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Trial record 2 of 3 for:    Nilotinib | Parkinson Disease

Nilotinib in Parkinson's Disease (NILO-PD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03205488
Recruitment Status : Completed
First Posted : July 2, 2017
Results First Posted : June 18, 2020
Last Update Posted : July 22, 2020
Sponsor:
Collaborators:
University of Rochester
University of Iowa
Michael J. Fox Foundation for Parkinson's Research
Information provided by (Responsible Party):
Tanya Simuni, Northwestern University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Parkinson Disease
Interventions Drug: Cohort 1:Nilotinib Oral Capsules (150mg or 300mg)
Drug: Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1)
Drug: Placebo
Enrollment 76
Recruitment Details From November 2017 to December 2018, 125 patients were assessed for eligibility. Of those screened, 76 participants were enrolled in the trial.
Pre-assignment Details Of the patients assessed for eligibility, 49 (39%) were excluded. 42 patients did not meet inclusion criteria and 7 declined study participation.
Arm/Group Title Placebo Nilotinib 150 Nilotinib 300
Hide Arm/Group Description Placebo enclosed in capsules to maintain the blind was administered orally once per day over the course of 6 months. Patients were administered 1 capsule of 150 mg Nilotinib and 1 capsule of matching placebo each day over the course of 6 months. Patients were administered 2 capsules of 150 mg Nilotinib each day over the course of 6 months.
Period Title: Overall Study
Started 25 25 26
Completed 24 23 21
Not Completed 1 2 5
Reason Not Completed
Adverse Event             1             2             4
Protocol Violation             0             0             1
Arm/Group Title Placebo Nilotinib 150 Nilotinib 300 Total
Hide Arm/Group Description Placebo enclosed in capsules to maintain the blind was administered orally once per day over the course of 6 months. Patients were administered 1 capsule of 150 mg Nilotinib and 1 capsule of matching placebo each day over the course of 6 months. Patients were administered 2 capsules of 150 mg Nilotinib each day over the course of 6 months. Total of all reporting groups
Overall Number of Baseline Participants 25 25 26 76
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants 25 participants 26 participants 76 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
13
  52.0%
16
  64.0%
9
  34.6%
38
  50.0%
>=65 years
12
  48.0%
9
  36.0%
17
  65.4%
38
  50.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 25 participants 25 participants 26 participants 76 participants
65.5  (6.8) 61.2  (7.4) 66.9  (7.3) 64.6  (7.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants 25 participants 26 participants 76 participants
Female
9
  36.0%
10
  40.0%
5
  19.2%
24
  31.6%
Male
16
  64.0%
15
  60.0%
21
  80.8%
52
  68.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants 25 participants 26 participants 76 participants
Hispanic or Latino
1
   4.0%
0
   0.0%
1
   3.8%
2
   2.6%
Not Hispanic or Latino
24
  96.0%
25
 100.0%
25
  96.2%
74
  97.4%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants 25 participants 26 participants 76 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
24
  96.0%
23
  92.0%
25
  96.2%
72
  94.7%
More than one race
1
   4.0%
1
   4.0%
0
   0.0%
2
   2.6%
Unknown or Not Reported
0
   0.0%
1
   4.0%
1
   3.8%
2
   2.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 25 participants 25 participants 26 participants 76 participants
25 25 26 76
Parkinson’s Disease History  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 25 participants 25 participants 26 participants 76 participants
Parkinson’s Disease Duration 9.4  (4.9) 8.5  (3.2) 11.7  (5.2) 9.9  (4.7)
Age at Diagnosis 56.2  (6.8) 52.7  (7.6) 55.2  (9.3) 54.7  (8.0)
MDS-UPDRS Total Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
MDS-UPDRS Total OFF Score 63.8  (21.3) 65.0  (16.0) 70.2  (20.2) 66.4  (19.3)
MDS-UPDRS Total ON Score 46.2  (17.8) 46.9  (15.1) 51.9  (15.7) 48.4  (16.2)
[1]
Measure Description: Measure Description: Baseline total Unified Parkinson Disease Rating Scale (UPDRS) score ranges from 0 to 295. The higher the value the more disability from PD the participant has.
