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A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis

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ClinicalTrials.gov Identifier: NCT02087059
Recruitment Status : Completed
First Posted : March 14, 2014
Results First Posted : July 11, 2016
Last Update Posted : July 11, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Primary Myelofibrosis (MF)
Intervention Drug: Ruxolitinib
Enrollment 51
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ruxolitinib
Hide Arm/Group Description Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Period Title: Overall Study
Started 51
Completed 44
Not Completed 7
Reason Not Completed
Disease Progression             1
Adverse Event             5
Lost to Follow-up             1
Arm/Group Title Ruxolitinib
Hide Arm/Group Description Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Overall Number of Baseline Participants 51
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 51 participants
65.7  (8.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 51 participants
Female
24
  47.1%
Male
27
  52.9%
1.Primary Outcome
Title Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Hide Description [Not Specified]
Time Frame 24 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Overall Number of Participants Analyzed 51
Measure Type: Number
Unit of Measure: Participants
AEs, regardless of study treatment relationship 50
AEs suspected to be related to study treatment 46
SAEs, regardless of study treatment relationship 12
SAEs suspected to be related to study treatment 5
AEs of CTC grade 3/4, regardless study treatment 36
AEs of CTC grade 3/4 suspected related treatment 33
AEs leading to the study treatment discontinuation 5
AEs requiring reduction or interruption treatment 38
AEs requiring concomitant medication 33
2.Secondary Outcome
Title Charge in Spleen Size From Baseline at Specified Week
Hide Description Number of patients with spleen length reduced by ≥ 50% at specified week
Time Frame Baseline, 24 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Overall Number of Participants Analyzed 51
Measure Type: Number
Unit of Measure: particiapants
Week 2 12
Week 4 14
Week 8 14
Week 12 11
Week 16 14
Week 20 13
Week 24 15
3.Secondary Outcome
Title Charge in Spleen Size From Baseline up to the Specified Week
Hide Description Number of patients with spleen length reduced by ≥ 50% up to specified week
Time Frame Baseline, 24 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Overall Number of Participants Analyzed 51
Measure Type: Number
Unit of Measure: particiapants
Week 4 18
Week 8 22
Week 12 23
Week 16 26
Week 20 26
Week 24 26
4.Secondary Outcome
Title Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time
Hide Description The modified MFSAF v2.0 diary captures a patient's symptom severity on a scale of 0 (absent) to 10 (worst imaginable),with a maximal summary score of 60
Time Frame 24 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Overall Number of Participants Analyzed 51
Mean (Standard Deviation)
Unit of Measure: score on a scale
Night Sweats baseline (n=51) 2.9  (3.07)
Night Sweats week 24 (n=43) 0.6  (1.35)
Itching baseline (n=51) 1.8  (2.39)
Itching week 24 (n=43 0.6  (1.25)
Abdominal Discomfort baseline( n=51) 4.4  (3.18)
Abdominal Discomfort week 24(n=43) 1.4  (1.84)
Pain under ribs on left Baseline (=51) 2.2  (2.72)
Pain under ribs on left week 24 (=43) 0.7  (1.26)
Feeling of fullness Baseline (n=51) 3.2  (2.91)
Feeling of fullness week 24 (n=43) 1.3  (1.8)
Bone/muscle pain Baseline (n=51) 2.3  (2.83)
Bone/muscle pain week 24 (n=43) 1.0  (1.83)
Degree of inactivity Baseline (n=51) 2.3  (2.84)
Degree of inactivity week 24 (n=43) 1.0  (1.66)
Total symptom score baseline (n=51) 16.8  (12.30)
Total symptom score week 24 (n=43) 5.7  (6.48)
5.Secondary Outcome
Title Summary of Summary of EORTC QLQ-C30 Responses by Time
Hide Description The QLQ-C30 version 1.0 (QLQ-C30) incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease.All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems.
Time Frame 24 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
Overall Number of Participants Analyzed 51
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical Functioning baseline 79.35  (17.951)
