Trial Outcomes & Findings for Study of Pexidartinib in Asian Subjects With Advanced Solid Tumors (NCT NCT02734433)
NCT ID: NCT02734433
Last Updated: 2022-04-25
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
12 participants
Primary outcome timeframe
Day 1 through Day 28 after last dose (within 18 months)
Results posted on
2022-04-25
Participant Flow
A total of 12 participants who met all inclusion and no exclusion criteria were enrolled in the study; 11 participants were treated.
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).
Participant milestones
| Measure |
Cohort 1 - 600 mg/Day
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
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 Study
STARTED
|
3
|
8
|
|
Overall Study
COMPLETED
|
1
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
8
|
Reasons for withdrawal
| Measure |
Cohort 1 - 600 mg/Day
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
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 Study
Disease progression
|
1
|
5
|
|
Overall Study
Clinical progression
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
2
|
Baseline Characteristics
Study of Pexidartinib in Asian Subjects With Advanced Solid Tumors
Baseline characteristics by cohort
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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
n=11 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
55.0 years
STANDARD_DEVIATION 27.9 • n=5 Participants
|
62.4 years
STANDARD_DEVIATION 12.2 • n=7 Participants
|
60.4 years
STANDARD_DEVIATION 16.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Taiwan
|
3 participants
n=5 Participants
|
8 participants
n=7 Participants
|
11 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 through Day 28 after last dose (within 18 months)Population: Best overall response was assessed in the Efficacy Analysis Set.
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.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=5 Participants
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 Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Complete response (CR)
|
0 Number of participants
|
0 Number of participants
|
|
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Partial response (PR)
|
1 Number of participants
|
0 Number of participants
|
|
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Stable disease (SD)
|
1 Number of participants
|
3 Number of participants
|
|
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Progressive disease (PD)
|
1 Number of participants
|
2 Number of participants
|
|
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Not evaluable (NE)
|
0 Number of participants
|
0 Number of participants
|
|
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Objective response rate (CR or PR)
|
1 Number of participants
|
0 Number of participants
|
|
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Disease control rate (CR or PR or SD)
|
2 Number of participants
|
3 Number of participants
|
SECONDARY outcome
Timeframe: Day 1 through Day 28 after last dose (within 18 months)Population: Duration of response or duration of stable disease was assessed in the Efficacy Analysis Set.
Based on 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 sum of diameters of target lesions, with reference to baseline sum diameters; stable disease (SD); neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), with reference to smallest sum diameters; PD, at least a 20% increase in the sum of diameters of target lesions, with reference to smallest sum on study and sum must also demonstrate an absolute increase of at least 5 mm; Duration of response for responders (CR or PR) is defined as the time interval between the date of the earliest qualifying response and the date of PD or death for any cause, whichever occurs earlier. Duration of SD is defined as the time interval, in the absence of either CR or PR that will be calculated between the date of the first administration of the study drug and the date of PD.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=1 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=3 Participants
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).