Parkinson’s Disease Classifications   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants 25 participants 26 participants 76 participants
Hoehn and Yahr Stage 0-2
4
  16.0%
5
  20.0%
1
   3.8%
10
  13.2%
Hoehn and Yahr Stage 3
21
  84.0%
20
  80.0%
25
  96.2%
66
  86.8%
Class of Symptomatic Therapy, MAOB Inhibitors
11
  44.0%
8
  32.0%
12
  46.2%
31
  40.8%
[1]
Measure Description: Measure Description: Hoehn and Yahr staging of severity of Parkinson's disease. Baseline H/Y stage ranges from >2 to <4. Larger value stands for more disability from PD. H&Y 2 = Bilateral involvement without impairment of balance. H&Y 3 = Mild to moderate involvement; some postural instability but physically independent; needs assistance to recover from pull test. 4 = Severe disability; still able to walk or stand unassisted.
Levodopa Equivalent Daily Dose  
Mean (Standard Deviation)
Unit of measure:  Mg
Number Analyzed 25 participants 25 participants 26 participants 76 participants
1066.5  (519.7) 971.8  (251.4) 1012.5  (390.5) 1016.9  (398.7)
Education Adjusted MoCA Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
26.9  (2.4) 27.4  (1.9) 27.0  (2.4) 27.1  (2.2)
[1]
Measure Description: Measure Description: Montreal Cognitive Assessment (MoCA) is one-page 30-point test to detect cognitive impairment. Baseline MoCA score ranges from 23 to 30. Higher scores indicate better cognition.
DRS-2   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
138.1  (5.7) 137.8  (7.8) 137.7  (6.3) 137.9  (6.6)
[1]
Measure Description: Measure Description: Mattis Dementia Rating Scale 2 (DRS-2) is a test to screen for dementia in Parkinson's disease. Five subscales provide additional information on specific abilities: Attention, Initiation/Perseveration, Construction, Conceptualization, and Memory. DRS-2 score range is 0-144. Higher scores indicate better cognition.
BDI-II   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
6.6  (4.9) 7.3  (5.4) 6.6  (4.3) 6.8  (4.8)
[1]
Measure Description: Measure Description: Baseline Beck Depression Inventory (BDI) score ranges from 0 to 23. Higher scores indicate greater symptom severity.
PDSS   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
106.6  (21.8) 115.3  (11.6) 103.2  (18.6) 108.3  (18.3)
[1]
Measure Description: Measure Description: Parkinson Disease Sleep Scale self-rate and quantify the level of sleep disruption being experienced in order to target treatment appropriately. Scores range from 0 to 150. Higher scores indicate greater symptom severity.
Modified S/E ADL   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
Modified S/E ADL OFF 73.2  (16.5) 77.3  (17.8) 77.0  (14.9) 75.8  (16.8)
Modified S/E ADL ON 86.8  (8.7) 92.2  (5.8) 87.0  (17.9) 88.6  (12.2)
[1]
Measure Description: Measure Description: Schwab and England Activities of Daily Living scale. Baseline SE/ADL ranges from 40 to 100. Smaller value stands for more disability from PD.
PDQ-39   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
19.0  (12.7) 19.0  (9.4) 18.4  (10.3) 18.8  (10.8)
[1]
Measure Description: Measure Description: Parkinson's Disease Questionnaire-39 is a 39-item self-report questionnaire, which assesses Parkinson's disease-specific health related quality over the past month. Baseline PDQ 39 score ranges from 0 to 52. Lower score shows better quality of life.
EQ-5D   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 25 participants 25 participants 26 participants 76 participants
EQ-5D Summary Index 0.79  (0.14) 0.83  (0.13) 0.78  (0.15) 0.80  (0.14)
EQ-5D Health Score 70.6  (25.1) 71.5  (23.3) 76.0  (15.9) 72.8  (21.5)
[1]
Measure Description: Measure Description: EQ-5D summary index is derived by applying a formula that essentially attaches values (weights) to each of the levels in the 5 dimensions. The total index range is from 0-1. The Health Score range is 0-100 point scale where the participant marks his or her health related quality that day. Higher scores show better quality of life.
