Trial Outcomes & Findings for PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors (NCT NCT02584647)
NCT ID: NCT02584647
Last Updated: 2025-03-21
Results Overview
The highest dose of a drug or treatment that does not cause unacceptable side effects, which will be used in Phase 2.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
39 participants
Primary outcome timeframe
Up to 3 years
Results posted on
2025-03-21
Participant Flow
Participant milestones
| Measure |
Cohort 1, Phase 1
Subjects with unresectable or metastatic sarcoma will take orally 800 mg pexidartinib in combination with 2 mg sirolimus daily (Dose Level 2).
|
Cohort 2, Phase 1
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 2 mg sirolimus daily (Dose Level 3).
|
Cohort 3, Phase 1
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 4 mg sirolimus daily (Dose Level 4).
|
Cohort 4, Phase 1
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 6 mg sirolimus daily (Dose Level 5).
|
Phase 2 Cohort
Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take the recommended Phase 2 dose (RP2D): 1000 mg pexidartinib and 2 mg sirolimus daily.
|
|---|---|---|---|---|---|
|
Dose Level 1 (S: 2mg, P: 600mg)
STARTED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 1 (S: 2mg, P: 600mg)
COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 1 (S: 2mg, P: 600mg)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 2 (S: 2mg, P:800mg)
STARTED
|
2
|
0
|
0
|
0
|
0
|
|
Dose Level 2 (S: 2mg, P:800mg)
COMPLETED
|
2
|
0
|
0
|
0
|
0
|
|
Dose Level 2 (S: 2mg, P:800mg)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
Dose Level 3 (S: 2mg, P: 1000mg)
STARTED
|
0
|
12
|
0
|
0
|
0
|
|
Dose Level 3 (S: 2mg, P: 1000mg)
COMPLETED
|
0
|
9
|
0
|
0
|
0
|
|
Dose Level 3 (S: 2mg, P: 1000mg)
NOT COMPLETED
|
0
|
3
|
0
|
0
|
0
|
|
Dose Level 4 (S: 4mg, P: 1000mg)
STARTED
|
0
|
0
|
9
|
0
|
0
|
|
Dose Level 4 (S: 4mg, P: 1000mg)
COMPLETED
|
0
|
0
|
6
|
0
|
0
|
|
Dose Level 4 (S: 4mg, P: 1000mg)
NOT COMPLETED
|
0
|
0
|
3
|
0
|
0
|
|
Dose Level 5 (S: 6mg, P: 1000mg)
STARTED
|
0
|
0
|
0
|
1
|
0
|
|
Dose Level 5 (S: 6mg, P: 1000mg)
COMPLETED
|
0
|
0
|
0
|
1
|
0
|
|
Dose Level 5 (S: 6mg, P: 1000mg)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
|
RP2D (S: 2mg, P: 1000mg)
STARTED
|
0
|
0
|
0
|
0
|
14
|
|
RP2D (S: 2mg, P: 1000mg)
COMPLETED
|
0
|
0
|
0
|
0
|
14
|
|
RP2D (S: 2mg, P: 1000mg)
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors
Baseline characteristics by cohort
| Measure |
Cohort 1, Phase 1
n=2 Participants
Subjects with unresectable or metastatic sarcoma will take orally 800 mg pexidartinib in combination with 2 mg sirolimus daily (Dose Level 2).
|
Cohort 2, Phase 1
n=12 Participants
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 2 mg sirolimus daily (Dose Level 3).
|
Cohort 3, Phase 1
n=9 Participants
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 4 mg sirolimus daily (Dose Level 4).
|
Cohort 4, Phase 1
n=1 Participants
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 6 mg sirolimus daily (Dose Level 5).
|
Phase 2 Cohort
n=14 Participants
Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take the recommended Phase 2 dose (RP2D): 1000 mg pexidartinib and 2 mg sirolimus daily.
|
Total
n=38 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Continuous
|
52.5 years
n=5 Participants
|
47.0 years
n=7 Participants
|
60 years
n=5 Participants
|
44 years
n=4 Participants
|
36.5 years
n=21 Participants
|
49.4 years
n=8 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
16 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
22 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
6 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
32 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
2 Participants
n=8 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
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
5 Participants
n=8 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
26 Participants
n=8 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=8 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
5 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
2 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
38 Participants
n=8 Participants
|
PRIMARY outcome
Timeframe: Up to 3 yearsThe highest dose of a drug or treatment that does not cause unacceptable side effects, which will be used in Phase 2.
