Trial Outcomes & Findings for Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients. (NCT NCT02966353)
NCT ID: NCT02966353
Last Updated: 2020-04-30
Results Overview
Percentage of participants achieving a 50% reduction in spleen length at week 24. For subjects with palpable spleen at baseline and non-palpable at post-baseline, the post-baseline spleen are imputed as 0. Subjects who had palpable, but missing spleen length at baseline is excluded from the analysis. Subjects with missing spleen length at Week 24 or who withdraw earlier from the study are considered as a non-responder. The 95% CI is computed using exact Clopper-Pearson method.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
51 participants
Primary outcome timeframe
Baseline up to week 24
Results posted on
2020-04-30
Participant Flow
Screening evaluations were performed to determine the eligibility for the study and establish a baseline prior to dosing.
Participant milestones
| Measure |
All Subjects
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Overall Study
STARTED
|
51
|
|
Overall Study
Completed After Data Cutoff
|
1
|
|
Overall Study
COMPLETED
|
29
|
|
Overall Study
NOT COMPLETED
|
22
|
Reasons for withdrawal
| Measure |
All Subjects
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
7
|
|
Overall Study
Adverse Event
|
4
|
|
Overall Study
Death
|
4
|
|
Overall Study
Physician Decision
|
3
|
|
Overall Study
Progressive disease
|
2
|
|
Overall Study
Protocol deviation
|
2
|
Baseline Characteristics
Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients.
Baseline characteristics by cohort
| Measure |
All Subjects
n=51 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
31 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
48 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
3 participants
n=5 Participants
|
|
Type of myelofibrosis
Primary myelofibrosis
|
34 participants
n=5 Participants
|
|
Type of myelofibrosis
Post-polycythemia vera myelofibrosis
|
6 participants
n=5 Participants
|
|
Type of myelofibrosis
Post-essential thrombocythemia myelofibrosis
|
11 participants
n=5 Participants
|
|
Time since intial diagnosis
|
32.9 months
STANDARD_DEVIATION 44.16 • n=5 Participants
|
|
Bone Marrow Fibrosis Grade at Diagnosis
Grade 0
|
0 participants
n=5 Participants
|
|
Bone Marrow Fibrosis Grade at Diagnosis
Grade 1
|
6 participants
n=5 Participants
|
|
Bone Marrow Fibrosis Grade at Diagnosis
Grade 2
|
26 participants
n=5 Participants
|
|
Bone Marrow Fibrosis Grade at Diagnosis
Grade 3
|
18 participants
n=5 Participants
|
|
Bone Marrow Fibrosis Grade at Diagnosis
Missing
|
1 participants
n=5 Participants
|
|
Constitutional symptoms
Present
|
29 participants
n=5 Participants
|
|
Constitutional symptoms
Absent
|
22 participants
n=5 Participants
|
|
Palpable spleen length (cm) below costal margin
|
11.7 centimeters
STANDARD_DEVIATION 6.20 • n=5 Participants
|
|
Hemoglobin level
|
88.6 g/dL
STANDARD_DEVIATION 9.74 • n=5 Participants
|
|
Platelets
|
236.7 10E9/L
STANDARD_DEVIATION 176.88 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to week 24Percentage of participants achieving a 50% reduction in spleen length at week 24. For subjects with palpable spleen at baseline and non-palpable at post-baseline, the post-baseline spleen are imputed as 0. Subjects who had palpable, but missing spleen length at baseline is excluded from the analysis. Subjects with missing spleen length at Week 24 or who withdraw earlier from the study are considered as a non-responder. The 95% CI is computed using exact Clopper-Pearson method.
Outcome measures
| Measure |
All Subjects
n=50 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Percentage of Participants With at Least 50% Reduction in Spleen Length From Baseline at Week 24
|
56.0 percentage of participants
Interval 41.3 to 70.0
|
SECONDARY outcome
Timeframe: Baseline up to week 48Population: Full analysis set
Percentage of participants achieving a 50% reduction in spleen length at week 48. For subjects with palpable spleen at baseline and non-palpable at post-baseline, the post-baseline spleen is imputed as 0. Subjects who had palpable, but missing spleen length at baseline is excluded from the analysis. Subjects with missing spleen length at Week 48 or who withdraw earlier from the study are considered as a non-responder. The 95% CI is computed using exact Clopper-Pearson method.
Outcome measures
| Measure |
All Subjects
n=50 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Percentage of Participants With at Least 50% Reduction in Spleen Length From Baseline at Week 48
|
36.0 percentage of participants
Interval 22.9 to 50.8
|
SECONDARY outcome
Timeframe: baseline, weeks 24 and 48Population: Full analysis set n: participants with a value at both Baseline and that time point.
Participants who achieved a ≤ 50% reduction in spleen length at week 24 and 48 (reduction) and participants who had no increase greater than or equal to 50% (increase). The edge of the spleen shall be determined by palpation, measured in centimeters, using a soft ruler, from the costal margin to the point of greatest splenic protrusion. For subjects with palpable spleen at baseline and non-palpable at post-baseline, the post-baseline spleen is imputed as 0.