1.Primary Outcome
Title Tolerability of Nilotinib Over Placebo
Hide Description The count of study participants who completed the 6-month study treatment period while active on their original assigned dose
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat population, all randomized subjects per arm. The Count of participants below are the number of participants that met the criteria for analysis of tolerability.
Arm/Group Title Placebo Nilotinib 150 mg Nilotinib 300 mg
Hide Arm/Group Description:
Placebo enclosed in capsules to maintain the blind was administered orally once per day over the course of 6 months
Patients were administered 1 capsule of 150 mg Nilotinib and 1 capsule of matching placebo each day over the course of 6 months.
Patients were administered 2 capsules of 150 mg Nilotinib each day over the course of 6 months.
Overall Number of Participants Analyzed 25 25 26
Measure Type: Count of Participants
Unit of Measure: Participants
21
  84.0%
19
  76.0%
20
  76.9%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3626
Comments [Not Specified]
Method Fisher Exact
Comments One-sided Fisher’s Exact tested the proportion of participants who met tolerability between the Nilotinib 150 group to the Placebo group.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 300 mg
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3895
Comments [Not Specified]
Method Fisher Exact
Comments One-sided Fisher’s Exact tested the proportion of participants who met tolerability between the Nilotinib 300 group to the Placebo group.
2.Primary Outcome
Title Safety of Nilotinib
Hide Description The count of study participants who experienced any treatment-related SAE in each treatment group
Time Frame We assessed adverse events that were collected from the first dose of study drug until 60 days after the participant's last dose.
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat population, all randomized subjects. The counts refer to the number of participants that experienced an SAE in each treatment arm.
Arm/Group Title Placebo Nilotinib 150 Nilotinib 300
Hide Arm/Group Description:
Placebo enclosed in capsules to maintain the blind was administered orally once per day over the course of 6 months.
Patients were administered 1 capsule of 150 mg Nilotinib and 1 capsule of matching placebo each day over the course of 6 months.
Patients were administered 2 capsules of 150 mg Nilotinib each day over the course of 6 months.
Overall Number of Participants Analyzed 25 25 26
Measure Type: Count of Participants
Unit of Measure: Participants
2
   8.0%
1
   4.0%
1
   3.8%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150
Comments [Not Specified]
Type of Statistical Test Equivalence
Comments H0 : p1 = p2, where p represents the proportion of SAEs for each group. HA : p1 ≠ p2
Statistical Test of Hypothesis P-Value 1.00
Comments [Not Specified]
Method Fisher Exact
Comments Fisher’s Exact test compared a proportion of participants who experienced any serious adverse event in the Nilotinib 150 group and the Placebo group.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150
Comments [Not Specified]
Type of Statistical Test Equivalence
Comments H0 : λ1 = λ2, where λ represents the rate of SAEs for each group. HA : λ1 ≠ λ2
Statistical Test of Hypothesis P-Value 0.5689
Comments [Not Specified]
Method Regression, Poisson
Comments Rates of serious adverse event between the Nilotinib 150 group and the placebo were also compared using a Poisson regression model
Method of Estimation Estimation Parameter Risk Ratio (RR)
Estimated Value 0.4977
Confidence Interval (2-Sided) 95%
0.0451 to 5.489
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 300
Comments [Not Specified]
Type of Statistical Test Equivalence
Comments H0 : p1 = p3, where p represents the proportion of SAEs for each group. HA : p1 ≠ p3
Statistical Test of Hypothesis P-Value 0.61
Comments [Not Specified]
Method Fisher Exact
Comments Fisher’s Exact test compared a proportion of participants who experienced any serious adverse event in the Nilotinib 300 group and the Placebo group.
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 300
Comments [Not Specified]
Type of Statistical Test Equivalence
Comments H0 : λ1 = λ3, where λ represents the rate of SAEs for each group. HA : λ1 ≠ λ3
Statistical Test of Hypothesis P-Value 0.5940
Comments [Not Specified]
Method Regression, Poisson
Comments Rates of serious adverse event between the Nilotinib 300 group and the placebo were also compared using a Poisson regression model
Method of Estimation Estimation Parameter Risk Ratio (RR)
Estimated Value 0.5206
Confidence Interval (2-Sided) 95%
0.0472 to 5.7409
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change in MDS-UPDRS Part III
Hide Description The Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III is a motor examination in both practically defined medications OFF state (12 hrs post dose) and ON state (based on the participant/site investigator defined best ON and/or approximately 1 hour post dose). Measure Description: The part III subscale score ranges from 0-165. The Larger the value stands for more disability from PD.
