Trial Outcomes & Findings for A Study of PLX3397 in Patients With Unresectable or Metastatic KIT-mutated Melanoma (NCT NCT02975700)
NCT ID: NCT02975700
Last Updated: 2026-01-13
Results Overview
For the assessment of best overall response, participants were classified 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.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
6 participants
Primary outcome timeframe
within 18 months postdose
Results posted on
2026-01-13
Participant Flow
A total of 8 participants entered the pilot portion of the study; no participants entered the phase 2 portion. Of the 8 participants, 2 failed screening criteria and did not receive treatment.
Participant milestones
| Measure |
PLX3397
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
PLX3397
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Progressive disease
|
3
|
|
Overall Study
Failed screening criteria
|
2
|
Baseline Characteristics
A Study of PLX3397 in Patients With Unresectable or Metastatic KIT-mutated Melanoma
Baseline characteristics by cohort
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=210 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=210 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=210 Participants
|
|
Age, Continuous
|
56.5 years
STANDARD_DEVIATION 14.5 • n=210 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=210 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=210 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Asian
|
6 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=210 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=210 Participants
|
|
Region of Enrollment
China
|
6 participants
n=210 Participants
|
PRIMARY outcome
Timeframe: within 18 months postdosePopulation: Best overall response was assessed in the Modified Intent-to-Treat Analysis Set.
For the assessment of best overall response, participants were classified 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.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Best Overall Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Complete response (CR)
|
0 Participants
|
|
Summary of Best Overall Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Partial response (PR)
|
1 Participants
|
|
Summary of Best Overall Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Stable disease (SD)
|
2 Participants
|
|
Summary of Best Overall Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Progressive disease (PD)
|
1 Participants
|
|
Summary of Best Overall Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Not evaluable (NE)
|
2 Participants
|
PRIMARY outcome
Timeframe: within 18 months postdosePopulation: Objective response rate among participants who achieved complete response or partial response was assessed in the Modified Intent-to-Treat Analysis Set.
Objective response rate was defined as complete response (CR) or partial response (PR).
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Objective Response Rate (ORR) Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
|
1 Participants
|
SECONDARY outcome
Timeframe: within 18 months postdosePopulation: Duration of response was assessed in the Modified Intent-to-Treat Analysis Set.
Duration of Response (DoR) was defined as the time from the first documentation of objective tumor response (complete response \[CR\] or partial response \[PR\]) to the first documentation of disease progression or to death due to any cause, whichever occurred first. DoR was to only be calculated for the subgroup of participants with an objective tumor response.
Outcome measures
| Measure |
PLX3397
n=1 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Duration of Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
|
NA months
Interval 8.15 to 8.15
Lack of a median value is due to fewer than 50% of the participants experiencing the event.
|
SECONDARY outcome
Timeframe: within 18 months postdosePopulation: Progression-free survival was assessed in the Modified Intent-to-Treat Analysis Set.
Progression-free survival (PFS) was to be calculated for each participant as the number of days from study enrollment to the date of the first documented disease progression or date of death from any cause, whichever occurred first.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Progression-free Survival Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
|
10.32 months
Interval 1.58 to
Missing upper bound is related to right-censored data. Last time point censored and estimate is essentially infinity, therefore it is NA.
|
SECONDARY outcome
Timeframe: within 18 months postdosePopulation: Overall survival was assessed in the Modified Intent-to-Treat Analysis Set.
Overall survival (OS) was defined as the time from study enrollment to death from any cause, censoring at the date of last contact or the end of the study.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Overall Survival Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
|
NA months
Interval 4.11 to
There were not enough events to estimate the median survival time.
|
SECONDARY outcome
Timeframe: within 18 months postdosePopulation: Disease control rate among participants who achieved CR, PR, or SD was assessed in the Modified Intent-to-Treat Analysis Set.
Disease control rate (DCR) was defined as the percentage of participants who had achieved complete response (CR), partial response (PR), or stable disease (SD).
