Trial Outcomes & Findings for Open Label Ruxolitinib (INCB018424) in Patients With Myelofibrosis and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis (NCT NCT00509899)
NCT ID: NCT00509899
Last Updated: 2018-03-12
Results Overview
Treatment-Emergent AEs are events occurring after first drug administration or worsened from baseline. Treatment-Related AEs are those with a definite, probable, possible or missing causality. A serious AE is a medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is a medical event requiring intervention to prevent 1 of the above. A severe or life-threatening AE is based on intensity, according to National Cancer Institute-Common Toxicity Criteria for Adverse Effects (NCI-CTCAE) v3.0.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
154 participants
Primary outcome timeframe
From Baseline to the interim clinical cut-off date (31 December 2009). The median time on study was 14.8 months, with a range of 26 days to 29.7 months. As of March 1, 2011 the total exposure to ruxolitinib was 269 patient-years.
Results posted on
2018-03-12
Participant Flow
This was a non-randomized dose-ranging study. Participants were enrolled into the currently available cohort based on the timeframe when they entered the study.
Participant milestones
| Measure |
10 mg Bid
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
25 mg qd
Participants received an initial dose of Ruxolitinib 25 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely with dose adjustments for safety and efficacy.
|
50 mg qd
Participants received an initial dose of Ruxolitinib 50 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
100 mg qd
Participants received an initial dose of Ruxolitinib 100 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
200 mg qd
Participants received an initial dose of Ruxolitinib 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
|---|---|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
30
|
35
|
47
|
5
|
6
|
22
|
6
|
3
|
|
Overall Study
COMPLETED
|
14
|
26
|
27
|
1
|
1
|
12
|
5
|
2
|
|
Overall Study
NOT COMPLETED
|
16
|
9
|
20
|
4
|
5
|
10
|
1
|
1
|
Reasons for withdrawal
| Measure |
10 mg Bid
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
25 mg qd
Participants received an initial dose of Ruxolitinib 25 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely with dose adjustments for safety and efficacy.
|
50 mg qd
Participants received an initial dose of Ruxolitinib 50 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
100 mg qd
Participants received an initial dose of Ruxolitinib 100 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
200 mg qd
Participants received an initial dose of Ruxolitinib 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
|---|---|---|---|---|---|---|---|---|
|
Overall Study
Progressive disease
|
1
|
3
|
5
|
1
|
0
|
1
|
0
|
0
|
|
Overall Study
Unacceptable Toxicity
|
1
|
0
|
0
|
2
|
0
|
0
|
0
|
0
|
|
Overall Study
Withdrawal by Subject
|
6
|
2
|
3
|
0
|
3
|
2
|
0
|
0
|
|
Overall Study
Physician Decision
|
4
|
2
|
7
|
0
|
1
|
4
|
0
|
0
|
|
Overall Study
Intercurrent Illness
|
1
|
0
|
0
|
1
|
0
|
1
|
0
|
0
|
|
Overall Study
Death
|
2
|
2
|
3
|
0
|
1
|
1
|
1
|
1
|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
2
|
0
|
0
|
1
|
0
|
0
|
Baseline Characteristics
Open Label Ruxolitinib (INCB018424) in Patients With Myelofibrosis and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis
Baseline characteristics by cohort
| Measure |
10 mg Bid
n=30 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=35 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=47 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=5 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
25 mg qd
n=6 Participants
Participants received an initial dose of Ruxolitinib 25 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely with dose adjustments for safety and efficacy.
|
50 mg qd
n=22 Participants
Participants received an initial dose of Ruxolitinib 50 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
100 mg qd
n=6 Participants
Participants received an initial dose of Ruxolitinib 100 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
200 mg qd
n=3 Participants
Participants received an initial dose of Ruxolitinib 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
Total
n=154 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|---|---|
|
JAK2 V617F mutation status
Unknown
|
7 participants
n=99 Participants
|
1 participants
n=107 Participants
|
3 participants
n=206 Participants
|
2 participants
n=7 Participants
|
0 participants
n=31 Participants
|
0 participants
n=30 Participants
|
3 participants
n=3 Participants
|
0 participants
n=6 Participants
|
16 participants
n=114 Participants
|
|
Age, Continuous
|
61.6 years
STANDARD_DEVIATION 8.29 • n=99 Participants
|
63.8 years
STANDARD_DEVIATION 9.57 • n=107 Participants
|
63.9 years
STANDARD_DEVIATION 9.06 • n=206 Participants
|
63.0 years
STANDARD_DEVIATION 8.37 • n=7 Participants
|
57.3 years
STANDARD_DEVIATION 9.89 • n=31 Participants
|
66.3 years
STANDARD_DEVIATION 6.66 • n=30 Participants
|
71.5 years
STANDARD_DEVIATION 6.89 • n=3 Participants
|
72.3 years
STANDARD_DEVIATION 6.43 • n=6 Participants
|
63.9 years
STANDARD_DEVIATION 8.86 • n=114 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
