Trial Outcomes & Findings for A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis (NCT NCT02087059)

NCT ID: NCT02087059

Last Updated: 2016-07-11

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 51 participants
• Primary outcome timeframe: 24 weeks
• Results posted on: 2016-07-11

Participant Flow

Participant milestones

Measure	Ruxolitinib 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 Study STARTED	51
Overall Study COMPLETED	44
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Measure	Ruxolitinib 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 Study Disease Progression	1
Overall Study Adverse Event	5
Overall Study Lost to Follow-up	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Ruxolitinib n=51 Participants 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.
Age, Continuous	65.7 years STANDARD_DEVIATION 8.8 • n=5 Participants
Sex: Female, Male Female	24 Participants n=5 Participants
Sex: Female, Male Male	27 Participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Outcome measures

Measure	Ruxolitinib n=51 Participants 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.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs requiring reduction or interruption treatment	38 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs requiring concomitant medication	33 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs, regardless of study treatment relationship	50 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs suspected to be related to study treatment	46 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability SAEs, regardless of study treatment relationship	12 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability SAEs suspected to be related to study treatment	5 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs of CTC grade 3/4, regardless study treatment	36 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs of CTC grade 3/4 suspected related treatment	33 Participants
Number of Participants With Adverse Events as a Measure of Safety and Tolerability AEs leading to the study treatment discontinuation	5 Participants

SECONDARY outcome

Timeframe: Baseline, 24 weeks

Number of patients with spleen length reduced by ≥ 50% at specified week

Outcome measures

Measure	Ruxolitinib n=51 Participants 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.
Charge in Spleen Size From Baseline at Specified Week Week 2	12 particiapants
Charge in Spleen Size From Baseline at Specified Week Week 4	14 particiapants
Charge in Spleen Size From Baseline at Specified Week Week 8	14 particiapants
Charge in Spleen Size From Baseline at Specified Week Week 16	14 particiapants
Charge in Spleen Size From Baseline at Specified Week Week 20	13 particiapants
Charge in Spleen Size From Baseline at Specified Week Week 24	15 particiapants
Charge in Spleen Size From Baseline at Specified Week Week 12	11 particiapants

SECONDARY outcome

Timeframe: Baseline, 24 weeks

Number of patients with spleen length reduced by ≥ 50% up to specified week

Outcome measures

Measure	Ruxolitinib n=51 Participants 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.
Charge in Spleen Size From Baseline up to the Specified Week Week 4	18 particiapants Interval 13.1 to 38.2
Charge in Spleen Size From Baseline up to the Specified Week Week 8	22 particiapants Interval 16.2 to 42.5
Charge in Spleen Size From Baseline up to the Specified Week Week 12	23 particiapants Interval 16.2 to 42.5
Charge in Spleen Size From Baseline up to the Specified Week Week 16	26 particiapants
Charge in Spleen Size From Baseline up to the Specified Week Week 20	26 particiapants
Charge in Spleen Size From Baseline up to the Specified Week Week 24	26 particiapants

SECONDARY outcome

Timeframe: 24 weeks

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

Outcome measures

Measure	Ruxolitinib n=51 Participants 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.
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Night Sweats baseline (n=51)	2.9 score on a scale Standard Deviation 3.07
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Night Sweats week 24 (n=43)	0.6 score on a scale Standard Deviation 1.35
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Itching baseline (n=51)	1.8 score on a scale Standard Deviation 2.39
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Itching week 24 (n=43	0.6 score on a scale Standard Deviation 1.25
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Abdominal Discomfort baseline( n=51)	4.4 score on a scale Standard Deviation 3.18
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Abdominal Discomfort week 24(n=43)	1.4 score on a scale Standard Deviation 1.84
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Pain under ribs on left Baseline (=51)	2.2 score on a scale Standard Deviation 2.72
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Pain under ribs on left week 24 (=43)	0.7 score on a scale Standard Deviation 1.26
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Total symptom score week 24 (n=43)	5.7 score on a scale Standard Deviation 6.48
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Feeling of fullness Baseline (n=51)	3.2 score on a scale Standard Deviation 2.91
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Feeling of fullness week 24 (n=43)	1.3 score on a scale Standard Deviation 1.8
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Bone/muscle pain Baseline (n=51)	2.3 score on a scale Standard Deviation 2.83
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Bone/muscle pain week 24 (n=43)	1.0 score on a scale Standard Deviation 1.83
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Degree of inactivity Baseline (n=51)	2.3 score on a scale Standard Deviation 2.84
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Degree of inactivity week 24 (n=43)	1.0 score on a scale Standard Deviation 1.66
Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time Total symptom score baseline (n=51)	16.8 score on a scale Standard Deviation 12.30

SECONDARY outcome

Timeframe: 24 weeks

Population: FAS

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.