Outcome measures
| Measure |
All Subjects
n=51 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 ≤ -50% reduction n=43
|
65.1 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 -50%, -25% reduction n=43
|
11.6 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 -25%, -5% reduction n=43
|
9.3 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 -5%, 5% reduction, increase n=43
|
4.7 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 5%, 25% increase n=43
|
9.3 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 25%, 50% increase n=43
|
0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 >50% increase n=43
|
0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 remained non-palpable n=43
|
0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 24 became palpable n=43
|
0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 ≤ -50% reduction n=36
|
50.0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 -50%, -25% reduction n=36
|
22.2 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 -25%, -5% reduction n=36
|
16.7 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 -5%, 5% reduction, increase n=36
|
5.6 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 5%, 25% increase n=36
|
2.8 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 25%, 50% increase n=36
|
2.8 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 >50% increase n=36
|
0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 remained non-palpable n=36
|
0 percentage of participants
|
|
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48
Wk 48 became palpable n=36
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline and week 24The MF-7 is a disease specific questionnaire comprised of 7 items that measures the severity of seven of the most prevalent associated symptoms including: tiredness, early satiety, abdominal discomfort, night sweats, itching (pruritus), bone pain (diffuse not joint or arthritis) and pain under ribs on left side. Each item was scored on a scale ranging from 0 (absent) to 10 (worst imaginable). The MF-7 score is computed as the sum of the observed scores in the individual items to achieve a 0 to 70 score. There would be one recall period of 24 hours used in this questionnaire. A separate question on Inactivity was to be measured for severity of this symptom on a scale from 0 (absent) to 10 (worst imaginable). This would allow the computation of the MFSAF v2.0 questionnaire results, as 6 out of 7 items in the latter PRO are in overlap with MF7 (they also share same 0-10 range Likert scale and ascending order, absent to worst imaginable).
Outcome measures
| Measure |
All Subjects
n=45 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Percentage of Participants With at Least a 50% Reduction in Myelofibrosis 7 Item Symptom Scale (MF-7) and Myelofibrosis Symptom Assessment Form (MFSAF) at Week 24
MF-7 Total Symptom score
|
51.1 percentage of participants
Interval 35.8 to 66.3
|
|
Percentage of Participants With at Least a 50% Reduction in Myelofibrosis 7 Item Symptom Scale (MF-7) and Myelofibrosis Symptom Assessment Form (MFSAF) at Week 24
Modified MFSAF v2.0 Total symptom score
|
55.6 percentage of participants
Interval 40.0 to 70.4
|
SECONDARY outcome
Timeframe: Baseline up to week 48Population: Participants with PGIC response at that time point
The PGIC is comprised of a single question intended to measure a subject's perspective of improvement or deterioration over time relative to treatment. The PGIC uses a seven-point scale where '1' equals very much improved and '7' equals very much worse.
Outcome measures
| Measure |
All Subjects
n=51 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 Very much improved
|
5 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 Much improved
|
20 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 Minimally improved
|
9 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 No change
|
6 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 Minimally worse
|
1 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 Much worse
|
0 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 24 n=41 Very much worse
|
0 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 Very much improved
|
5 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 Much improved
|
15 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 Minimally improved
|
9 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 No change
|
4 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 Minimally worse
|
0 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 Much worse
|
0 Participants
|
|
Patient Global Impression of Change (PGIC) at Week 24 and Week 48
Week 48 n=33 Very much worse
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline up to week 96Population: Number of participants that were transfusion dependent at baseline
Percentage is based on number of subjects who are transfusion dependent at baseline. Transfusion dependence (TD) is defined as subjects receiving 6 or more units of transfusions 12 weeks prior to baseline. Transfusion independence (TI) rate is defined as subjects who are transfusion dependent at baseline and require no unit of transfusion for ≥ 12 weeks at any time during the study. Transfusion response rate is defined as subjects who are TD at baseline and have 5 or less units of transfusion for ≥ 12 weeks at any time during the study.
Outcome measures
| Measure |
All Subjects
n=9 Participants
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 24 transfusion independent rate
|
0 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 24 transfusion responder rate
|
44.4 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 48 transfusion independent rate
|
0 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 48 transfusion responder rate
|
66.7 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 72 transfusion independent rate
|
0 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 72 transfusion responder rate
|
66.7 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 96 transfusion independent rate
|
0 percentage of participants
|
|
Percentage of Participants Transfusion Independency From Baseline up to Week 96
Week 96 transfusion responder rate
|
66.7 percentage of participants
|
Adverse Events
All Patients
Serious events: 17 serious events
Other events: 40 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
All Patients
n=51 participants at risk
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Blood and lymphatic system disorders
Haemolytic anaemia
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Blood and lymphatic system disorders
Pancytopenia
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Cardiac disorders
Aortic valve disease
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Cardiac disorders
Atrial fibrillation
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Cardiac disorders
Cardiac failure
|
3.9%
2/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Cardiac disorders
Cardiac failure acute
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Cardiac disorders
Myocardial ischaemia
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Crohn's disease
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Hepatobiliary disorders
Bile duct stone
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Bronchitis
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Escherichia sepsis
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Escherichia urinary tract infection
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Gastroenteritis
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Pneumonia
|
3.9%
2/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Respiratory syncytial virus bronchitis
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Sepsis
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Septic shock
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Staphylococcal bacteraemia
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Nervous system disorders
Hepatic encephalopathy
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Nephrolithiasis
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Renal failure
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
2.0%
1/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
Other adverse events
| Measure |
All Patients
n=51 participants at risk
10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
33.3%
17/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
27.5%
14/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Constipation
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Diarrhoea
|
11.8%
6/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Asthenia
|
11.8%
6/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Fatigue
|
9.8%
5/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Oedema peripheral
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Pyrexia
|
7.8%
4/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Bronchitis
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Oral herpes
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Urinary tract infection
|
9.8%
5/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Alanine aminotransferase increased
|
9.8%
5/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Aspartate aminotransferase increased
|
9.8%
5/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Blood alkaline phosphatase increased
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Blood creatinine increased
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Gamma-glutamyltransferase increased
|
11.8%
6/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Platelet count decreased
|
7.8%
4/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
7.8%
4/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Acute kidney injury
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
7.8%
4/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.9%
3/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
7.8%
4/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Vascular disorders
Haematoma
|
7.8%
4/51 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 100 weeks
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER