Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study of Pexidartinib in Asian Subjects With Advanced Solid Tumors

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: NCT02734433
Recruitment Status : Active, not recruiting
First Posted : April 12, 2016
Results First Posted : April 24, 2020
Last Update Posted : November 9, 2020
Sponsor:
Information provided by (Responsible Party):
Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. )

Study Type Interventional
Study Design Intervention Model: Sequential Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Advanced Solid Tumors
Intervention Drug: Pexidartinib
Enrollment 12
Recruitment Details A total of 12 participants who met all inclusion and no exclusion criteria were enrolled in the study; 11 participants were treated.
Pre-assignment Details The study was conducted in a dose-escalation 3 + 3 design and was comprised of 2 dose levels (Cohort 1 [600 mg/day] and Cohort 2 [1000 mg/day]). Selection of the pexidartinib dose of 1000 mg/day as the highest dose and the split-dose administration schedule was based on data from a Phase 1 study on patients with solid tumors (Study PLX108-01).
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening). Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Period Title: Overall Study
Started 3 8
Completed 1 0
Not Completed 2 8
Reason Not Completed
Disease progression             1             5
Clinical progression             0             1
Withdrawal by Subject             1             2
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day Total
Hide Arm/Group Description Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening). Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening). Total of all reporting groups
Overall Number of Baseline Participants 3 8 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 11 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
1
  33.3%
5
  62.5%
6
  54.5%
>=65 years
2
  66.7%
3
  37.5%
5
  45.5%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 8 participants 11 participants
55.0  (27.9) 62.4  (12.2) 60.4  (16.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 11 participants
Female
1
  33.3%
4
  50.0%
5
  45.5%
Male
2
  66.7%
4
  50.0%
6
  54.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 8 participants 11 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
3
 100.0%
8
 100.0%
11
 100.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
0
   0.0%
0
   0.0%
0
   0.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Taiwan Number Analyzed 3 participants 8 participants 11 participants
3 8 11
1.Primary Outcome
Title Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Hide Description For the assessment of tumor response, participants were classified into the best of the following tumor response categories by RECIST v1.1: complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; stable disease (SD); neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease, taking as reference the smallest sum diameters while on study; progressive disease (PD), at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study and the sum must also demonstrate an absolute increase of at least 5 mm; or not evaluable (NE) for analysis. Objective response rate was defined as CR or PR and disease control rate was defined as CR, PR, or SD.
Time Frame Day 1 through Day 28 after last dose (within 18 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Best overall response was assessed in the Efficacy Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 5
Measure Type: Number
Unit of Measure: Number of participants
Complete response (CR) 0 0
Partial response (PR) 1 0
Stable disease (SD) 1 3
Progressive disease (PD) 1 2
Not evaluable (NE) 0 0
Objective response rate (CR or PR) 1 0
Disease control rate (CR or PR or SD) 2 3
2.Secondary Outcome
Title Duration of Response or Duration of Stable Disease Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Hide Description [Not Specified]
Time Frame Day 1 through Day 28 after last dose (within 18 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Duration of response or duration of stable disease was assessed in the Efficacy Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 1 3
Mean (Standard Deviation)
Unit of Measure: months
Duration of response (n=1,0) Number Analyzed 1 participants 0 participants
7.62  (0)
Duration of stable disease (n=1,3) Number Analyzed 1 participants 3 participants
1.87  (0) 4.62  (1.6)
3.Secondary Outcome
Title A Summary of Pexidartinib Pharmacokinetic Parameter (Cmax) by Cohort and Day Following Oral Doses of Pexidartinib
Hide Description Maximum concentration (Cmax) of pexidartinib was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 3 participants 8 participants
3210  (1320) 3650  (1170)
Day 15 Number Analyzed 3 participants 7 participants
8490  (1430) 10800  (2860)
4.Secondary Outcome
Title A Summary of Pexidartinib Pharmacokinetic Parameter (AUC[0-8h]) by Cohort and Day Following Oral Doses of Pexidartinib
Hide Description Area under the curve from 0 to 8 hours (AUC[0-8h]) was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: ng*h/mL
Day 1 Number Analyzed 3 participants 8 participants
13300  (4720) 16500  (4760)
Day 15 Number Analyzed 3 participants 7 participants
50900  (18300) 64600  (15900)
5.Secondary Outcome
Title A Summary of Pexidartinib Pharmacokinetic Parameter (Tmax) by Cohort and Day Following Oral Doses of Pexidartinib
Hide Description Time at maximum pexidartinib concentration (Tmax) was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: hours
Day 1 Number Analyzed 3 participants 8 participants
3.95  (3.51) 2.48  (1.18)
Day 15 Number Analyzed 3 participants 7 participants
1.00  (1.00) 1.26  (0.94)
6.Secondary Outcome
Title A Summary of Pexidartinib Pharmacokinetic Parameter (R[AUC]) by Cohort Following Oral Doses of Pexidartinib
Hide Description Accumulation ratio of area under the curve (R[AUC]) was assessed.
