Nilotinib in Parkinson's Disease (NILO-PD)
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|ClinicalTrials.gov Identifier: NCT03205488|
Recruitment Status : Recruiting
First Posted : July 2, 2017
Last Update Posted : February 22, 2018
|Condition or disease||Intervention/treatment||Phase|
|Parkinson Disease||Drug: Cohort 1:Nilotinib Oral Capsules (150mg or 300mg) Drug: Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1) Drug: Placebo||Phase 2|
The purpose of this study is to determine if nilotinib is safe, if it can be tolerated by patients with Parkinson's disease (PD) and to learn if nilotinib has the possibility of effectively treating PD symptoms. Nilotinib has been approved by the Food and Drug Administration (FDA) to treat certain types of cancer (leukemia) but is considered investigational in this study because it has not been approved for treating PD. Twenty-five sites will enroll participants into 2 cohorts,approximately 75 in Cohort 1 and 60 in Cohort 2. Participants with moderate to advanced PD symptoms will be enrolled in Cohort 1, randomly assigned to take nilotinib (150 mg or 300mg) or placebo, and will complete 13 in-person study visits over 8.5 months.
The results from Cohort 1 will determine if either dose of nilotinib (150mg or 300 mg) is safe and tolerable enough to move forward and evaluate in Cohort 2. If either dose is found to be safe and tolerable, participants with early PD will be enrolled into Cohort 2.
Participants in Cohort 2 will be randomly assigned to either nilotinib (dose to be determined from Cohort 1 results) or placebo and will complete 17 in-person visits over 14.5 months. For both cohorts, the study visits will include clinical assessment of motor, neuropsychiatric and cognitive testing as well as collection of blood and cerebral spinal fluid, collected by lumbar puncture.
This study will also evaluate if nilotinib can help improve motor symptoms associated with PD. All participants in Cohort 1 and participants in Cohort 2 who have started PD medications will have an assessment of the motor exam (Part III) in a practically defined OFF state (12 hours post dose) and ON state (at least one-hour post dose).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||135 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled, Phase IIa, Parallel Group, Two Cohort Study to Define the Safety, Tolerability, Clinical and Exploratory Biological Activity of the Chronic Administration of Nilotinib in Participants With Parkinson's Disease|
|Actual Study Start Date :||October 16, 2017|
|Estimated Primary Completion Date :||October 2020|
|Estimated Study Completion Date :||October 2020|
Active Comparator: Cohort 1
Moderate to Advanced PD Population Randomized 1:1:1
Drug: Cohort 1:Nilotinib Oral Capsules (150mg or 300mg)
2 capsules taken once dailyDrug: Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1)
2 capsules taken once daily
Active Comparator: Cohort 2
Early/de novo Randomized 2:1
Drug: Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1)
2 capsules taken once dailyDrug: Placebo
2 capsules taken once daily
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Cohort 1 = 6 months, Cohort 2 = 12 months ]the frequency of treatment-related serious adverse events across all groups within each cohort
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Cohort 1 = 6 months, Cohort 2 = 12 months ]The ability to complete the study on the assigned dose
- Motor Function [ Time Frame: 6 months ]Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part III (motor function) will be evaluated to determine if Nilotinib has the potential to reduce the MDS-UPDRS motor score.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03205488
|Contact: Tina Ward, MS||877-483-1834||NILO_STUDY@chet.rochester.edu|
Show 25 Study Locations
|Principal Investigator:||Tanya Simuni, MD||Northwestern University|