|
|---|---|---|
|
Duration of Response or Duration of Stable Disease Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Duration of response (n=1,0)
|
7.62 months
Standard Deviation 0
|
—
|
|
Duration of Response or Duration of Stable Disease Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Duration of stable disease (n=1,3)
|
1.87 months
Standard Deviation 0
|
4.62 months
Standard Deviation 1.6
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Maximum concentration (Cmax) of pexidartinib was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (Cmax) by Cohort and Day Following Oral Doses of Pexidartinib
Day 1
|
3210 ng/mL
Standard Deviation 1320
|
3650 ng/mL
Standard Deviation 1170
|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (Cmax) by Cohort and Day Following Oral Doses of Pexidartinib
Day 15
|
8490 ng/mL
Standard Deviation 1430
|
10800 ng/mL
Standard Deviation 2860
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Area under the curve from 0 to 8 hours (AUC\[0-8h\]) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (AUC[0-8h]) by Cohort and Day Following Oral Doses of Pexidartinib
Day 1
|
13300 ng*h/mL
Standard Deviation 4720
|
16500 ng*h/mL
Standard Deviation 4760
|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (AUC[0-8h]) by Cohort and Day Following Oral Doses of Pexidartinib
Day 15
|
50900 ng*h/mL
Standard Deviation 18300
|
64600 ng*h/mL
Standard Deviation 15900
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Time at maximum pexidartinib concentration (Tmax) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (Tmax) by Cohort and Day Following Oral Doses of Pexidartinib
Day 1
|
3.95 hours
Standard Deviation 3.51
|
2.48 hours
Standard Deviation 1.18
|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (Tmax) by Cohort and Day Following Oral Doses of Pexidartinib
Day 15
|
1.00 hours
Standard Deviation 1.00
|
1.26 hours
Standard Deviation 0.94
|
SECONDARY outcome
Timeframe: Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Accumulation ratio of area under the curve (R\[AUC\]) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (R[AUC]) by Cohort Following Oral Doses of Pexidartinib
|
4.29 accumulation ratio of AUC
Standard Deviation 2.29
|
4.58 accumulation ratio of AUC
Standard Deviation 2.23
|
SECONDARY outcome
Timeframe: Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Accumulation ratio of maximum concentration of pexidartinib (R\[Cmax\]) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Pexidartinib Pharmacokinetic Parameter (R[Cmax]) by Cohort Following Oral Doses of Pexidartinib
|
2.94 accumulation ratio of Cmax
Standard Deviation 1.19
|
4.58 accumulation ratio of Cmax
Standard Deviation 2.23
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Area under the curve from 0 to 8 hours (AUC\[0-8h\]) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (AUC[0-8h]) Following Oral Doses of Pexidartinib
Day 1
|
14800 ng*h/mL
Standard Deviation 8010
|
15600 ng*h/mL
Standard Deviation 9130
|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (AUC[0-8h]) Following Oral Doses of Pexidartinib
Day 15
|
91600 ng*h/mL
Standard Deviation 37500
|
120000 ng*h/mL
Standard Deviation 61100
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Maximum concentration (Cmax) of pexidartinib was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Cmax) Following Oral Doses of Pexidartinib
Day 1
|
2930 ng/mL
Standard Deviation 935
|
3200 ng/mL
Standard Deviation 1630
|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Cmax) Following Oral Doses of Pexidartinib
Day 15
|
13200 ng/mL
Standard Deviation 5120
|
17800 ng/mL
Standard Deviation 9630
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Time at maximum pexidartinib concentration (Tmax) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Tmax) Following Oral Doses of Pexidartinib
Day 1
|
6.55 hours
Standard Deviation 2.24
|
5.00 hours
Standard Deviation 2.87
|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Tmax) Following Oral Doses of Pexidartinib
Day 15
|
1.5 hours
Standard Deviation 2.18
|
1.43 hours
Standard Deviation 0.87
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Metabolite to parent drug ratio of area under the curve from 0-8 h (MR AUC\[0-8h\]) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR AUC[0-8h]) Following Oral Doses of Pexidartinib
Day 1
|
0.801 metabolite to parent drug ratio
Standard Deviation 0.454
|
0.658 metabolite to parent drug ratio
Standard Deviation 0.298
|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR AUC[0-8h]) Following Oral Doses of Pexidartinib
Day 15
|
1.27 metabolite to parent drug ratio
Standard Deviation 0.416
|
1.33 metabolite to parent drug ratio
Standard Deviation 0.711
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)Population: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Metabolite to parent drug ratio of maximum pexidartinib concentration (MR Cmax) was assessed using standard non-compartmental methods.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR Cmax) Following Oral Doses of Pexidartinib
Day 1
|
0.669 metabolite to parent drug ratio
Standard Deviation 0.189
|
0.639 metabolite to parent drug ratio
Standard Deviation 0.314
|
|
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR Cmax) Following Oral Doses of Pexidartinib
Day 15
|
1.10 metabolite to parent drug ratio
Standard Deviation 0.440
|
1.18 metabolite to parent drug ratio
Standard Deviation 0.695
|
SECONDARY outcome
Timeframe: Baseline through study completion, up to 18 monthsPopulation: Adverse events were assessed in the Safety Analysis Set.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment. All TEAEs were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (NCI-CTCAE) Version 4.0.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Fatigue
|
1 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Any TEAE
|
3 Participants
|
8 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Chronic gastritis
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Constipation
|
0 Participants
|
2 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Diarrhoea
|
0 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Nausea
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Stomatitis
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Face oedema
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Malaise
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Alanine aminotransferase increased
|
1 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Aspartate aminotransferase increased