Time Frame The MDS-UPDRS Part III ON state was collected at baseline, day 14, day 30, month 3, month 6, 30 and 60 days post treatment. The OFF state was collected at baseline, month 3, month 6, 30 and 60 days post treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Nilotinib 150 Nilotinib 300
Hide Arm/Group Description:
Placebo enclosed in capsules to maintain the blind was administered orally twice per day over the course of 6 months.
Patients were administered 1 capsule of 150 mg Nilotinib and 1 capsule of matching placebo each day over the course of 6 months.
Patients were administered 2 capsules of 150 mg Nilotinib each day over the course of 6 months.
Overall Number of Participants Analyzed 25 25 26
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline MDS-UPDRS Part III (ON) 22.80  (8.85) 24.76  (11.62) 25.15  (8.17)
Day 14 MDS-UPDRS Part III (ON) 23.13  (11.29) 27.58  (13.53) 29.33  (9.77)
Day 30 MDS-UPDRS Part III (ON) 22.36  (10.00) 26.84  (13.08) 29.73  (10.83)
Month 3 MDS-UPDRS Part III (ON) 19.83  (9.23) 24.88  (13.70) 25.76  (8.71)
Month 6 MDS-UPDRS Part III (ON) 19.63  (9.86) 24.43  (11.50) 25.61  (8.93)
30 Day Post MDS-UPDRS Part III (ON) 20.52  (8.82) 25.13  (12.26) 26.79  (9.10)
60 Day Post MDS-UPDRS Part III (ON) 20.88  (10.88) 23.91  (12.93) 26.42  (10.58)
Baseline MDS-UPDRS Part III (OFF) 40.36  (13.51) 42.84  (12.53) 43.50  (14.01)
Month 3 MDS-UPDRS Part III (OFF) 37.92  (14.46) 39.92  (12.41) 41.04  (13.38)
Month 6 MDS-UPDRS Part III (OFF) 36.21  (14.91) 41.91  (13.40) 43.96  (11.62)
30 Day Post MDS-UPDRS Part III (OFF) 38.92  (14.15) 41.48  (12.19) 45.26  (14.13)
60 Day Post MDS-UPDRS Part III (OFF) 37.36  (14.20) 40.27  (11.33) 43.00  (13.63)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150
Comments The key secondary objective is to conduct a futility analysis within each treatment group which examines the observed change in the MDS-UPDRS Part III ON within the two dose groups compared to previously reported changes from the Pagan et al (2016) study (NCT02281474).
Type of Statistical Test Non-Inferiority
Comments The hypotheses of interest (H0: δ ≤ -9.8 (7 x 1.4) vs. HA: δ > -9.8) where δ represents the change from baseline to 6 months using the MDS-UPDRS Part III ON in the Nilotinib 150 mg treatment group. The hypotheses of interest were evaluated based on an assessment of parameter estimates from a non-linear mixed effects model, adjusted for a participant’s Levodopa Equivalent Daily Dose (LEDD) at each time point.
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Slope
Estimated Value 1.05
Confidence Interval (1-Sided) 90%
-0.78
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 300
Comments The key secondary objective is to conduct a futility analysis within each treatment group which examines the observed change in the MDS-UPDRS Part III ON within the two dose groups compared to previously reported changes from the Pagan et al (2016) study (NCT02281474).
Type of Statistical Test Non-Inferiority
Comments The hypotheses of interest (H0: δ ≤ -9.8 (7 x 1.4) vs. HA: δ > -9.8) where δ represents the change from baseline to 6 months using the MDS-UPDRS Part III ON in the Nilotinib 300 mg treatment group. The hypotheses of interest were evaluated based on an assessment of parameter estimates from a non-linear mixed effects model, adjusted for a participant’s Levodopa Equivalent Daily Dose (LEDD) at each time point.
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Slope
Estimated Value 0.93
Confidence Interval (1-Sided) 90%
-0.89
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150, Nilotinib 300
Comments Additional secondary objective #1 is to establish the degree of symptomatic effect of Nilotinib as measured by the change in the MDS_UPDRS Part III ON score between baseline and 1 month.
Type of Statistical Test Equivalence
Comments H0 : β1 = β 2 = β 3, where β represents the slope for each group. HA : at least one β i ≠ β j. Using the same LMM as in the key secondary analysis, a two degree of freedom test was used to test for any differences in the slopes from baseline to 1 month for the three treatment groups.
Statistical Test of Hypothesis P-Value 0.031
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Other Statistical Analysis In order to assess which group may be driving these findings, pairwise comparisons were also utilized (Nilotinib 150 vs PBO at 1 Month, Nilotinib 300 vs PBO at 1 month, Nilotinib 300 vs Nilotinib 150 at 1 month).
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150, Nilotinib 300
Comments Additional secondary objective #2 is to establish the degree of symptomatic effect of Nilotinib as measured by the change in the MDS_UPDRS Part III ON score between the final visit on study drug and 30 days off study drug.
Type of Statistical Test Equivalence
Comments H0 : β1 = β 2 = β 3, where β represents the slope for each group. HA : at least one β i ≠ β j. For this analysis, a separate LMM was constructed, modeling the change from final visit on study drug to the 30 and 60 day follow up visits, while adjusting for the MDS-UPDRS Part III ON scores at the final visit on study drug as well as the Levodopa Equivalent Daily Dose (LEDD) at each visit.
Statistical Test of Hypothesis P-Value 0.47
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150, Nilotinib 300
Comments Additional secondary objective #3 is to assess the impact of Nilotinib on the progression of PD disability as measured by the change in the MDS_UPDRS Part III ON score between baseline and 6 months.
Type of Statistical Test Equivalence
Comments H0 : β1 = β 2 = β 3, where β represents the slope for each group. HA : at least one β i ≠ β j. Using the same LMM as in the key secondary analysis, a two degree of freedom test was used to test for any differences in the slopes from baseline to 6 months for the three treatment groups.
Statistical Test of Hypothesis P-Value 0.077
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Other Statistical Analysis Pairwise comparisons were also examined for trends (Active 150 vs PBO at 6 Months, Active 300 vs PBO at 6 months).
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Placebo, Nilotinib 150, Nilotinib 300
Comments Additional secondary objective #3 is to assess the impact of Nilotinib on the progression of PD disability as measured by the change in the MDS_UPDRS Part III OFF score between baseline and 6 months.
Type of Statistical Test Equivalence
Comments H0 : β1 = β 2 = β 3, where β represents the slope for each group. HA : at least one β i ≠ β j. Using a similar LMM as in the key secondary analysis, except simplified to only include baseline, month 3 and month 6, a two degree of freedom test was used to test for any differences in the slopes from baseline to 6 months for the three treatment groups.
Statistical Test of Hypothesis P-Value 0.17
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Time Frame Treatment emergent adverse events were collected following the first dose of study drug until 30 days after the participant’s last dose of study drug.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Nilotinib 150 Nilotinib 300
Hide Arm/Group Description Placebo enclosed in capsules to maintain the blind was administered orally once per day over the course of 6 months. Patients were administered 1 capsule of 150 mg Nilotinib and 1 capsule of matching placebo once per day over the course of 6 months. Patients were administered 2 capsules of 150 mg Nilotinib once per day over the course of 6 months.