Physical Functioning 24(n=43) 86.67  (17.959)
Role Functioning Baseline(n=51) 77.78  (23.254)
Role Functioning Week 24(n=43) 83.72  (19.068)
Emotional Functioning Baseline(n=51) 85.46  (17.705)
Emotional Functioning Week 24(n=43) 92.05  (13.357)
Cognitive Functioning Baseline(n=51) 78.43  (20.356)
Cognitive Functioning week 24 (n=43) 79.84  (17.653)
Social Functioning Baseline (n=51) 85.62  (19.154)
Social Functioning week 24(n=43) 87.60  (17.852)
Global Health Status/QOL Baseline (n=51) 55.72  (22.882)
Global Health Status/QOL week 24 (n=43) 66.47  (22.456)
Fatigue Baseline(n=51) 40.31  (23.787)
Fatigue week 24 (n=43) 28.68  (23.412)
Nausea and Vomiting baseline (n=51) 4.90  (13.862)
Nausea and Vomiting week 24 (n=43) 0.39  (2.542)
Pain Baseline 21.57  (21.678)
Pain week 24 (n=43) 10.08  (15.911)
Dyspnoea baseline (n=51) 22.88  (25.377)
Dyspnoea Week 24 (n=43) 25.58  (23.946)
Insomnia baseline (n=51) 23.53  (29.283)
Insomnia Week 24 (n=43) 15.50  (23.400)
Appetite Lose baseline (n=51) 21.57  (27.787)
Appetite Lose Week 24 (n=43) 9.30  (16.786)
Constipation baseline (n=51) 8.50  (20.916)
Constipation Week 24 (n=43) 8.53  (16.416)
Diarrhoea baseline (n=51) 18.30  (24.325)
Diarrhoea Week 24 (n=43) 12.40  (23.029)
Financial Difficulties baseline (n=51) 15.69  (25.257)
Financial Difficulties Week 24 (n=43) 20.93  (30.012)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ruxolitinib
Hide Arm/Group Description Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.
All-Cause Mortality
Ruxolitinib
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Ruxolitinib
Affected / at Risk (%)
Total   12/51 (23.53%) 
Blood and lymphatic system disorders   
Anaemia  1  1/51 (1.96%) 
Disseminated intravascular coagulation  1  1/51 (1.96%) 
Cardiac disorders   
Cardiac failure congestive  1  1/51 (1.96%) 
Gastrointestinal disorders   
Gastrointestinal haemorrhage  1  1/51 (1.96%) 
Oesophageal varices haemorrhage  1  1/51 (1.96%) 
General disorders   
Multi-organ failure  1  1/51 (1.96%) 
Hepatobiliary disorders   
Drug-induced liver injury  1  1/51 (1.96%) 
Infections and infestations   
Empyema  1  1/51 (1.96%) 
Sepsis  1  1/51 (1.96%) 
Urinary tract infection  1  1/51 (1.96%) 
Injury, poisoning and procedural complications   
Spinal compression fracture  1  1/51 (1.96%) 
Metabolism and nutrition disorders   
Gout  1  1/51 (1.96%) 
Tumour lysis syndrome  1  1/51 (1.96%) 
Psychiatric disorders   
Withdrawal syndrome  1  1/51 (1.96%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnoea  1  1/51 (1.96%) 
Interstitial lung disease  1  1/51 (1.96%) 
Pleural effusion  1  1/51 (1.96%) 
Pneumonitis  1  1/51 (1.96%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ruxolitinib
Affected / at Risk (%)
Total   49/51 (96.08%) 
Blood and lymphatic system disorders   
Anaemia  1  32/51 (62.75%) 
Leukopenia  1  3/51 (5.88%) 
Lymphopenia  1  3/51 (5.88%) 
Thrombocytopenia  1  15/51 (29.41%) 
Gastrointestinal disorders   
Abdominal pain  1  4/51 (7.84%) 
Constipation  1  7/51 (13.73%) 
Diarrhoea  1  4/51 (7.84%) 
Nausea  1  3/51 (5.88%) 
Vomiting  1  3/51 (5.88%) 
General disorders   
Oedema peripheral  1  3/51 (5.88%) 
Pyrexia  1  5/51 (9.80%) 
Hepatobiliary disorders   
Hepatic function abnormal  1  6/51 (11.76%) 
Infections and infestations   
Nasopharyngitis  1  6/51 (11.76%) 
Pneumonia  1  4/51 (7.84%) 
Investigations   
Electrocardiogram QT prolonged  1  5/51 (9.80%) 
Lymphocyte count decreased  1  4/51 (7.84%) 
Neutrophil count decreased  1  3/51 (5.88%) 
Platelet count decreased  1  12/51 (23.53%) 
Weight increased  1  3/51 (5.88%) 
Musculoskeletal and connective tissue disorders   
Back pain  1  4/51 (7.84%) 
Nervous system disorders   
Headache  1  3/51 (5.88%) 
Psychiatric disorders   
Insomnia  1  3/51 (5.88%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

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.

Results Point of Contact
Name/Title: Clinical Disclosure Office
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02087059     History of Changes
Other Study ID Numbers: CINC424AJP01
First Submitted: March 12, 2014
First Posted: March 14, 2014
Results First Submitted: April 11, 2016
Results First Posted: July 11, 2016
Last Update Posted: July 11, 2016