Trial Outcomes & Findings for Ruxolitinib in Estrogen Receptor Positive Breast Cancer (NCT NCT01594216)
NCT ID: NCT01594216
Last Updated: 2024-06-12
Results Overview
Safety will be assessed by evaluation of the number and severity adverse events as defined by the NCI CTCAE Version 4.0
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
3 years
Results posted on
2024-06-12
Participant Flow
Participant milestones
| Measure |
First Stage
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
10
|
|
Overall Study
COMPLETED
|
15
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Ruxolitinib in Estrogen Receptor Positive Breast Cancer
Baseline characteristics by cohort
| Measure |
First Stage
n=15 Participants
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
n=10 Participants
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Total
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
59 years
n=5 Participants
|
62 years
n=7 Participants
|
60 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 yearsPopulation: 25 patients analyzed
Safety will be assessed by evaluation of the number and severity adverse events as defined by the NCI CTCAE Version 4.0
Outcome measures
| Measure |
First Stage
n=15 Participants
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
n=10 Participants
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
|---|---|---|
|
Number of Participants With Adverse Events
|
5 participants
|
1 participants
|
PRIMARY outcome
Timeframe: 3 yearsPer Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by CT or MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.
Outcome measures
| Measure |
First Stage
n=15 Participants
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
n=10 Participants
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
|---|---|---|
|
Time to Progression
|
2.8 months
Interval 2.6 to 3.9
|
2.8 months
Interval 2.6 to 3.9
|
SECONDARY outcome
Timeframe: 3 yearsOutcome measures
| Measure |
First Stage
n=10 Participants
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
n=7 Participants
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
|---|---|---|
|
Pharmacodynamic Assessment of Phospho-STAT3 Inhibition
|
24.5 % inhibition of phosphorylated STAT3
Standard Deviation 12.7
|
29 % inhibition of phosphorylated STAT3
Standard Deviation 26.4
|
SECONDARY outcome
Timeframe: 3 yearsOutcome measures
Outcome data not reported
Adverse Events
First Stage
Serious events: 1 serious events
Other events: 15 other events
Deaths: 0 deaths
Second Stage
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
First Stage
n=15 participants at risk
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
n=10 participants at risk
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
|---|---|---|
|
Blood and lymphatic system disorders
neutropenic fever
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Investigations
elevated creatinine
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Hepatobiliary disorders
hepatic failure
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
Other adverse events
| Measure |
First Stage
n=15 participants at risk
Ruxolitinib at 25 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
Second Stage
n=10 participants at risk
Ruxolitinib at 15 mg orally, twice daily and Exemestane, 25 mg orally once daily
Ruxolitinib
Exemestane
|
|---|---|---|
|
Blood and lymphatic system disorders
anemia (grade 2)
|
26.7%
4/15 • Number of events 4
|
10.0%
1/10 • Number of events 1
|
|
Blood and lymphatic system disorders
neutropenia (grade 2)
|
6.7%
1/15 • Number of events 1
|
10.0%
1/10 • Number of events 1
|
|
Infections and infestations
infection (grade 2)
|
6.7%
1/15 • Number of events 1
|
20.0%
2/10 • Number of events 2
|
|
Blood and lymphatic system disorders
thrombocytopenia (grade 2)
|
13.3%
2/15 • Number of events 2
|
0.00%
0/10
|
|
General disorders
pain (grade 2)
|
46.7%
7/15 • Number of events 7
|
80.0%
8/10 • Number of events 8
|
|
Psychiatric disorders
psychiatric (grade 2)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary (grade 2)
|
20.0%
3/15 • Number of events 3
|
20.0%
2/10 • Number of events 2
|
|
Gastrointestinal disorders
nausea/vomiting (grade 2)
|
0.00%
0/15
|
20.0%
2/10 • Number of events 2
|
|
Gastrointestinal disorders
other gastrointestinal (grade 2)
|
40.0%
6/15 • Number of events 6
|
30.0%
3/10 • Number of events 3
|
|
Investigations
elevated liver function test (grade 2)
|
6.7%
1/15 • Number of events 1
|
10.0%
1/10 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
pain (grade 1)
|
46.7%
7/15 • Number of events 7
|
80.0%
8/10 • Number of events 8
|
|
Gastrointestinal disorders
constipation (grade 1)
|
33.3%
5/15 • Number of events 5
|
20.0%
2/10 • Number of events 2
|
|
General disorders
fatigue (grade 2)
|
26.7%
4/15 • Number of events 4
|
0.00%
0/10
|
|
General disorders
fatigue (grade 1)
|
0.00%
0/15
|
40.0%
4/10 • Number of events 4
|
|
General disorders
fatigue (grade 3)
|
0.00%
0/15
|
40.0%
4/10 • Number of events 4
|
|
Cardiac disorders
hypertension (grade 2)
|
20.0%
3/15 • Number of events 3
|
10.0%
1/10 • Number of events 1
|
|
Cardiac disorders
hypertension (grade 3)
|
6.7%
1/15 • Number of events 1
|
30.0%
3/10 • Number of events 3
|
|
Gastrointestinal disorders
other gastrointestinal (grade 1)
|
20.0%
3/15 • Number of events 3
|
30.0%
3/10 • Number of events 3
|
|
Gastrointestinal disorders
diarrhea (grade 1)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Gastrointestinal disorders
diarrhea (grade 3)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Nervous system disorders
headache (grade 1)
|
40.0%
6/15 • Number of events 6
|
10.0%
1/10 • Number of events 1
|
|
Nervous system disorders
headache (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Gastrointestinal disorders
