α-synuclein. Therefore, PD patients treated with nilotinib, which lowers α-synuclein and Tau in vivo and in vitro studies, will have improvement or stabilization of their motor UPDRS and cognition.
Measured: 6 months
Safety and tolerability, as measured by number of Participants with Adverse Events
Working Hypothesis: By following strict safety guidelines, monitoring patients through physical examinations, self-examinations, laboratory and neurological examinations, nilotinib will be a safe drug to use in patients with PD and PD related patients.
Determine if any clinical benefit is observed in this small, short, limited clinical trial.
Working Hypothesis: In cell culture and animal models of PD, dopaminergic neurons have shown increased cell death with accumulating α-synuclein. Therefore, PD patients treated with nilotinib, which lowers α-synuclein and Tau in vivo and in vitro studies, will have improvement or stabilization of their motor UPDRS and cognition.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2014 (estimated)
- Study startNov 2014
- Primary completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- More trials run by Georgetown UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Georgetown University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02281474
- Sponsor's protocol ID
- IIT-2014-001
- Status last verified
- Dec 2015
- First submitted
- 27 Oct 2014
- Last updated
- 16 Dec 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Joshi D, Kulkarni M, Parekh P, Shah S, Greig NH, Acharya S. Targeting protein kinases in Parkinson's disease: the emerging role of phytoconstituents. Nutr Neurosci. 2025 Dec;28(12):1532-1563. doi: 10.1080/1028415X.2025.2531356. Epub 2025 Jul 18.
PubMed 40680102
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Dose Escalation of Nilotinib in Cognitively Impaired Parkinson Disease Patients With Elevated Cerebrospinal Fluid and Blood α-Synuclein
- ClinicalTrials.gov ID
- NCT02281474
- Acronym / sponsor ID
- IIT-2014-001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Georgetown UniversityUniversity, hospital or other
- Study start
- Nov 2014
- Primary completion
- May 2015 (actual)
- First posted
- 3 Nov 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This pilot study will test Nilotinib's ability to alter the abnormal protein build up in Parkinson disease and Diffuse Lewey Body Disease patients . Patients will receive Nilotinib at different doses for 6 months. Patients will then be tested to see if there is change in three areas: 1) has the disease symptoms changed. 2) has levels of a specific misfolded protein changed in the fluid around their brain and spine. 3) Have inflammatory markers changed in the patient's blood and fluid around their brain and spine. If successful, this drug could be used to slow down or stop the progression of disorders that involve abnormal collection of misfolded proteins. However, the main purpose of this pilot study is to check for the safety of using this medication at this level.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 150mg dosing | Active comparator | Drug: NilotinibThis arm will take 150mg of Nilotinib by mouth daily for the 6 month drug period to establish a safe and efficacious dose. |
| 300mg dosing | Active comparator | Drug: NilotinibThis arm will take 300mg of Nilotinib by mouth daily for the 6 month drug period to establish a safe and efficacious dose. |
Who may qualify
- Ages
- 40 to 90 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 1. Patients aged 40 to 90 with Idiopathic Parkinson's Disease (Significant Sinemet response) on a stable medication drug regimen L-dopa and/or Dopamine agonist (at least 1 month before enrollment with no new medication change) and with moderate to severe cognitive impairment (MOCA ≤24).
- Written informed consent
- Capability and willingness to comply with the study related criteria
- Patients between the age of 40-90 y
- Diagnosis of PD according to the UK Brain Bank Diagnostic Criteria
Key exclusion criteria (14 listed)
- Patients with a known genetic form of PD that does not involve alpha-synuclein.
- Unwillingness to undergo lumbar punctures
- Immeasurable CSF α-synuclein.
- Presence of dementia or severe cognitive impairment that would not permit the patient to give adequate feedback for potential side effects.
- Unwilling to be in an off state for UPDRS assessment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NilotinibAlso: Tasigna | Drug | 150mg dosing, 300mg dosing |
Primary outcome
Change in α-synuclein and Tau concentrations in the CSF and serum of patients
Time frame: 6 months
Working Hypothesis: PD patients have been shown to have elevated levels of α-synuclein in their CSF. Nilotinib has been shown to reduce α-synuclein and Tau in the gastrointestinal tract and central nervous system in animal models, and similarly, we propose will show changes in CSF and serum α-synuclein concentrations in nilotinib treated PD patients.
2 secondary outcomes
Determine nilotinib's efficacy by improvement in motor and non-motor symptoms
Time frame: 6 months
Safety and tolerability, as measured by number of Participants with Adverse Events
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 2015.
- Derived from this study: PubMed 40680102Joshi D, Kulkarni M, Parekh P, Shah S, Greig NH, Acharya S. Targeting protein kinases in Parkinson's disease: the emerging role of phytoconstituents. Nutr Neurosci. 2025 Dec;28(12):1532-1563. doi: 10.1080/1028415X.2025.2531356. Epub 2025 Jul 18.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nilotinib in people with Parkinson's Disease and Parkinson's Disease Dementia, run by Georgetown University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in α-synuclein and Tau concentrations in the CSF and serum of patients (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02281474, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Dec 2015.