Trial Outcomes & Findings for Pilot Study of PLX3397 in Patients With Advanced Castration-Resistant Prostate Cancer (CRPC) (NCT NCT01499043)
NCT ID: NCT01499043
Last Updated: 2020-03-04
Results Overview
Effects of PLX3397 on CTC number in men with radiographically progressive CRPC and high CTC counts (≥10 CTCs/7.5 mL of blood using CellSearch Assay) CTC counts were to be evaluated at the following time points: Screening, Baseline, every 4 weeks after treatment initiation, and at Safety Follow-up. Radiographic tumor evaluation were to be performed every 8 weeks. Progression at the first reassessment required a confirmatory scan at a minimum of 6 weeks later, and treatment with study medication was to continue until the progression had been confirmed.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
See measure description for time frame.
Results posted on
2020-03-04
Participant Flow
A total of 6 participants received study drug at 2 clinic sites.
A total of 20 participants were planned to be enrolled at approximately 2 to 4 clinic sites.
Participant milestones
| Measure |
PLX3397
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
PLX3397
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
|---|---|
|
Overall Study
Progressive disease (radiographic)
|
2
|
|
Overall Study
Progressive disease (clinical)
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
Pilot Study of PLX3397 in Patients With Advanced Castration-Resistant Prostate Cancer (CRPC)
Baseline characteristics by cohort
| Measure |
PLX3397
n=6 Participants
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
|---|---|
|
Age, Continuous
|
71.0 years
STANDARD_DEVIATION 7.24 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: See measure description for time frame.Population: The study was discontinued and the primary outcome measure was not evaluated. Consequently, no efficacy was evaluated and only safety results are reported. Raw data collected but no efficacy analysis was performed/reported.
Effects of PLX3397 on CTC number in men with radiographically progressive CRPC and high CTC counts (≥10 CTCs/7.5 mL of blood using CellSearch Assay) CTC counts were to be evaluated at the following time points: Screening, Baseline, every 4 weeks after treatment initiation, and at Safety Follow-up. Radiographic tumor evaluation were to be performed every 8 weeks. Progression at the first reassessment required a confirmatory scan at a minimum of 6 weeks later, and treatment with study medication was to continue until the progression had been confirmed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Assessed from first dose through at least 4 weeks after the last dose.Population: Adverse events were assessed in the Safety Population.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
NCI CTCAE Grade 2
Participants who received PLX3397 and experienced a Grade 2 treatment-emergent adverse event.
|
NCI CTCAE Grade 3
Participants who received PLX3397 and experienced a Grade 3 treatment-emergent adverse event.
|
NCI CTCAE Grade 4
Participants who received PLX3397 and experienced a Grade 4 treatment-emergent adverse event.
|
NCI CTCAE Grade 5
Participants who received PLX3397 and experienced a Grade 5 treatment-emergent adverse event.
|
|---|---|---|---|---|---|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Pain in extremity
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Dyspnoea
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Anaemia
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Thrombocytopenia
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Constipation
|
2 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Diarrhoea
|
2 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Nausea
|
4 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Fatigue
|
3 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Mucosal inflammation
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Alanine aminotransferase increased
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Aspartate aminotransferase increased
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Blood creatinine phosphokinase increased
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Blood glucose increased
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Blood sodium increased
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Lipase increased
|
1 Participants
|
—
|
—
|
—
|
—
|
|
Number of Participants Reporting Treatment-Related Adverse Events by System Organ Class and Preferred Term
Decreased appetite
|
3 Participants
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Assessed from first dose through at least 4 weeks after the last dose.Population: Adverse events were assessed in the Safety Population.
Adverse events (AEs) were assessed using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0, where 1=mild, 2=moderate, 3=severe, 4=life-threatening, and 5=death related to AE.
Outcome measures
| Measure |
PLX3397
n=6 Participants
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
NCI CTCAE Grade 2
n=6 Participants
Participants who received PLX3397 and experienced a Grade 2 treatment-emergent adverse event.
|
NCI CTCAE Grade 3
n=6 Participants
Participants who received PLX3397 and experienced a Grade 3 treatment-emergent adverse event.
|
NCI CTCAE Grade 4
n=6 Participants
Participants who received PLX3397 and experienced a Grade 4 treatment-emergent adverse event.
|
NCI CTCAE Grade 5
n=6 Participants
Participants who received PLX3397 and experienced a Grade 5 treatment-emergent adverse event.
|
|---|---|---|---|---|---|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Anaemia
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Oedema peripheral
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Thrombocytopenia
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Constipation
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Diarrhoea
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Nausea
|
4 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Fatigue
|
1 Participants
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Mucosal inflammation
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Pyrexia
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Oedema
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Pain
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Influenza
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Pneumonia
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Fall
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
ALT increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
AST increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Blood CPK increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Lipase increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Blood glucose increased
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Blood sodium increased
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Decreased appetite
|
3 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Musculoskeletal pain
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Pain in extremity
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Peripheral sensory neuropathy
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Spinal cord disorder
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Nocturia
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Urinary retention
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Pollakiuria
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Reporting Treatment-Emergent Adverse Events by Worst Severity Grade
Dyspnoea
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
PLX3397
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
PLX3397
n=6 participants at risk
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
|---|---|
|
Infections and infestations
Influenza
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Infections and infestations
Pneumonia
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Injury, poisoning and procedural complications
Fall
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Renal and urinary disorders
Urinary retention
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
Other adverse events
| Measure |
PLX3397
n=6 participants at risk
Participants who received a daily oral dose of PLX3397 for 28 day cycles and continued on therapy until disease progression or toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Gastrointestinal disorders
Constipation
|
33.3%
2/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
2/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Gastrointestinal disorders
Nausea
|
66.7%
4/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
General disorders
Fatigue
|
50.0%
3/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
General disorders
Mucosal inflammation
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
General disorders
Oedema
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
General disorders
Oedema peripheral
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
General disorders
Pain
|
33.3%
2/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
General disorders
Pyrexia
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Infections and infestations
Influenza
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Infections and infestations
Pneumonia
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Injury, poisoning and procedural complications
Fall
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Investigations
Alanine aminotransferase increased
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Investigations
Aspartate aminotransferase increased
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Investigations
Blood creatinine phosphokinase increased
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Investigations
Blood glucose increased
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Investigations
Blood sodium increased
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Investigations
Lipase increased
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
50.0%
3/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Nervous system disorders
Spinal cord disorder
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Renal and urinary disorders
Nocturia
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Renal and urinary disorders
Pollakiuria
|
33.3%
2/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Renal and urinary disorders
Urinary retention
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
16.7%
1/6 • Adverse events were collected from after first dose to 28 days after last dose.
Adverse events that emerge (or worsen) from after the first dose of study drug and within 28 days after last dose.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place