nausea/vomiting (grade 1)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Gastrointestinal disorders
nausea/vomiting (grade 3)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
General disorders
pain (grade 3)
|
13.3%
2/15 • Number of events 2
|
10.0%
1/10 • Number of events 1
|
|
Blood and lymphatic system disorders
anemia (grade 1)
|
33.3%
5/15 • Number of events 5
|
50.0%
5/10 • Number of events 5
|
|
Blood and lymphatic system disorders
anemia (grade 3)
|
20.0%
3/15 • Number of events 3
|
10.0%
1/10 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary (grade 1)
|
46.7%
7/15 • Number of events 7
|
80.0%
8/10 • Number of events 8
|
|
Renal and urinary disorders
urinary (grade 1)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary (grade 3)
|
13.3%
2/15 • Number of events 2
|
20.0%
2/10 • Number of events 2
|
|
Investigations
elevated hepatic function (grade 1)
|
53.3%
8/15 • Number of events 8
|
40.0%
4/10 • Number of events 4
|
|
Investigations
neutropenia (grade 1)
|
20.0%
3/15 • Number of events 3
|
30.0%
3/10 • Number of events 3
|
|
Infections and infestations
flu-like symptoms (grade 1)
|
33.3%
5/15 • Number of events 5
|
30.0%
3/10 • Number of events 3
|
|
Infections and infestations
infection (grade 1)
|
20.0%
3/15 • Number of events 3
|
20.0%
2/10 • Number of events 2
|
|
Psychiatric disorders
psychiatric (grade 1)
|
66.7%
10/15 • Number of events 10
|
20.0%
2/10 • Number of events 2
|
|
Nervous system disorders
neurological (grade 1)
|
26.7%
4/15 • Number of events 4
|
20.0%
2/10 • Number of events 2
|
|
Metabolism and nutrition disorders
hyponatremia (grade 1)
|
0.00%
0/15
|
20.0%
2/10 • Number of events 2
|
|
Nervous system disorders
dizziness (grade 2)
|
6.7%
1/15 • Number of events 1
|
10.0%
1/10 • Number of events 1
|
|
General disorders
edema (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
General disorders
edema (grade 2)
|
13.3%
2/15 • Number of events 2
|
0.00%
0/10
|
|
Metabolism and nutrition disorders
hyponatremia (grade 3)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Psychiatric disorders
psychiatric (grade 3)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Cardiac disorders
palpitations (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Cardiac disorders
pericarditis (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Investigations
decreased white blood cells (grade 1)
|
26.7%
4/15 • Number of events 4
|
10.0%
1/10 • Number of events 1
|
|
Investigations
decreased white blood cells (grade 2)
|
13.3%
2/15 • Number of events 2
|
20.0%
2/10 • Number of events 2
|
|
Reproductive system and breast disorders
vaginal bleeding (grade 1)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Reproductive system and breast disorders
premature menopause (grade 3)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Vascular disorders
hot flashes (grade 1)
|
20.0%
3/15 • Number of events 3
|
10.0%
1/10 • Number of events 1
|
|
Vascular disorders
hot flashes (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Renal and urinary disorders
elevated creatinine (grade 1)
|
13.3%
2/15 • Number of events 2
|
10.0%
1/10 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
arthritis (grade 1)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Metabolism and nutrition disorders
dehydration (grade 1)
|
6.7%
1/15 • Number of events 1
|
10.0%
1/10 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
pruritis (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Eye disorders
blurred vision (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Renal and urinary disorders
urinary incontinence (grade 3)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Renal and urinary disorders
urinary tract infection (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Investigations
neutropenia (grade 3)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Investigations
decreased white blood cells (grade 3)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Metabolism and nutrition disorders
hypercalcemia (grade 2)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Metabolism and nutrition disorders
hypermagnesemia (grade 1)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Endocrine disorders
type 2 diabetes (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Metabolism and nutrition disorders
hypokalemia (grade 1)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Cardiac disorders
presyncope (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Investigations
decreased chloride (grade 1)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
osteoporosis (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Musculoskeletal and connective tissue disorders
osteoporosis (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Endocrine disorders
hypothyroidism (grade 2)
|
20.0%
3/15 • Number of events 3
|
0.00%
0/10
|
|
Endocrine disorders
hypothyroidism (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Infections and infestations
flu-like symptoms (grade 2)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
fracture (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Musculoskeletal and connective tissue disorders
leg cramps (grade 1)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
General disorders
chest pressure (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Investigations
decreased AST (grade 2)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Metabolism and nutrition disorders
hypoalbuminemia (grade 2)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
|
Injury, poisoning and procedural complications
fall (grade 2)
|
0.00%
0/15
|
10.0%
1/10 • Number of events 1
|
|
Investigations
elevated BNP (grade 1)
|
6.7%
1/15 • Number of events 1
|
0.00%
0/10
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place