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COMPLETED
NCT02954978
PHASE2

Impact of Nilotinib on Safety, Tolerability, Pharmacokinetics and Biomarkers in Parkinson's Disease

Sponsor: Georgetown University

View on ClinicalTrials.gov

Summary

Parkinson's disease (PD) is the second most common neurodegenerative disorder causing motor and non-motor symptoms. PD is characterized by death of dopaminergic (DA) neurons in the substantia nigra (SN) pars compacta and formation of inclusions known as Lewy bodies (LBs) that primarily contain aggregated alpha-Synuclein. Nilotinib (Tasigna®, AMN107, Novartis, Switzerland) is approved by U.S. Food and Drug Administration (FDA) and is well tolerated for CML treatment at oral doses of 600-800mg daily. Nilotinib penetrates the brain and promotes autophagic degradation of alpha-Synuclein and p-Tau, leading to survival of DA neurons and improvement of motor function in PD models. For these studies, Nilotinib (1-10mg/kg daily) was used at significantly less than the clinically approved dose (up to 1200mg daily) in CML.

Official title: A Randomized, Double Blind, Placebo-controlled Study to Evaluate the Impact of Low Doses of Nilotinib Treatment on Safety, Tolerability, Pharmacokinetics and Biomarkers in Parkinson's Disease

Key Details

Gender

All

Age Range

40 Years - 90 Years

Study Type

INTERVENTIONAL

Enrollment

75

Start Date

2017-01

Completion Date

2020-07

Last Updated

2026-06-12

Healthy Volunteers

No

Interventions

DRUG

Placebo Oral Capsule

25 patients in group 1 will receive Placebo ("sugar pill") one (1) capsule once daily (taken without a meal) for 12 months and 3 months follow up.

DRUG

Nilotinib 150mg oral capsule [Tasigna]

25 patients in group 2 will receive 150mg Nilotinib one (1) capsule once daily (taken without a meal) for 12 months and 3 months follow up.

DRUG

Nilotinib 300mg oral capsule [Tasigna]

25 patients in group 3 will receive 300mg Nilotinib one (1) capsule once daily (taken without a meal) for 12 months and 3 months follow up.

Locations (1)

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, United States