Nilotinib in Cognitively Impaired Parkinson Disease Patients 001

Completed · Phase 1

Conditions studied: Parkinson's Disease, Parkinson's Disease Dementia, Diffuse Lewy Body Disease

In brief

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.

Key facts

Study ID: NCT02281474
Run by: Georgetown University
People needed: 12
Starts: 2014-11-01
Last updated by the study team: 2015-12-16

Who can join

Age: 40 and older, up to 90. Sex: any. Healthy volunteers: accepted.

You may qualify if…

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
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

You may not qualify if…

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
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.

Where it is running

MedStar Georgetown University Hospital — Washington D.C., District of Columbia, United States

Full record on ClinicalTrials.gov

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