12 Participants
n=30 Participants
|
1 Participants
n=3 Participants
|
1 Participants
n=6 Participants
|
57 Participants
n=114 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=99 Participants
|
23 Participants
n=107 Participants
|
31 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=31 Participants
|
10 Participants
n=30 Participants
|
5 Participants
n=3 Participants
|
2 Participants
n=6 Participants
|
97 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
0 Participants
n=30 Participants
|
1 Participants
n=3 Participants
|
0 Participants
n=6 Participants
|
2 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
1 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=6 Participants
|
2 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
White
|
28 Participants
n=99 Participants
|
35 Participants
n=107 Participants
|
45 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=31 Participants
|
20 Participants
n=30 Participants
|
5 Participants
n=3 Participants
|
3 Participants
n=6 Participants
|
147 Participants
n=114 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
1 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=6 Participants
|
3 Participants
n=114 Participants
|
|
Disease sub-type
Primary myelofibrosis
|
12 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=31 Participants
|
14 Participants
n=30 Participants
|
5 Participants
n=3 Participants
|
0 Participants
n=6 Participants
|
81 Participants
n=114 Participants
|
|
Disease sub-type
Post-polycythemia vera-myelofibrosis
|
13 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
2 Participants
n=7 Participants
|
1 Participants
n=31 Participants
|
4 Participants
n=30 Participants
|
1 Participants
n=3 Participants
|
3 Participants
n=6 Participants
|
49 Participants
n=114 Participants
|
|
Disease sub-type
Post-essential thrombocythemia-myelofibrosis
|
5 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=31 Participants
|
4 Participants
n=30 Participants
|
0 Participants
n=3 Participants
|
0 Participants
n=6 Participants
|
24 Participants
n=114 Participants
|
|
Body Mass Index (BMI)
|
25.8 kg/m^2
STANDARD_DEVIATION 3.8 • n=99 Participants
|
25.3 kg/m^2
STANDARD_DEVIATION 4.4 • n=107 Participants
|
26.2 kg/m^2
STANDARD_DEVIATION 5.9 • n=206 Participants
|
24.8 kg/m^2
STANDARD_DEVIATION 2.9 • n=7 Participants
|
24.7 kg/m^2
STANDARD_DEVIATION 2.9 • n=31 Participants
|
25.3 kg/m^2
STANDARD_DEVIATION 4.5 • n=30 Participants
|
25.8 kg/m^2
STANDARD_DEVIATION 6.3 • n=3 Participants
|
26.3 kg/m^2
STANDARD_DEVIATION 3.8 • n=6 Participants
|
25.7 kg/m^2
STANDARD_DEVIATION 4.7 • n=114 Participants
|
|
Palpable spleen length below the costal margin
|
19.11 cm
STANDARD_DEVIATION 8.2 • n=99 Participants
|
18.47 cm
STANDARD_DEVIATION 5.7 • n=107 Participants
|
17.13 cm
STANDARD_DEVIATION 8.9 • n=206 Participants
|
13.0 cm
STANDARD_DEVIATION 10.1 • n=7 Participants
|
19.58 cm
STANDARD_DEVIATION 10.5 • n=31 Participants
|
20.0 cm
STANDARD_DEVIATION 7.3 • n=30 Participants
|
20.67 cm
STANDARD_DEVIATION 6.6 • n=3 Participants
|
18.33 cm
STANDARD_DEVIATION 3.5 • n=6 Participants
|
18.37 cm
STANDARD_DEVIATION 7.8 • n=114 Participants
|
|
JAK2 V617F mutation status
Negative
|
1 participants
n=99 Participants
|
5 participants
n=107 Participants
|
5 participants
n=206 Participants
|
1 participants
n=7 Participants
|
4 participants
n=31 Participants
|
7 participants
n=30 Participants
|
1 participants
n=3 Participants
|
0 participants
n=6 Participants
|
24 participants
n=114 Participants
|
|
JAK2 V617F mutation status
Positive
|
22 participants
n=99 Participants
|
29 participants
n=107 Participants
|
39 participants
n=206 Participants
|
2 participants
n=7 Participants
|
2 participants
n=31 Participants
|
15 participants
n=30 Participants
|
2 participants
n=3 Participants
|
3 participants
n=6 Participants
|
114 participants
n=114 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
0
|
7 participants
n=99 Participants
|
0 participants
n=107 Participants
|
9 participants
n=206 Participants
|
2 participants
n=7 Participants
|
1 participants
n=31 Participants
|
5 participants
n=30 Participants
|
1 participants
n=3 Participants
|
0 participants
n=6 Participants
|
25 participants
n=114 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
1
|
19 participants
n=99 Participants
|
32 participants
n=107 Participants
|
33 participants
n=206 Participants
|
3 participants
n=7 Participants
|
4 participants
n=31 Participants
|
14 participants
n=30 Participants
|
5 participants
n=3 Participants
|
3 participants
n=6 Participants
|
113 participants
n=114 Participants
|
|
Eastern Cooperative Oncology Group Performance Status
2
|
4 participants
n=99 Participants
|
3 participants
n=107 Participants
|
5 participants
n=206 Participants
|
0 participants
n=7 Participants
|
1 participants
n=31 Participants
|
3 participants
n=30 Participants
|
0 participants
n=3 Participants
|
0 participants
n=6 Participants
|
16 participants
n=114 Participants
|
PRIMARY outcome
Timeframe: From Baseline to the interim clinical cut-off date (31 December 2009). The median time on study was 14.8 months, with a range of 26 days to 29.7 months. As of March 1, 2011 the total exposure to ruxolitinib was 269 patient-years.Population: Safety population included all patients who received at least 1 dose of study medication.
Treatment-Emergent AEs are events occurring after first drug administration or worsened from baseline. Treatment-Related AEs are those with a definite, probable, possible or missing causality. A serious AE is a medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is a medical event requiring intervention to prevent 1 of the above. A severe or life-threatening AE is based on intensity, according to National Cancer Institute-Common Toxicity Criteria for Adverse Effects (NCI-CTCAE) v3.0.
Outcome measures
| Measure |
10 mg Bid
n=30 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=35 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=47 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=5 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
n=37 Participants
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Number of Participants With Adverse Events (AEs)
Any Treatment-Emergent Adverse Event
|
30 participants
|
35 participants
|
47 participants
|
5 participants
|
37 participants
|
|
Number of Participants With Adverse Events (AEs)
Treatment-Related Adverse Events
|
21 participants
|
22 participants
|
41 participants
|
4 participants
|
29 participants
|
|
Number of Participants With Adverse Events (AEs)
Serious Adverse Events
|
8 participants
|
17 participants
|
21 participants
|
2 participants
|
23 participants
|
|
Number of Participants With Adverse Events (AEs)
Severe or Life-threatening Adverse Events
|
21 participants
|
25 participants
|
39 participants
|
3 participants
|
28 participants
|
|
Number of Participants With Adverse Events (AEs)
Study Drug Interrupted Due to Adverse Events
|
9 participants
|
9 participants
|
20 participants
|
3 participants
|
23 participants
|
|
Number of Participants With Adverse Events (AEs)
Discontinued Study Drug Due to Adverse Events
|
6 participants
|
0 participants
|
7 participants
|
2 participants
|
5 participants
|
|
Number of Participants With Adverse Events (AEs)
Study Drug Reduced Due to Adverse Events
|
8 participants
|
12 participants
|
26 participants
|
1 participants
|
11 participants
|
PRIMARY outcome
Timeframe: Week 12, 24, 36, 48 and 60Population: The intent-to-treat population included all patients who received at least 1 dose of study medication and had at least 1 follow-up assessment for safety and efficacy. N = the number of patients who had clinical response assessed during the time interval.
Clinical improvement was defined according to the International Working Group Myelofibrosis Research and Treatment criteria, and required 1 of the following: 1. A ≥ 2 g/dL increase in Hemoglobin level or becoming transfusion independent; 2. Either a ≥ 50% reduction in palpable splenomegaly if spleen was ≥ 10 cm at Baseline or a spleen palpable at \> 5 cm at Baseline becomes not palpable; 3. A ≥ 100% increase in platelet count and an absolute platelet count of ≥ 50,000 x 10\^9/L or 4. A ≥ 100% increase in absolute neutrophil count (ANC) and an ANC of ≥ 0.5 x 10\^9/L.
Outcome measures
| Measure |
10 mg Bid
n=25 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=34 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=42 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=5 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
n=32 Participants
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Percentage of Participants With Clinical Improvement (CI) Over Time
12 Weeks [N=25, 34, 42, 5, 32]
|
36.0 percentage of participants
|
38.2 percentage of participants
|
38.1 percentage of participants
|
40.0 percentage of participants
|
34.4 percentage of participants
|
|
Percentage of Participants With Clinical Improvement (CI) Over Time
24 Weeks [N=21, 31, 40, 4, 29]
|
42.9 percentage of participants
|
51.6 percentage of participants
|
37.5 percentage of participants
|
75.0 percentage of participants
|
37.9 percentage of participants
|
|
Percentage of Participants With Clinical Improvement (CI) Over Time
36 Weeks [N=18, 30, 36, 4, 25]
|
66.7 percentage of participants
|
56.7 percentage of participants
|
38.9 percentage of participants
|
50.0 percentage of participants
|
56.0 percentage of participants
|
|
Percentage of Participants With Clinical Improvement (CI) Over Time
48 Weeks [N=17, 28, 36, 3, 23]
|
47.1 percentage of participants
|
67.9 percentage of participants
|
41.7 percentage of participants
|
33.3 percentage of participants
|
56.5 percentage of participants
|
|
Percentage of Participants With Clinical Improvement (CI) Over Time
60 Weeks [N=15, 21, 33, 2, 21]
|
53.3 percentage of participants
|
66.7 percentage of participants
|
54.5 percentage of participants
|
50.0 percentage of participants
|
61.9 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline and Weeks 4, 8, 12, 24, 36, 48 and 60Population: Intent to treat population. A total of 16 patients had either a splenectomy prior to study entry, missing Baseline spleen values or had spleen lengths reported as 0 cm and were excluded.
For each visit, patients who had a missing value at the visit, dropped out of the study due to any reasons prior to the visit or had non-palpable spleen at baseline and then became palpable at the time of the visit were all considered as having not achieved the ≥ 50% reduction in spleen palpation length.