Outcome measures

Measure	Ruxolitinib n=51 Participants 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.
Summary of Summary of EORTC QLQ-C30 Responses by Time Physical Functioning baseline	79.35 units on a scale Standard Deviation 17.951
Summary of Summary of EORTC QLQ-C30 Responses by Time Physical Functioning 24(n=43)	86.67 units on a scale Standard Deviation 17.959
Summary of Summary of EORTC QLQ-C30 Responses by Time Role Functioning Baseline(n=51)	77.78 units on a scale Standard Deviation 23.254
Summary of Summary of EORTC QLQ-C30 Responses by Time Role Functioning Week 24(n=43)	83.72 units on a scale Standard Deviation 19.068
Summary of Summary of EORTC QLQ-C30 Responses by Time Emotional Functioning Baseline(n=51)	85.46 units on a scale Standard Deviation 17.705
Summary of Summary of EORTC QLQ-C30 Responses by Time Emotional Functioning Week 24(n=43)	92.05 units on a scale Standard Deviation 13.357
Summary of Summary of EORTC QLQ-C30 Responses by Time Cognitive Functioning Baseline(n=51)	78.43 units on a scale Standard Deviation 20.356
Summary of Summary of EORTC QLQ-C30 Responses by Time Cognitive Functioning week 24 (n=43)	79.84 units on a scale Standard Deviation 17.653
Summary of Summary of EORTC QLQ-C30 Responses by Time Social Functioning Baseline (n=51)	85.62 units on a scale Standard Deviation 19.154
Summary of Summary of EORTC QLQ-C30 Responses by Time Social Functioning week 24(n=43)	87.60 units on a scale Standard Deviation 17.852
Summary of Summary of EORTC QLQ-C30 Responses by Time Global Health Status/QOL Baseline (n=51)	55.72 units on a scale Standard Deviation 22.882
Summary of Summary of EORTC QLQ-C30 Responses by Time Global Health Status/QOL week 24 (n=43)	66.47 units on a scale Standard Deviation 22.456
Summary of Summary of EORTC QLQ-C30 Responses by Time Fatigue Baseline(n=51)	40.31 units on a scale Standard Deviation 23.787
Summary of Summary of EORTC QLQ-C30 Responses by Time Fatigue week 24 (n=43)	28.68 units on a scale Standard Deviation 23.412
Summary of Summary of EORTC QLQ-C30 Responses by Time Nausea and Vomiting baseline (n=51)	4.90 units on a scale Standard Deviation 13.862
Summary of Summary of EORTC QLQ-C30 Responses by Time Nausea and Vomiting week 24 (n=43)	0.39 units on a scale Standard Deviation 2.542
Summary of Summary of EORTC QLQ-C30 Responses by Time Pain Baseline	21.57 units on a scale Standard Deviation 21.678
Summary of Summary of EORTC QLQ-C30 Responses by Time Pain week 24 (n=43)	10.08 units on a scale Standard Deviation 15.911
Summary of Summary of EORTC QLQ-C30 Responses by Time Dyspnoea baseline (n=51)	22.88 units on a scale Standard Deviation 25.377
Summary of Summary of EORTC QLQ-C30 Responses by Time Dyspnoea Week 24 (n=43)	25.58 units on a scale Standard Deviation 23.946
Summary of Summary of EORTC QLQ-C30 Responses by Time Insomnia baseline (n=51)	23.53 units on a scale Standard Deviation 29.283
Summary of Summary of EORTC QLQ-C30 Responses by Time Insomnia Week 24 (n=43)	15.50 units on a scale Standard Deviation 23.400
Summary of Summary of EORTC QLQ-C30 Responses by Time Appetite Lose baseline (n=51)	21.57 units on a scale Standard Deviation 27.787
Summary of Summary of EORTC QLQ-C30 Responses by Time Appetite Lose Week 24 (n=43)	9.30 units on a scale Standard Deviation 16.786
Summary of Summary of EORTC QLQ-C30 Responses by Time Constipation baseline (n=51)	8.50 units on a scale Standard Deviation 20.916
Summary of Summary of EORTC QLQ-C30 Responses by Time Constipation Week 24 (n=43)	8.53 units on a scale Standard Deviation 16.416
Summary of Summary of EORTC QLQ-C30 Responses by Time Diarrhoea baseline (n=51)	18.30 units on a scale Standard Deviation 24.325
Summary of Summary of EORTC QLQ-C30 Responses by Time Diarrhoea Week 24 (n=43)	12.40 units on a scale Standard Deviation 23.029
Summary of Summary of EORTC QLQ-C30 Responses by Time Financial Difficulties baseline (n=51)	15.69 units on a scale Standard Deviation 25.257
Summary of Summary of EORTC QLQ-C30 Responses by Time Financial Difficulties Week 24 (n=43)	20.93 units on a scale Standard Deviation 30.012