All-Cause Mortality
Placebo Nilotinib 150 Nilotinib 300
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/25 (0.00%)      0/25 (0.00%)      0/26 (0.00%)    
Hide Serious Adverse Events
Placebo Nilotinib 150 Nilotinib 300
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/25 (8.00%)      1/25 (4.00%)      1/26 (3.85%)    
Cardiac disorders       
Arrhythmia *  0/25 (0.00%)  0 0/25 (0.00%)  0 1/26 (3.85%)  1
Gastrointestinal disorders       
Abdominal Pain *  1/25 (4.00%)  1 0/25 (0.00%)  0 0/26 (0.00%)  0
Gastrooesophageal reflux disease *  1/25 (4.00%)  1 0/25 (0.00%)  0 0/26 (0.00%)  0
Psychiatric disorders       
Suicidal ideation *  0/25 (0.00%)  0 1/25 (4.00%)  1 0/26 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Nilotinib 150 Nilotinib 300
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   22/25 (88.00%)      23/25 (92.00%)      23/26 (88.46%)    
Gastrointestinal disorders       
Abdominal pain upper *  2/25 (8.00%)  2 0/25 (0.00%)  0 0/26 (0.00%)  0
Constipation *  1/25 (4.00%)  1 2/25 (8.00%)  2 1/26 (3.85%)  1
Diarrhoea *  0/25 (0.00%)  0 2/25 (8.00%)  2 2/26 (7.69%)  2
Gastrooesophageal reflux disease *  3/25 (12.00%)  4 0/25 (0.00%)  0 0/26 (0.00%)  0
Nausea *  0/25 (0.00%)  0 2/25 (8.00%)  2 4/26 (15.38%)  5
General disorders       
Fatigue *  4/25 (16.00%)  5 4/25 (16.00%)  4 0/26 (0.00%)  0
Infections and infestations       
Nasopharyngitis *  5/25 (20.00%)  5 2/25 (8.00%)  2 2/26 (7.69%)  3
Urinary tract infection *  2/25 (8.00%)  3 1/25 (4.00%)  1 0/26 (0.00%)  0
Injury, poisoning and procedural complications       
Fall *  5/25 (20.00%)  11 4/25 (16.00%)  4 0/26 (0.00%)  0
Skin abrasion *  3/25 (12.00%)  4 0/25 (0.00%)  0 0/26 (0.00%)  0
Investigations       
Alanine aminotransferase increased *  2/25 (8.00%)  2 1/25 (4.00%)  1 0/26 (0.00%)  0
Amylase Increased *  2/25 (8.00%)  2 4/25 (16.00%)  4 4/26 (15.38%)  6
Blood bilirubin increased *  0/25 (0.00%)  0 0/25 (0.00%)  0 2/26 (7.69%)  2
CSF protein increased *  0/25 (0.00%)  0 0/25 (0.00%)  0 2/26 (7.69%)  2
Lipase increased *  4/25 (16.00%)  5 7/25 (28.00%)  11 6/26 (23.08%)  9
Musculoskeletal and connective tissue disorders       
Muscle spasms *  2/25 (8.00%)  2 2/25 (8.00%)  2 2/26 (7.69%)  2
Musculoskeletal stiffness *  0/25 (0.00%)  0 2/25 (8.00%)  2 0/26 (0.00%)  0
Myalgia *  3/25 (12.00%)  3 0/25 (0.00%)  0 0/26 (0.00%)  0
Pain in extremity *  1/25 (4.00%)  1 2/25 (8.00%)  2 0/26 (0.00%)  0
Nervous system disorders       
Dizziness *  1/25 (4.00%)  1 4/25 (16.00%)  4 0/26 (0.00%)  0
Dyskinesia *  1/25 (4.00%)  1 2/25 (8.00%)  2 0/26 (0.00%)  0
Headache *  2/25 (8.00%)  2 1/25 (4.00%)  1 3/26 (11.54%)  3
Tremor *  1/25 (4.00%)  2 2/25 (8.00%)  2 1/26 (3.85%)  1
Psychiatric disorders       
Anxiety *  0/25 (0.00%)  0 3/25 (12.00%)  3 1/26 (3.85%)  1
Skin and subcutaneous tissue disorders       
Alopecia *  1/25 (4.00%)  1 2/25 (8.00%)  2 0/26 (0.00%)  0
Vascular disorders       
Hypertension *  1/25 (4.00%)  1 2/25 (8.00%)  2 1/26 (3.85%)  1
Orthostatic hypotension *  0/25 (0.00%)  0 1/25 (4.00%)  1 2/26 (7.69%)  2
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Tanya Simuni
Organization: Northwestern University
Phone: 312-503-2970
EMail: Tatyana.Simuni@nm.org
Layout table for additonal information
Responsible Party: Tanya Simuni, Northwestern University
ClinicalTrials.gov Identifier: NCT03205488    
Other Study ID Numbers: NILO-PD
First Submitted: June 28, 2017
First Posted: July 2, 2017
Results First Submitted: May 28, 2020
Results First Posted: June 18, 2020
Last Update Posted: July 22, 2020