Time Frame Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: accumulation ratio of AUC
4.29  (2.29) 4.58  (2.23)
7.Secondary Outcome
Title A Summary of Pexidartinib Pharmacokinetic Parameter (R[Cmax]) by Cohort Following Oral Doses of Pexidartinib
Hide Description Accumulation ratio of maximum concentration of pexidartinib (R[Cmax]) was assessed.
Time Frame Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: accumulation ratio of Cmax
2.94  (1.19) 4.58  (2.23)
8.Secondary Outcome
Title A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (AUC[0-8h]) Following Oral Doses of Pexidartinib
Hide Description Area under the curve from 0 to 8 hours (AUC[0-8h]) was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: ng*h/mL
Day 1 Number Analyzed 3 participants 8 participants
14800  (8010) 15600  (9130)
Day 15 Number Analyzed 3 participants 7 participants
91600  (37500) 120000  (61100)
9.Secondary Outcome
Title A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Cmax) Following Oral Doses of Pexidartinib
Hide Description Maximum concentration (Cmax) of pexidartinib was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 3 participants 8 participants
2930  (935) 3200  (1630)
Day 15 Number Analyzed 3 participants 7 participants
13200  (5120) 17800  (9630)
10.Secondary Outcome
Title A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Tmax) Following Oral Doses of Pexidartinib
Hide Description Time at maximum pexidartinib concentration (Tmax) was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: hours
Day 1 Number Analyzed 3 participants 8 participants
6.55  (2.24) 5.00  (2.87)
Day 15 Number Analyzed 3 participants 7 participants
1.5  (2.18) 1.43  (0.87)
11.Secondary Outcome
Title A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR AUC[0-8h]) Following Oral Doses of Pexidartinib
Hide Description Metabolite to parent drug ratio of area under the curve from 0-8 h (MR AUC[0-8h]) was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: metabolite to parent drug ratio
Day 1 Number Analyzed 3 participants 8 participants
0.801  (0.454) 0.658  (0.298)
Day 15 Number Analyzed 3 participants 7 participants
1.27  (0.416) 1.33  (0.711)
12.Secondary Outcome
Title A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR Cmax) Following Oral Doses of Pexidartinib
Hide Description Metabolite to parent drug ratio of maximum pexidartinib concentration (MR Cmax) was assessed.
Time Frame Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Mean (Standard Deviation)
Unit of Measure: metabolite to parent drug ratio
Day 1 Number Analyzed 3 participants 8 participants
0.669  (0.189) 0.639  (0.314)
Day 15 Number Analyzed 3 participants 7 participants
1.10  (0.440) 1.18  (0.695)
13.Secondary Outcome
Title Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Hide Description [Not Specified]
Time Frame Baseline up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Adverse events were assessed in the Safety Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Measure Type: Count of Participants
Unit of Measure: Participants
Any TEAE
3
 100.0%
8
 100.0%
Chronic gastritis
0
   0.0%
1
  12.5%
Constipation
0
   0.0%
2
  25.0%
Diarrhoea
0
   0.0%
3
  37.5%
Nausea
0
   0.0%
1
  12.5%
Stomatitis
0
   0.0%
1
  12.5%
Face oedema
0
   0.0%
1
  12.5%
Fatigue
1
  33.3%
3
  37.5%
Malaise
1
  33.3%
0
   0.0%
Alanine aminotransferase increased
1
  33.3%
3
  37.5%
Aspartate aminotransferase increased
2
  66.7%
3
  37.5%
Blood alkaline phosphatase increased
1
  33.3%
3
  37.5%
Blood bilirubin increased
1
  33.3%
1
  12.5%
Blood cholesterol increased
1
  33.3%
0
   0.0%
Gamma-glutamyltransferase increased
0
   0.0%
2
  25.0%
Weight increased
1
  33.3%
0
   0.0%
Dysuria
0
   0.0%
1
  12.5%
Proteinuria
1
  33.3%
0
   0.0%
Urinary retention
0
   0.0%
1
  12.5%
Cough
1
  33.3%
2
  25.0%
Haemoptysis
0
   0.0%
1
  12.5%
Hair colour changes
2
  66.7%
2
  25.0%
Pruritus generalised
1
  33.3%
0
   0.0%
Rash generalised
1
  33.3%
0
   0.0%
Skin hypopigmentation
0
   0.0%
1
  12.5%
14.Secondary Outcome
Title Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Hide Description [Not Specified]
Time Frame Baseline up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Adverse events were assessed in the Safety Analysis Set.