Outcome measures
| Measure |
10 mg Bid
n=27 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=34 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=37 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=4 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
n=36 Participants
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 4 [N=27, 34, 37, 4, 36]
|
33.3 percentage of participants
|
38.2 percentage of participants
|
43.2 percentage of participants
|
75.0 percentage of participants
|
30.6 percentage of participants
|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 8 [N=27, 34, 37, 4, 36]
|
29.6 percentage of participants
|
50.0 percentage of participants
|
43.2 percentage of participants
|
75.0 percentage of participants
|
30.6 percentage of participants
|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 12 [N=27, 34, 37, 4, 36]
|
33.3 percentage of participants
|
47.1 percentage of participants
|
54.1 percentage of participants
|
75.0 percentage of participants
|
33.3 percentage of participants
|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 24 [N=26, 34, 37, 4, 36]
|
34.6 percentage of participants
|
52.9 percentage of participants
|
48.6 percentage of participants
|
50.0 percentage of participants
|
36.1 percentage of participants
|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 36 [N=26, 34, 37, 4, 36]
|
30.8 percentage of participants
|
47.1 percentage of participants
|
43.2 percentage of participants
|
50.0 percentage of participants
|
36.1 percentage of participants
|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 48 [N=26, 33, 37, 4, 36]
|
23.1 percentage of participants
|
54.5 percentage of participants
|
40.5 percentage of participants
|
25.0 percentage of participants
|
27.8 percentage of participants
|
|
Percentage of Participants Achieving ≥ 50% Reduction From Baseline in Spleen Palpation Length Over Time
Week 60 [N=25, 27, 37, 4, 36]
|
20.0 percentage of participants
|
51.9 percentage of participants
|
43.2 percentage of participants
|
25.0 percentage of participants
|
25.0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 4, 12, 24 and 48Population: Patients with at least 1 Spleen-Volume Measurement. Patients who had not reached the visit were excluded from the analysis. In addition, at Week 48, 5 patients who did not have MRI measurement due to a protocol amendment were considered as not evaluable and were excluded from the analysis.
Spleen volume was assessed in a subgroup of 27 patients using magnetic resonance imaging (MRI) scans (or computed tomography (CT) scans in patients who were not candidates for MRI) of the abdomen in order to allow objective measurement of spleen volume using standard estimation techniques. For each visit, patients who had a missing value at the visit or dropped out of the study due to any reason prior to the visit were considered as not having achieved the ≥35% reduction in spleen volume.
Outcome measures
| Measure |
10 mg Bid
n=2 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=25 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume Over Time
Week 4 [N= 2, 25]
|
0.0 percentage of participants
|
44.0 percentage of participants
|
—
|
—
|
—
|
|
Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume Over Time
Week 12 [N= 2, 25]
|
0.0 percentage of participants
|
48.0 percentage of participants
|
—
|
—
|
—
|
|
Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume Over Time
Week 24 [N= 2, 25]
|
0.0 percentage of participants
|
44.0 percentage of participants
|
—
|
—
|
—
|
|
Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume Over Time
Week 48 [N= 2, 20]
|
0.0 percentage of participants
|
40.0 percentage of participants
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 24Population: Intent-to-treat population for whom data was available. The MFSAF was implemented by protocol amendment while the study was ongoing. Hence data are available for only approximately 50% of enrolled patients. This analysis includes patients with a Baseline total symptom score ≥ 0; 8 patients were excluded because they had a Baseline score = 0.
Symptoms of myelofibrosis were assessed using a modified Myelofibrosis Symptom Assessment Form (MFSAF). Abdominal discomfort, itching, muscle or bone pain, and night sweats are prominent and troubling symptoms in patients with MF. Therefore, the MFSAF-derived responses for these symptoms were analyzed as a total symptom score. Each symptom was assessed on a scale from 0 (absent), 1 (most favorable) to 10 (worst). The total symptom score is a sum of the individual scores and ranges from 0-40. A higher score indicates worse symptoms hence a negative change from baseline indicates improvement.
Outcome measures
| Measure |
10 mg Bid
n=9 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=26 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=11 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=17 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Change From Baseline in Myelofibrosis Total Symptom Score at Week 24
|
-4.7 scores on a scale
Standard Deviation 5.89
|
-5.6 scores on a scale
Standard Deviation 7.82
|
0.9 scores on a scale
Standard Deviation 8.37
|
-6.5 scores on a scale
Standard Deviation 6.38
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 24Population: The safety population included all subjects who received at least 1 dose of study medication, and for whom data was available at both time points. The EORTC QLQ C30 was implemented by protocol amendment and as a result this data are available for only approximately 50% of the enrolled patients.
Health-related Quality of Life was assessed using the Global Health Status/Quality of Life Scale of the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30). This scale ranges from 0 to 100, with higher scores indicating higher quality of life.