Outcome measures
| Measure |
Phase 1
n=24 Participants
Subjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily during Phase 1 of the study.
|
|---|---|
|
Maximum Tolerated Dose (MTD) - Phase 1
Pexidartinib
|
1000 mg
|
|
Maximum Tolerated Dose (MTD) - Phase 1
Sirolimus
|
2 mg
|
PRIMARY outcome
Timeframe: Up to 3 yearsThe time from the start of treatment until disease progression or death from any cause, calculated in weeks.
Outcome measures
| Measure |
Phase 1
n=14 Participants
Subjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily during Phase 1 of the study.
|
|---|---|
|
Progression Free Survival (PFS) Rate - Phase 2
|
6 weeks
Interval 6.0 to 19.1
|
SECONDARY outcome
Timeframe: Up to 3 yearsPopulation: Only assessed for Phase 2
The time from the start of treatment until death, estimated using the Kaplan Meier method.
Outcome measures
| Measure |
Phase 1
n=14 Participants
Subjects with unresectable or metastatic sarcoma will take orally PLX3397 (600 - 1000mg) in combination with Sirolimus (2-6mg) daily during Phase 1 of the study.
|
|---|---|
|
Overall Survival Rate
|
17.9 weeks
Interval 13.7 to
insufficient number of participants with events
|
Adverse Events
Cohort 1, Phase 1
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2, Phase 1
Serious events: 6 serious events
Other events: 12 other events
Deaths: 0 deaths
Cohort 3, Phase 1
Serious events: 7 serious events
Other events: 9 other events
Deaths: 1 deaths
Cohort 4, Phase 1
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Phase 2 Cohort
Serious events: 5 serious events
Other events: 14 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Cohort 1, Phase 1
n=2 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 800 mg pexidartinib in combination with 2 mg sirolimus daily.
|
Cohort 2, Phase 1
n=12 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 2 mg sirolimus daily.
|
Cohort 3, Phase 1
n=9 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 4 mg sirolimus daily.
|
Cohort 4, Phase 1
n=1 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 6 mg sirolimus daily.
|
Phase 2 Cohort
n=14 participants at risk
Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take pexidartinib and sirolimus at the recommended Phase 2 dose (RP2D): 1000 mg pexidartinib in combination with 2 mg sirolimus daily.
|
|---|---|---|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms
|
50.0%
1/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Social circumstances
Social circumstances
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Immune system disorders
Sepsis
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Pain
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Hepatobiliary disorders
Hepatic failure
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Dehydration
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Vomiting
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Nervous system disorders
Nervous system disorders
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hyperkalemia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Administration site condition
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Nausea
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Gastrointestinal disorders
Obstruction gastric
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Infections and infestations
Wound infection
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Gastrointestinal disorders
Colonic perforation
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Infections and infestations
Lung infection
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness upper limb
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
General disorders
Syncope
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
Other adverse events
| Measure |
Cohort 1, Phase 1
n=2 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 800 mg pexidartinib in combination with 2 mg sirolimus daily.
|
Cohort 2, Phase 1
n=12 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 2 mg sirolimus daily.
|
Cohort 3, Phase 1
n=9 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 4 mg sirolimus daily.
|
Cohort 4, Phase 1
n=1 participants at risk
Subjects with unresectable or metastatic sarcoma will take orally 1000 mg pexidartinib in combination with 6 mg sirolimus daily.
|
Phase 2 Cohort
n=14 participants at risk
Subjects with unresectable or metastatic Malignant Peripheral Nerve Sheath Tumors (MPNSTs) will take pexidartinib and sirolimus at the recommended Phase 2 dose (RP2D): 1000 mg pexidartinib in combination with 2 mg sirolimus daily.