Adverse Events

Ruxolitinib

Serious events: 12 serious events

Other events: 49 other events

Deaths: 0 deaths

Serious adverse events

Measure	Ruxolitinib n=51 participants at risk 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.
Blood and lymphatic system disorders Anaemia	2.0% 1/51
Blood and lymphatic system disorders Disseminated intravascular coagulation	2.0% 1/51
Cardiac disorders Cardiac failure congestive	2.0% 1/51
Gastrointestinal disorders Gastrointestinal haemorrhage	2.0% 1/51
Gastrointestinal disorders Oesophageal varices haemorrhage	2.0% 1/51
General disorders Multi-organ failure	2.0% 1/51
Hepatobiliary disorders Drug-induced liver injury	2.0% 1/51
Infections and infestations Empyema	2.0% 1/51
Infections and infestations Sepsis	2.0% 1/51
Infections and infestations Urinary tract infection	2.0% 1/51
Injury, poisoning and procedural complications Spinal compression fracture	2.0% 1/51
Metabolism and nutrition disorders Gout	2.0% 1/51
Metabolism and nutrition disorders Tumour lysis syndrome	2.0% 1/51
Psychiatric disorders Withdrawal syndrome	2.0% 1/51
Respiratory, thoracic and mediastinal disorders Dyspnoea	2.0% 1/51
Respiratory, thoracic and mediastinal disorders Interstitial lung disease	2.0% 1/51
Respiratory, thoracic and mediastinal disorders Pleural effusion	2.0% 1/51
Respiratory, thoracic and mediastinal disorders Pneumonitis	2.0% 1/51

Other adverse events

Measure	Ruxolitinib n=51 participants at risk 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.
Blood and lymphatic system disorders Anaemia	62.7% 32/51
Blood and lymphatic system disorders Leukopenia	5.9% 3/51
Blood and lymphatic system disorders Lymphopenia	5.9% 3/51
Blood and lymphatic system disorders Thrombocytopenia	29.4% 15/51
Gastrointestinal disorders Abdominal pain	7.8% 4/51
Gastrointestinal disorders Constipation	13.7% 7/51
Gastrointestinal disorders Diarrhoea	7.8% 4/51
Gastrointestinal disorders Nausea	5.9% 3/51
Gastrointestinal disorders Vomiting	5.9% 3/51
General disorders Oedema peripheral	5.9% 3/51
General disorders Pyrexia	9.8% 5/51
Hepatobiliary disorders Hepatic function abnormal	11.8% 6/51
Infections and infestations Nasopharyngitis	11.8% 6/51
Infections and infestations Pneumonia	7.8% 4/51
Investigations Electrocardiogram QT prolonged	9.8% 5/51
Investigations Lymphocyte count decreased	7.8% 4/51
Investigations Neutrophil count decreased	5.9% 3/51
Investigations Platelet count decreased	23.5% 12/51
Investigations Weight increased	5.9% 3/51
Musculoskeletal and connective tissue disorders Back pain	7.8% 4/51
Nervous system disorders Headache	5.9% 3/51
Psychiatric disorders Insomnia	5.9% 3/51

Additional Information

Clinical Disclosure Office

Novartis Pharmaceuticals

Phone: 862-778-8300

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