Outcome measures
| Measure |
10 mg Bid
n=9 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=28 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=13 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=20 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Change From Baseline to Week 24 in Health-Related Quality of Life
|
14.81 units on a scale
Standard Deviation 21.968
|
12.80 units on a scale
Standard Deviation 22.620
|
0.63 units on a scale
Standard Deviation 20.254
|
9.16 units on a scale
Standard Deviation 28.725
|
—
|
SECONDARY outcome
Timeframe: Baseline and Weeks 4, 8, 12, 24, 36, 48 and 60.Population: Intent-to-treat population for patients who had reached each time point and for whom data was available.
Outcome measures
| Measure |
10 mg Bid
n=146 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Change From Baseline in Body Weight Over Time
Week 4 [n=139]
|
0.30 kg
Standard Deviation 2.656
|
—
|
—
|
—
|
—
|
|
Change From Baseline in Body Weight Over Time
Week 8 [n=137]
|
1.59 kg
Standard Deviation 2.773
|
—
|
—
|
—
|
—
|
|
Change From Baseline in Body Weight Over Time
Week 12 [n=129]
|
2.44 kg
Standard Deviation 3.596
|
—
|
—
|
—
|
—
|
|
Change From Baseline in Body Weight Over Time
Week 24 [n=115]
|
4.44 kg
Standard Deviation 4.422
|
—
|
—
|
—
|
—
|
|
Change From Baseline in Body Weight Over Time
Week 36 [n=104]
|
5.58 kg
Standard Deviation 4.790
|
—
|
—
|
—
|
—
|
|
Change From Baseline in Body Weight Over Time
Week 48 [n=99]
|
6.35 kg
Standard Deviation 5.845
|
—
|
—
|
—
|
—
|
|
Change From Baseline in Body Weight Over Time
Week 60 [n=83]
|
6.60 kg
Standard Deviation 5.451
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline and Week 24Population: Intent-to-treat population for patients who had reached each time point and for whom data was available.
The ECOG performance status measures patients' functional status on the following scale: * 0=Fully active, no restrictions; * 1=Restricted in physically strenuous activity but ambulatory, able to carry out light work; * 2=Ambulatory and capable of all selfcare, unable to carry out any work activities; Up and about \> 50% of waking hours; * 3=Limited selfcare, confined to bed or chair more than 50% of waking hours; * 4=Completely disabled. Totally confined to bed or chair; * 5=Dead. Data reported indicate the number of participants with a change from Baseline score of -2, -1, 0 and 1.
Outcome measures
| Measure |
10 mg Bid
n=20 Participants
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=31 Participants
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=39 Participants
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=4 Participants
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
n=29 Participants
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Change From Baseline to Week 24 in Eastern Cooperative Oncology Group (ECOG) Performance Status
Change from Baseline of -2
|
1 participants
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Change From Baseline to Week 24 in Eastern Cooperative Oncology Group (ECOG) Performance Status
Change from Baseline of -1
|
4 participants
|
15 participants
|
16 participants
|
2 participants
|
15 participants
|
|
Change From Baseline to Week 24 in Eastern Cooperative Oncology Group (ECOG) Performance Status
Change from Baseline of 0
|
12 participants
|
16 participants
|
18 participants
|
1 participants
|
11 participants
|
|
Change From Baseline to Week 24 in Eastern Cooperative Oncology Group (ECOG) Performance Status
Change from Baseline of 1
|
3 participants
|
0 participants
|
4 participants
|
1 participants
|
3 participants
|
Adverse Events
10 mg Bid
Serious events: 8 serious events
Other events: 29 other events
Deaths: 0 deaths
15 mg Bid
Serious events: 17 serious events
Other events: 35 other events
Deaths: 0 deaths
25 mg Bid
Serious events: 21 serious events
Other events: 47 other events
Deaths: 0 deaths
50 mg Bid
Serious events: 2 serious events
Other events: 5 other events
Deaths: 0 deaths
All QD
Serious events: 23 serious events
Other events: 36 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
10 mg Bid
n=30 participants at risk
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=35 participants at risk
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=47 participants at risk
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=5 participants at risk
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
n=37 participants at risk
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
20.0%
1/5
|
8.1%
3/37
|
|
Blood and lymphatic system disorders
Bone marrow failure
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Blood and lymphatic system disorders
Extramedullary haemopoiesis
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Blood and lymphatic system disorders
Splenomegaly
|
3.3%
1/30
|
8.6%
3/35
|
4.3%
2/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Cardiac disorders
Atrial tachycardia
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Cardiac disorders
Atrioventricular block
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Cardiac disorders
Cardiac arrest
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Cardiac disorders
Cardiac failure
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Cardiac disorders
Cardiac failure congestive
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Cardiac disorders
Coronary artery disease
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Cardiac disorders
Myocardial infarction
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Cardiac disorders
Myocardial ischaemia
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Cardiac disorders
Pericarditis
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Odynophagia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Pancreatic mass
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
General disorders
Asthenia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
5.4%
2/37
|
|
General disorders
Chest pain
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
General disorders
Disease progression
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
General disorders
General symptom
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
General disorders
Hyperpyrexia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
General disorders
Multi-organ failure
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
General disorders
Pyrexia
|
0.00%
0/30
|
0.00%
0/35
|
6.4%
3/47
|
0.00%
0/5
|
5.4%
2/37
|
|
General disorders
Systemic inflammatory response syndrome
|
0.00%
0/30
|
2.9%
1/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Hepatobiliary disorders
Cholestasis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Hepatobiliary disorders
Portal vein thrombosis
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Appendicitis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Bronchitis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Cellulitis
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Infections and infestations
Clostridium difficile colitis
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Diverticulitis
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Herpes zoster
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Infection
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Lung infection
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Pharyngotonsillitis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Pneumocystis jiroveci pneumonia
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Pneumonia
|
6.7%
2/30
|
0.00%
0/35
|
6.4%
3/47
|
20.0%
1/5
|
13.5%
5/37
|
|
Infections and infestations
Pseudomembranous colitis
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Sepsis
|
0.00%
0/30
|
0.00%
0/35
|
4.3%
2/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Septic shock
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Sinusitis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Injury, poisoning and procedural complications
Patella fracture
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Injury, poisoning and procedural complications
Splenic rupture
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Musculoskeletal and connective tissue disorders
Gouty arthritis
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Musculoskeletal and connective tissue disorders
Spinal column stenosis
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
B-cell lymphoma
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer
|
0.00%
0/30
|
2.9%
1/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Chronic myelomonocytic leukaemia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hairy cell leukaemia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Nervous system disorders
Carotid artery stenosis
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Nervous system disorders
Carpal tunnel syndrome
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Nervous system disorders
Cerebral haemorrhage
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Nervous system disorders
Nerve compression
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Nervous system disorders
Post herpetic neuralgia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Nervous system disorders
Syncope
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Psychiatric disorders
Depression
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Psychiatric disorders
Mood altered
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Renal and urinary disorders
Hydronephrosis
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Renal and urinary disorders
Renal pain
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Reproductive system and breast disorders
Ovarian cyst ruptured
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Reproductive system and breast disorders
Prostatic haemorrhage
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Reproductive system and breast disorders
Scrotal pain
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/30
|
0.00%
0/35
|
4.3%
2/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Respiratory, thoracic and mediastinal disorders
Obstructive airways disorder
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Respiratory, thoracic and mediastinal disorders
Orthopnoea
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Vascular disorders
Haematoma
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Vascular disorders
Hypotension
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
Other adverse events
| Measure |
10 mg Bid
n=30 participants at risk
Participants received an initial dose of Ruxolitinib 10 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
15 mg Bid
n=35 participants at risk
Participants received an initial dose of Ruxolitinib 15 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy to a maximum of 25 mg bid.
|
25 mg Bid
n=47 participants at risk
Participants received an initial dose of Ruxolitinib 25 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
50 mg Bid
n=5 participants at risk
Participants received an initial dose of Ruxolitinib 50 mg twice a day (bid). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy.
|
All QD
n=37 participants at risk
Participants received an initial dose of Ruxolitinib 25, 50, 100 or 200 mg once a day (qd). Participants could continue in the study on their prescribed regimen indefinitely if receiving benefit with dose adjustments for safety and efficacy. Since the numbers of patients in 3 of the qd treatment groups were small, the 4 qd doses were combined to allow meaningful comparisons against the bid treatment groups.