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Disease Control Rate Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
|
3 Participants
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5 h, 1h, 2h, 4h, and 6h postdosePopulation: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Pharmacokinetic Parameter of Maximum Concentration (Cmax) of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Day 1
|
4660 ng/mL
Standard Deviation 1830
|
|
Summary of Pharmacokinetic Parameter of Maximum Concentration (Cmax) of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Day 15
|
9570 ng/mL
Standard Deviation 2940
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5 h, 1h, 2h, 4h, and 6h postdosePopulation: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Pharmacokinetic Parameter of Area Under the Curve From Zero to 6 Hours (AUC0-6) of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Day 1
|
14500 h*ng/mL
Standard Deviation 4660
|
|
Summary of Pharmacokinetic Parameter of Area Under the Curve From Zero to 6 Hours (AUC0-6) of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Day 15
|
43800 h*ng/mL
Standard Deviation 7010
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5 h, 1h, 2h, 4h, and 6h postdosePopulation: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Pharmacokinetic Parameter of Time to Maximum Concentration and Last Measurable Time of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Time to maximum concentration; Day 1
|
2.72 hours
Standard Deviation 1.62
|
|
Summary of Pharmacokinetic Parameter of Time to Maximum Concentration and Last Measurable Time of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Time to maximum concentration; Day 15
|
2.77 hours
Standard Deviation 2.31
|
|
Summary of Pharmacokinetic Parameter of Time to Maximum Concentration and Last Measurable Time of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Last measurable time; Day 1
|
5.99 hours
Standard Deviation 0.05
|
|
Summary of Pharmacokinetic Parameter of Time to Maximum Concentration and Last Measurable Time of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
Last measurable time; Day 15
|
6.03 hours
Standard Deviation 0.06
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5 h, 1h, 2h, 4h, and 6h postdosePopulation: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Outcome measures
| Measure |
PLX3397
n=5 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Pharmacokinetic Parameter of Accumulation Ratio of Day 15 to Day 1 for AUC0-6h (Robs) of PL3397 at Day 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
|
3.39 Ratio
Standard Deviation 0.85
|
SECONDARY outcome
Timeframe: Cycle 1, Day 1 and Day 15 at predose, 0.5 h, 1h, 2h, 4h, and 6h postdosePopulation: Pharmacokinetic parameters were assessed in the Pharmacokinetic Analysis Set.
Outcome measures
| Measure |
PLX3397
n=5 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Pharmacokinetic Parameter of Accumulation Ratio of Day 15 to Day 1 for Cmax (Rcmax) of PL3397 at Day 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma
|
2.34 Ratio
Standard Deviation 0.69
|
SECONDARY outcome
Timeframe: within 28 days after administration of the last dose of study drug, up to 18 months postdosePopulation: Safety events were assessed in the Safety Analysis Set.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Anaemia
|
5 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Gamma-glutamyltransferase increased
|
4 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Hair colour changes
|
4 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Neutrophil count decreased
|
4 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Bilirubin conjugated increased
|
3 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood alkaline phosphatase increased
|
3 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood lactate dehydrogenase increased
|
3 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Interstitial lung disease
|
3 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Pyrexia
|
3 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Total bile acids increased
|
3 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Face oedema
|
5 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Alanine aminotransferase increased
|
4 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood bilirubin increased
|
4 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Aspartate aminotransferase increased
|
6 Participants
|
|
Summary of Most Common (≥50% of Participants) Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
White blood cell count decreased
|
6 Participants
|
SECONDARY outcome
Timeframe: within 28 days after administration of the last dose of study drug, up to 18 months postdosePopulation: Safety events were assessed in the Safety Analysis Set.
Grade ≥ 3 was used to indicate moderate-to-severe treatment-emergent adverse events in which moderate was defined as discomfort enough to cause interference with normal daily activities and severe defined as the inability to perform normal daily activities.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Any TEAEs CTCAE Grade ≥ 3
|
4 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Gamma-glutamyltransferase increased
|
4 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Alanine aminotransferase increased
|
2 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Total bile acids increased
|
2 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Anaemia
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Aspartate aminotransferase increased
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Bilirubin conjugated increased
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood alkaline phosphatase increased
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Neutrophil count decreased
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
White blood cell count decreased
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood bilirubin increased
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Drug-induced liver injury
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Herpes zoster
|
1 Participants
|
|
Summary of Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Hypertension
|
1 Participants
|
SECONDARY outcome
Timeframe: within 28 days after administration of the last dose of study drug, up to 18 months postdosePopulation: Safety events were assessed in the Safety Analysis Set.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Aspartate aminotransferase increased
|
6 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
White blood cell count decreased
|
6 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Anaemia
|
5 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Face oedema
|
5 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood lactate dehydrogenase increased
|
3 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Interstitial lung disease
|
3 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Total bile acids increased
|
3 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Alanine aminotransferase increased
|
4 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood bilirubin increased
|
4 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Gamma-glutamyltransferase increased
|
4 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Hair colour changes
|
4 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Neutrophil count decreased
|
4 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Bilirubin conjugated increased
|
3 Participants
|
|
Summary of Most Common (≥50%) Drug-related Treatment-emergent Adverse Events Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood alkaline phosphatase increased
|
3 Participants
|
SECONDARY outcome
Timeframe: within 28 days after administration of the last dose of study drug, up to 18 months postdosePopulation: Safety events were assessed in the Safety Analysis Set.