|
2 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Blood alkaline phosphatase increased
|
1 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Blood bilirubin increased
|
1 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Blood cholesterol increased
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Gamma-glutamyltransferase increased
|
0 Participants
|
2 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Weight increased
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Dysuria
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Proteinuria
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Urinary retention
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Cough
|
1 Participants
|
2 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Haemoptysis
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Hair colour changes
|
2 Participants
|
2 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Pruritus generalised
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Rash generalised
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
Skin hypopigmentation
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline through study completion, up to 18 monthsPopulation: Adverse events were assessed in the Safety Analysis Set.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment. All TEAEs were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (NCI-CTCAE) Version 4.0.
Outcome measures
| Measure |
Cohort 1 - 600 mg/Day
n=3 Participants
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 Participants
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).
|
|---|---|---|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Any treatment-related TEAE
|
3 Participants
|
6 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Diarrhoea
|
0 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Face oedema
|
0 Participants
|
1 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Fatigue
|
1 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Alanine aminotransferase increased
|
1 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Aspartate aminotransferase increased
|
2 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Blood alkaline phosphatase increased
|
1 Participants
|
3 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Blood bilirubin increased
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Blood cholesterol increased
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Gamma-glutamyltransferase increased
|
0 Participants
|
2 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Proteinuria
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Hair colour changes
|
2 Participants
|
2 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Pruritus generalised
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Rash generalised
|
1 Participants
|
0 Participants
|
|
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
Skin hypopigmentation
|
0 Participants
|
1 Participants
|
Adverse Events
Cohort 1 - 600 mg/Day
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2 - 1000 mg/Day
Serious events: 1 serious events
Other events: 8 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Cohort 1 - 600 mg/Day
n=3 participants at risk
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 participants at risk
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).
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Alanine aminotransferase increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Aspartate aminotransferase increased
|
66.7%
2/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Blood alkaline phosphatase increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Blood bilirubin increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant neoplasm progression
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
Other adverse events
| Measure |
Cohort 1 - 600 mg/Day
n=3 participants at risk
Participants who received pexidartinib 600 mg/day (200 mg in the morning and 400 mg in the evening).
|
Cohort 2 - 1000 mg/Day
n=8 participants at risk
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).
|
|---|---|---|
|
Investigations
Blood cholesterol increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Weight increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
25.0%
2/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
25.0%
2/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Eye disorders
Dacryostenosis acquired
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
37.5%
3/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
25.0%
2/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Gastrointestinal disorders
Chronic gastritis
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
General disorders
Fatigue
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
37.5%
3/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
General disorders
Face oedema
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
General disorders
Malaise
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Aspartate aminotransferase increased
|
66.7%
2/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
37.5%
3/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Alanine aminotransferase increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
37.5%
3/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Blood alkaline phosphatase increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
37.5%
3/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Blood bilirubin increased
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
25.0%
2/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant neoplasm progression
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Renal and urinary disorders
Proteinuria
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
25.0%
2/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Skin and subcutaneous tissue disorders
Hair colour changes
|
66.7%
2/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
25.0%
2/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Skin and subcutaneous tissue disorders
Pruritus generalised
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Skin and subcutaneous tissue disorders
Rash generalised
|
33.3%
1/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
0.00%
0/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
0.00%
0/3 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
12.5%
1/8 • Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.
An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place