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
46.7%
14/30
|
40.0%
14/35
|
57.4%
27/47
|
0.00%
0/5
|
48.6%
18/37
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
40.0%
12/30
|
31.4%
11/35
|
61.7%
29/47
|
80.0%
4/5
|
37.8%
14/37
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
3.3%
1/30
|
5.7%
2/35
|
4.3%
2/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Blood and lymphatic system disorders
Neutropenia
|
3.3%
1/30
|
5.7%
2/35
|
0.00%
0/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Blood and lymphatic system disorders
Coagulopathy
|
0.00%
0/30
|
5.7%
2/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Blood and lymphatic system disorders
Thrombocythaemia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Cardiac disorders
Extrasystoles
|
6.7%
2/30
|
2.9%
1/35
|
2.1%
1/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
5.4%
2/37
|
|
Cardiac disorders
Palpitations
|
6.7%
2/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Ear and labyrinth disorders
Vertigo
|
3.3%
1/30
|
5.7%
2/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Diarrhoea
|
26.7%
8/30
|
34.3%
12/35
|
31.9%
15/47
|
20.0%
1/5
|
16.2%
6/37
|
|
Gastrointestinal disorders
Nausea
|
23.3%
7/30
|
11.4%
4/35
|
12.8%
6/47
|
20.0%
1/5
|
16.2%
6/37
|
|
Gastrointestinal disorders
Vomiting
|
13.3%
4/30
|
11.4%
4/35
|
12.8%
6/47
|
0.00%
0/5
|
13.5%
5/37
|
|
Gastrointestinal disorders
Abdominal pain
|
16.7%
5/30
|
17.1%
6/35
|
10.6%
5/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Gastrointestinal disorders
Abdominal distension
|
3.3%
1/30
|
11.4%
4/35
|
8.5%
4/47
|
20.0%
1/5
|
8.1%
3/37
|
|
Gastrointestinal disorders
Constipation
|
10.0%
3/30
|
14.3%
5/35
|
8.5%
4/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/30
|
2.9%
1/35
|
10.6%
5/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Flatulence
|
0.00%
0/30
|
11.4%
4/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Abdominal pain upper
|
3.3%
1/30
|
5.7%
2/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Abdominal discomfort
|
3.3%
1/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Gastrointestinal disorders
Ascites
|
6.7%
2/30
|
2.9%
1/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Gastrointestinal disorders
Epigastric discomfort
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Gastrointestinal disorders
Glossodynia
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
General disorders
Fatigue
|
23.3%
7/30
|
28.6%
10/35
|
23.4%
11/47
|
20.0%
1/5
|
18.9%
7/37
|
|
General disorders
Edema peripheral
|
6.7%
2/30
|
22.9%
8/35
|
21.3%
10/47
|
60.0%
3/5
|
0.00%
0/37
|
|
General disorders
Pyrexia
|
3.3%
1/30
|
11.4%
4/35
|
12.8%
6/47
|
40.0%
2/5
|
13.5%
5/37
|
|
General disorders
Oedema
|
6.7%
2/30
|
2.9%
1/35
|
10.6%
5/47
|
0.00%
0/5
|
0.00%
0/37
|
|
General disorders
Chills
|
3.3%
1/30
|
5.7%
2/35
|
4.3%
2/47
|
0.00%
0/5
|
2.7%
1/37
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/30
|
2.9%
1/35
|
2.1%
1/47
|
0.00%
0/5
|
10.8%
4/37
|
|
General disorders
Pitting oedema
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Hepatobiliary disorders
Hepatomegaly
|
3.3%
1/30
|
2.9%
1/35
|
6.4%
3/47
|
20.0%
1/5
|
8.1%
3/37
|
|
Hepatobiliary disorders
Caput medusae
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
2/30
|
11.4%
4/35
|
10.6%
5/47
|
20.0%
1/5
|
13.5%
5/37
|
|
Infections and infestations
Pneumonia
|
0.00%
0/30
|
8.6%
3/35
|
8.5%
4/47
|
0.00%
0/5
|
16.2%
6/37
|
|
Infections and infestations
Urinary tract infection
|
10.0%
3/30
|
5.7%
2/35
|
8.5%
4/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Infections and infestations
Bronchitis
|
6.7%
2/30
|
8.6%
3/35
|
6.4%
3/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Infections and infestations
Herpes zoster
|
13.3%
4/30
|
0.00%
0/35
|
10.6%
5/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Infections and infestations
Nasopharyngitis
|
10.0%
3/30
|
0.00%
0/35
|
8.5%
4/47
|
40.0%
2/5
|
2.7%
1/37
|
|
Infections and infestations
Sinusitis
|
0.00%
0/30
|
5.7%
2/35
|
8.5%
4/47
|
0.00%
0/5
|
10.8%
4/37
|
|
Infections and infestations
Influenza
|
3.3%
1/30
|
8.6%
3/35
|
4.3%
2/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Infections and infestations
Folliculitis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Infections and infestations
Tinea pedis
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Infections and infestations
Tinea versicolour
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Injury, poisoning and procedural complications
Contusion
|
10.0%
3/30
|
11.4%
4/35
|
8.5%
4/47
|
0.00%
0/5
|
16.2%
6/37
|
|
Injury, poisoning and procedural complications
Excoriation
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Investigations
Cardiac murmur
|
20.0%
6/30
|
20.0%
7/35
|
14.9%
7/47
|
0.00%
0/5
|
18.9%
7/37
|
|
Investigations
Weight increased
|
10.0%
3/30
|
11.4%
4/35
|
19.1%
9/47
|
20.0%
1/5
|
21.6%
8/37
|
|
Investigations
Electrocardiogram QT prolonged
|
0.00%
0/30
|
0.00%
0/35
|
8.5%
4/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Investigations
Haemoglobin decreased
|
3.3%
1/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
8.1%
3/37
|
|
Investigations
Weight decreased
|
6.7%
2/30
|
2.9%
1/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Investigations
Neutrophil count decreased
|
3.3%
1/30
|
2.9%
1/35
|
2.1%
1/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Investigations
Gamma-glutamyltransferase increased
|
0.00%
0/30
|
5.7%
2/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Investigations
Arterial bruit
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Investigations
Troponin increased
|
0.00%
0/30
|
5.7%