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description:
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
Overall Number of Participants Analyzed 3 8
Measure Type: Count of Participants
Unit of Measure: Participants
Any treatment-related TEAE
3
 100.0%
6
  75.0%
Diarrhoea
0
   0.0%
3
  37.5%
Face oedema
0
   0.0%
1
  12.5%
Fatigue
1
  33.3%
3
  37.5%
Alanine aminotransferase increased
1
  33.3%
3
  37.5%
Aspartate aminotransferase increased
2
  66.7%
3
  37.5%
Blood alkaline phosphatase increased
1
  33.3%
3
  37.5%
Blood bilirubin increased
1
  33.3%
0
   0.0%
Blood cholesterol increased
1
  33.3%
0
   0.0%
Gamma-glutamyltransferase increased
0
   0.0%
2
  25.0%
Proteinuria
1
  33.3%
0
   0.0%
Hair colour changes
1
  33.3%
2
  25.0%
Pruritus generalised
1
  33.3%
0
   0.0%
Rash generalised
1
  33.3%
0
   0.0%
Skin hypopigmentation
0
   0.0%
1
  12.5%
Time Frame Adverse event data were collected after the first dose and 28 days after the last dose, up to 18 months postdose.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Hide Arm/Group Description Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening). Participants who received pexidartinib 1000 mg/day (400 mg in the morning and 600 mg in the evening) for the first 2 weeks. Thereafter, the dose was reduced to 800 mg/day (400 mg in the morning and 400 mg in the evening).
All-Cause Mortality
Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)   1/8 (12.50%) 
Hide Serious Adverse Events
Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Affected / at Risk (%) Affected / at Risk (%)
Total   1/3 (33.33%)   1/8 (12.50%) 
Blood and lymphatic system disorders     
Anaemia  1  0/3 (0.00%)  1/8 (12.50%) 
Investigations     
Alanine aminotransferase increased  1  1/3 (33.33%)  0/8 (0.00%) 
Aspartate aminotransferase increased  1  1/3 (33.33%)  0/8 (0.00%) 
Blood alkaline phosphatase increased  1  1/3 (33.33%)  0/8 (0.00%) 
Blood bilirubin increased  1  1/3 (33.33%)  0/8 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Malignant neoplasm progression  1  0/3 (0.00%)  1/8 (12.50%) 
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1 - 600 mg/Day Cohort 2 - 1000 mg/Day
Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   8/8 (100.00%) 
Blood and lymphatic system disorders     
Anaemia  1  0/3 (0.00%)  2/8 (25.00%) 
Eye disorders     
Dacryostenosis acquired  1  0/3 (0.00%)  1/8 (12.50%) 
Gastrointestinal disorders     
Diarrhoea  1  0/3 (0.00%)  3/8 (37.50%) 
Constipation  1  0/3 (0.00%)  2/8 (25.00%) 
Chronic gastritis  1  0/3 (0.00%)  1/8 (12.50%) 
Nausea  1  0/3 (0.00%)  1/8 (12.50%) 
Stomatitis  1  0/3 (0.00%)  1/8 (12.50%) 
General disorders     
Fatigue  1  1/3 (33.33%)  3/8 (37.50%) 
Face oedema  1  0/3 (0.00%)  1/8 (12.50%) 
Malaise  1  1/3 (33.33%)  0/8 (0.00%) 
Infections and infestations     
Conjunctivitis  1  0/3 (0.00%)  1/8 (12.50%) 
Investigations     
Aspartate aminotransferase increased  1  2/3 (66.67%)  3/8 (37.50%) 
Alanine aminotransferase increased  1  1/3 (33.33%)  3/8 (37.50%) 
Blood alkaline phosphatase increased  1  1/3 (33.33%)  3/8 (37.50%) 
Blood bilirubin increased  1  1/3 (33.33%)  1/8 (12.50%) 
Gamma-glutamyltransferase increased  1  0/3 (0.00%)  2/8 (25.00%) 
Blood cholesterol increased  1  1/3 (33.33%)  0/8 (0.00%) 
Weight increased  1  1/3 (33.33%)  0/8 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain  1  0/3 (0.00%)  2/8 (25.00%) 
Pain in extremity  1  0/3 (0.00%)  1/8 (12.50%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Malignant neoplasm progression  1  0/3 (0.00%)  1/8 (12.50%) 
Renal and urinary disorders     
Dysuria  1  0/3 (0.00%)  1/8 (12.50%) 
Proteinuria  1  1/3 (33.33%)  0/8 (0.00%) 
Urinary retention  1  0/3 (0.00%)  1/8 (12.50%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  1/3 (33.33%)  2/8 (25.00%) 
Haemoptysis  1  0/3 (0.00%)  1/8 (12.50%) 
Skin and subcutaneous tissue disorders     
Hair colour changes  1  2/3 (66.67%)  2/8 (25.00%) 
Pruritus generalised  1  1/3 (33.33%)  0/8 (0.00%) 
Rash generalised  1  1/3 (33.33%)  0/8 (0.00%) 
Skin hypopigmentation  1  0/3 (0.00%)  1/8 (12.50%) 
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by 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: Contact for Clinical Trial Information
Organization: Daiichi Sankyo
Phone: 908-992-6400
EMail: CTRinfo@dsi.com
Layout table for additonal information
Responsible Party: Daiichi Sankyo, Inc. ( Daiichi Sankyo Co., Ltd. )
ClinicalTrials.gov Identifier: NCT02734433    
Other Study ID Numbers: PL3397-A-A103
First Submitted: March 22, 2016
First Posted: April 12, 2016
Results First Submitted: March 23, 2020
Results First Posted: April 24, 2020
Last Update Posted: November 9, 2020