|
|---|---|---|---|---|---|
|
Hepatobiliary disorders
Alanine aminotransferase increased
|
100.0%
2/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
Hepatobiliary disorders
Alkaline phosphatase increased
|
100.0%
2/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
General disorders
Allergic rhinitis
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/2 • Up to 4 years
|
83.3%
10/12 • Up to 4 years
|
55.6%
5/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
General disorders
Anorexia
|
0.00%
0/2 • Up to 4 years
|
41.7%
5/12 • Up to 4 years
|
66.7%
6/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
42.9%
6/14 • Up to 4 years
|
|
General disorders
Anxiety
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Aspartate aminotransferase increased
|
100.0%
2/2 • Up to 4 years
|
75.0%
9/12 • Up to 4 years
|
55.6%
5/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
General disorders
Bloating
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Blood bilirubin increased
|
0.00%
0/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Blurred vision
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Bruising
|
50.0%
1/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Cardiac disorders
Cardiac arrest
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Chills
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Confusion
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
50.0%
7/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
100.0%
2/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
General disorders
Creatinine increased
|
100.0%
2/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Dehydration
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Depression
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
1/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
42.9%
6/14 • Up to 4 years
|
|
General disorders
Dry mouth
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Dysgeusia
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
General disorders
Edema
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
General disorders
Epistaxis
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Fatigue
|
0.00%
0/2 • Up to 4 years
|
58.3%
7/12 • Up to 4 years
|
77.8%
7/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
42.9%
6/14 • Up to 4 years
|
|
General disorders
Fever
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
General disorders
Hallucinations
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Headache
|
50.0%
1/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hematoma
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Renal and urinary disorders
Hematuria
|
50.0%
1/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypercalcemia
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hyperglycemia
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hyperkalemia
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypernatremia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Hypertension
|
50.0%
1/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hyperuricemia
|
50.0%
1/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypoalbuminemia
|
0.00%
0/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypocalcemia
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
55.6%
5/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypoglycemia
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypokalemia
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hyponatremia
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Hypophosphatemia
|
0.00%
0/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Hypoxia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
INR increased
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Insomnia
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
Vascular disorders
Laryngeal hemorrhage
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Infections and infestations
Lip infection
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Lymphocyte count decreased
|
0.00%
0/2 • Up to 4 years
|
41.7%
5/12 • Up to 4 years
|
55.6%
5/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
General disorders
Mucositis oral
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Nasal congestion
|
50.0%
1/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Nausea
|
0.00%
0/2 • Up to 4 years
|
50.0%
6/12 • Up to 4 years
|
66.7%
6/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
28.6%
4/14 • Up to 4 years
|
|
Nervous system disorders
Nervous system disorders
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Immune system disorders
Neutrophil count decreased
|
0.00%
0/2 • Up to 4 years
|
33.3%
4/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
42.9%
6/14 • Up to 4 years
|
|
General disorders
Oral dysesthesia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Pain
|
100.0%
2/2 • Up to 4 years
|
58.3%
7/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
85.7%
12/14 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Paresthesia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Periorbital edema
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
0.00%
0/2 • Up to 4 years
|
16.7%
2/12 • Up to 4 years
|
44.4%
4/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
Presyncope
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
21.4%
3/14 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
55.6%
5/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
14.3%
2/14 • Up to 4 years
|
|
Cardiac disorders
Sinus tachycardia
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Renal and urinary disorders
Urinary frequency
|
50.0%
1/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
7.1%
1/14 • Up to 4 years
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/2 • Up to 4 years
|
25.0%
3/12 • Up to 4 years
|
0.00%
0/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Cardiac disorders
Ventricular tachycardia
|
0.00%
0/2 • Up to 4 years
|
0.00%
0/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
General disorders
Vomiting
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
22.2%
2/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Metabolism and nutrition disorders
Weight loss
|
0.00%
0/2 • Up to 4 years
|
8.3%
1/12 • Up to 4 years
|
11.1%
1/9 • Up to 4 years
|
100.0%
1/1 • Up to 4 years
|
0.00%
0/14 • Up to 4 years
|
|
Blood and lymphatic system disorders
White blood cell decreased
|
0.00%
0/2 • Up to 4 years
|
41.7%
5/12 • Up to 4 years
|
33.3%
3/9 • Up to 4 years
|
0.00%
0/1 • Up to 4 years
|
28.6%
4/14 • Up to 4 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place