Grade ≥ 3 was used to indicate moderate-to-severe treatment-emergent adverse events in which moderate was defined as discomfort enough to cause interference with normal daily activities and severe defined as the inability to perform normal daily activities.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Any Drug-related TEAEs CTCAE Grade ≥ 3
|
4 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Gamma-glutamyltransferase increased
|
4 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Alanine aminotransferase increased
|
2 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Total bile acids increased
|
2 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Aspartate aminotransferase increased
|
1 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Bilirubin conjugated increased
|
1 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood alkaline phosphatase increased
|
1 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Blood bilirubin increased
|
1 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Drug-induced liver injury
|
1 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
Neutrophil count decreased
|
1 Participants
|
|
Summary of Drug-related Treatment-emergent Adverse Events Grade ≥ 3 Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma
White blood cell count decreased
|
1 Participants
|
Adverse Events
PLX3397
Serious events: 2 serious events
Other events: 6 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
PLX3397
n=6 participants at risk
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Hepatobiliary disorders
Drug-induced liver injury
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
Other adverse events
| Measure |
PLX3397
n=6 participants at risk
Participants who received PLX3397 1000 mg/day (400 mg in the morning and 600 mg in the evening).
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
100.0%
6/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Skin and subcutaneous tissue disorders
Hair colour changes
|
66.7%
4/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Gastrointestinal disorders
Constipation
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Gastrointestinal disorders
Mouth ulceration
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Protein urine present
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Glucose urine present
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Urine ketone body present
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
General disorders
Face oedema
|
83.3%
5/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
General disorders
Pyrexia
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
General disorders
Fatigue
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Aspartate aminotransferase increased
|
100.0%
6/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
White blood cell count decreased
|
100.0%
6/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Alanine aminotransferase increased
|
66.7%
4/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood bilirubin increased
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Gamma-glutamyltransferase increased
|
66.7%
4/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Neutrophil count decreased
|
66.7%
4/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Bilirubin conjugated increased
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood alkaline phosphatase increased
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood lactate dehydrogenase increased
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Total bile acids increased
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Red blood cell count decreased
|
66.7%
4/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood creatinine phosphokinase increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood potassium decreased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood triglycerides increased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood urea increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood uric acid increased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Creatinine renal clearance decreased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Electrocardiogram QT prolonged
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
50.0%
3/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Eye disorders
Eyelid oedema
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Hepatobiliary disorders
Drug-induced liver injury
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Infections and infestations
Conjunctivitis
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Infections and infestations
Herpes zoster
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Infections and infestations
Upper respiratory tract infection
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Nervous system disorders
Headache
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Renal and urinary disorders
Proteinuria
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Vascular disorders
Hypertension
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Alpha hydroxybutyrate dehydrogenase increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood chloride decreased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood cholesterol increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood creatine phosphokinase MB increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood glucose increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
High density lipoprotein increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Low density lipoprotein increased
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Albumin globulin ratio increased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Blood magnesium increased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
C-reactive protein increased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Electrocardiogram high voltage
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Electrocardiogram ST-T change
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Electrocardiogram T wave abnormal
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Haemoglobin decreased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Mean cell volume abnormal
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
Urobilinogen urine increased
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Investigations
White blood cells urine positive
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Infections and infestations
Corona virus infection
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Infections and infestations
Urinary tract infection
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
2/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Nervous system disorders
Amnesia
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Renal and urinary disorders
Haematuria
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
|
Reproductive system and breast disorders
Cervical polyp
|
16.7%
1/6 • Adverse events were collected from after initiation of study drug up to 28 days after administration of last dose of study drug, up to 18 months postdose.
|
Additional Information
Contact for Clinical Trial Information
Daiichi Sankyo, Inc.
Phone: 908-992-6400
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place