2/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Investigations
Blood pressure increased
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Investigations
Body temperature increased
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
3.3%
1/30
|
5.7%
2/35
|
14.9%
7/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/30
|
5.7%
2/35
|
6.4%
3/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
3.3%
1/30
|
5.7%
2/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
0.00%
0/30
|
8.6%
3/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
3.3%
1/30
|
0.00%
0/35
|
6.4%
3/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Metabolism and nutrition disorders
Iron overload
|
6.7%
2/30
|
0.00%
0/35
|
4.3%
2/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.0%
3/30
|
11.4%
4/35
|
23.4%
11/47
|
20.0%
1/5
|
13.5%
5/37
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.7%
2/30
|
22.9%
8/35
|
10.6%
5/47
|
40.0%
2/5
|
13.5%
5/37
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.7%
2/30
|
11.4%
4/35
|
12.8%
6/47
|
0.00%
0/5
|
8.1%
3/37
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
13.3%
4/30
|
8.6%
3/35
|
8.5%
4/47
|
0.00%
0/5
|
8.1%
3/37
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.7%
2/30
|
5.7%
2/35
|
8.5%
4/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
3.3%
1/30
|
5.7%
2/35
|
8.5%
4/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
10.0%
3/30
|
2.9%
1/35
|
6.4%
3/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
3/30
|
5.7%
2/35
|
2.1%
1/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
0.00%
0/30
|
0.00%
0/35
|
8.5%
4/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
0.00%
0/30
|
0.00%
0/35
|
4.3%
2/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Dysplastic naevus syndrome
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Fibrous histiocytoma
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Seborrhoeic keratosis
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Nervous system disorders
Headache
|
20.0%
6/30
|
17.1%
6/35
|
10.6%
5/47
|
60.0%
3/5
|
16.2%
6/37
|
|
Nervous system disorders
Dizziness
|
6.7%
2/30
|
8.6%
3/35
|
6.4%
3/47
|
0.00%
0/5
|
13.5%
5/37
|
|
Nervous system disorders
Neuropathy peripheral
|
6.7%
2/30
|
2.9%
1/35
|
4.3%
2/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/30
|
5.7%
2/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Psychiatric disorders
Insomnia
|
6.7%
2/30
|
11.4%
4/35
|
6.4%
3/47
|
0.00%
0/5
|
16.2%
6/37
|
|
Psychiatric disorders
Depression
|
6.7%
2/30
|
2.9%
1/35
|
8.5%
4/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Psychiatric disorders
Anxiety
|
3.3%
1/30
|
0.00%
0/35
|
6.4%
3/47
|
20.0%
1/5
|
5.4%
2/37
|
|
Renal and urinary disorders
Pollakiuria
|
3.3%
1/30
|
8.6%
3/35
|
4.3%
2/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/30
|
5.7%
2/35
|
6.4%
3/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.7%
2/30
|
20.0%
7/35
|
19.1%
9/47
|
20.0%
1/5
|
10.8%
4/37
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.7%
2/30
|
11.4%
4/35
|
14.9%
7/47
|
0.00%
0/5
|
27.0%
10/37
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.0%
3/30
|
0.00%
0/35
|
10.6%
5/47
|
20.0%
1/5
|
5.4%
2/37
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
10.0%
3/30
|
2.9%
1/35
|
4.3%
2/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
3.3%
1/30
|
2.9%
1/35
|
6.4%
3/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/30
|
0.00%
0/35
|
6.4%
3/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
0.00%
0/30
|
5.7%
2/35
|
0.00%
0/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal blistering
|
0.00%
0/30
|
2.9%
1/35
|
0.00%
0/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
6.7%
2/30
|
14.3%
5/35
|
19.1%
9/47
|
0.00%
0/5
|
16.2%
6/37
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
6.7%
2/30
|
17.1%
6/35
|
10.6%
5/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.7%
2/30
|
5.7%
2/35
|
12.8%
6/47
|
0.00%
0/5
|
8.1%
3/37
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.7%
2/30
|
5.7%
2/35
|
4.3%
2/47
|
0.00%
0/5
|
10.8%
4/37
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
6.7%
2/30
|
0.00%
0/35
|
8.5%
4/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
3.3%
1/30
|
0.00%
0/35
|
2.1%
1/47
|
20.0%
1/5
|
5.4%
2/37
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/30
|
0.00%
0/35
|
6.4%
3/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Skin and subcutaneous tissue disorders
Photodermatosis
|
3.3%
1/30
|
5.7%
2/35
|
0.00%
0/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
0.00%
0/30
|
5.7%
2/35
|
2.1%
1/47
|
0.00%
0/5
|
2.7%
1/37
|
|
Skin and subcutaneous tissue disorders
Actinic keratosis
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
20.0%
1/5
|
0.00%
0/37
|
|
Vascular disorders
Haematoma
|
3.3%
1/30
|
8.6%
3/35
|
2.1%
1/47
|
0.00%
0/5
|
0.00%
0/37
|
|
Vascular disorders
Hypertension
|
0.00%
0/30
|
5.7%
2/35
|
2.1%
1/47
|
20.0%
1/5
|
2.7%
1/37
|
|
Metabolism and nutrition disorders
Anorexia
|
3.3%
1/30
|
0.00%
0/35
|
4.3%
2/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Nervous system disorders
Lethargy
|
0.00%
0/30
|
0.00%
0/35
|
2.1%
1/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Psychiatric disorders
Agitation
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Psychiatric disorders
Mental status changes
|
3.3%
1/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary congestion
|
0.00%
0/30
|
0.00%
0/35
|
0.00%
0/47
|
0.00%
0/5
|
5.4%
2/37
|
Additional Information
Study Director
Incyte Corporation
Phone: 1-855-463-3463
Results disclosure agreements
- Principal investigator is a sponsor employee Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study; provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
- Publication restrictions are in place
Restriction type: OTHER