Neoplasms

Blood and Lymph Conditions

All Conditions

Heart and Blood Diseases

Rare Diseases

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Myeloproliferative Disorders

Bone Marrow Diseases

Hematologic Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Essential Thrombocythemia

Chronic Myeloproliferative Disorders

All Drugs and Chemicals

Janus Kinase Inhibitors

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.

Ruxolitinib

Novartis Pharmaceuticals

This is an open-label, multicenter clinical study in order to collect and examine data concerning the safety and efficacy of ruxolitinib in patients with Primary Myelofibrosis (MF), Post-Polycythemia Vera (PV) MF, Post-Essential Thrombocythemia (ET) MF.

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

A Multicenter, Open-label Clinical Study of the JAK Inhibitor Ruxolitinib (INC424) in Patients With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis

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

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

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

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.

Age, Continuous

Sex: Female, Male

Clinical Disclosure Office

AEs, regardless of study treatment relationship

AEs suspected to be related to study treatment

SAEs, regardless of study treatment relationship

SAEs suspected to be related to study treatment

AEs of CTC grade 3/4, regardless study treatment

AEs of CTC grade 3/4 suspected related treatment

AEs leading to the study treatment discontinuation

AEs requiring reduction or interruption treatment

AEs requiring concomitant medication

Number of Participants With Adverse Events as a Measure of Safety and Tolerability

Week 2

Week 4

Week 8

Week 12

Week 16

Week 20

Week 24

Charge in Spleen Size From Baseline at Specified Week

Charge in Spleen Size From Baseline up to the Specified Week

Night Sweats baseline (n=51)

Night Sweats week 24 (n=43)

Itching baseline (n=51)

Itching week 24 (n=43

Abdominal Discomfort baseline( n=51)

Abdominal Discomfort week 24(n=43)

Pain under ribs on left Baseline (=51)

Pain under ribs on left week 24 (=43)

Feeling of fullness Baseline (n=51)

Feeling of fullness week 24 (n=43)

Bone/muscle pain Baseline (n=51)

Bone/muscle pain week 24 (n=43)

Degree of inactivity Baseline (n=51)

Degree of inactivity week 24 (n=43)

Total symptom score baseline (n=51)

Total symptom score week 24 (n=43)

Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time

Physical Functioning baseline

Physical Functioning 24(n=43)

Role Functioning Baseline(n=51)

Role Functioning Week 24(n=43)

Emotional Functioning Baseline(n=51)

Emotional Functioning Week 24(n=43)

Cognitive Functioning Baseline(n=51)

Cognitive Functioning week 24 (n=43)

Social Functioning Baseline (n=51)

Social Functioning week 24(n=43)

Global Health Status/QOL Baseline (n=51)

Global Health Status/QOL week 24 (n=43)

Fatigue Baseline(n=51)

Fatigue week 24 (n=43)

Nausea and Vomiting baseline (n=51)

Nausea and Vomiting week 24 (n=43)

Pain Baseline

Pain week 24 (n=43)

Dyspnoea baseline (n=51)

Dyspnoea Week 24 (n=43)

Insomnia baseline (n=51)

Insomnia Week 24 (n=43)

Appetite Lose baseline (n=51)

Appetite Lose Week 24 (n=43)

Constipation baseline (n=51)

Constipation Week 24 (n=43)

Diarrhoea baseline (n=51)

Diarrhoea Week 24 (n=43)

Financial Difficulties baseline (n=51)

Financial Difficulties Week 24 (n=43)

Summary of Summary of EORTC QLQ-C30 Responses by Time

Overall Study

Ruxolitinib was taken twice daily, unless instructed. Starting dose 15 mg BID for patients with baseline platelet count of 100,000/μL to 200,000/μL (inclusive) or 20 mg BID for those with baseline platelet count \>200,000/μL (approximately 12 hours apart: morning and night), increased or decreased per standardized dosing paradigm.

INC424 Tablet for oral use, provided in 5 mg bottles. The dosage strength was 5 mg/tablet INC424 phosphate (free base equivalent).

The objective of this study was to determine the efficacy of INC424 as assessed by reduction in spleen volume in patients with primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF. The safety and tolerability of INC424 and the effects of INC424 on patient reported outcomes and the duration of response as assessed by reduction in spleen volume was also assessed.

Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF

A Multi-national Open-label Phase II Study of the JAK Inhibitor INC424 in Patients With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

The primary measure of spleen size was by MRI. MRIs were performed with a body coil because the objective was to measure organ volume only, not to assess for lesions. MRIs were performed by local radiologists who were instructed not to provide a quantitative measure of spleen volume, but could provide a qualitative assessment such as enlarged, smaller, larger, etc. The scans from an individual patient were to be read by a central reader upon transfer from the site radiologist.

The best response rate was defined as the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline at any post-baseline assessment. The best response rate was estimated with an associated 95% confidence interval.

For patients who had at least one ≥ 35% reduction in spleen volume from baseline at postbaseline, the duration of response was calculated. The start date of the duration was defined as the first spleen volume measurement that was ≥ 35% reduction from baseline, and the end date of the response duration was defined as the earliest of the following: death, A ≥ 25% increase in spleen volume by MRI (or CT in applicable patients) compared to baseline, Splenic irradiation, Leukemic transformation as defined by a bone marrow or a peripheral blood blast count of ≥ 20%, Splenectomy. Duration of response is calculated only for participants who achieved at least one measured \>= 35% reduction in spleen volume at any time.

Patient reported outcomes regarding the impact of MF on patients were assessed using the EORTC QLQ-C30. Data from the EORTC QLQ-C30 questionnaire was analyzed using the standardized scores. There were 2 categories to this scale: Functional/QOL scale and Symptom and Other items scale. For each sub-scale, the raw scores were standardized in order to obtain scores ranging from 0 to 100. For Functional/QOL subscales: a higher score represents a higher/better level of functioning. For Symptoms and Other items subscales: a higher score represents worse level of symptoms. The absolute change from baseline was calculated for each scale and summarized descriptively by scheduled visit.

The Seven-day modified MFSAF v2.0 is a 7-item PRO instrument based on the modified MFSAF v2.0 diary administered at specified visits. Symptoms of myelofibrosis (MF) were assessed using this instrument \& included filling up quickly/early satiety, abdominal discomfort, pain under the ribs, night sweats, itching, bone/muscle pain \& inactivity. The first 6 items assessed MF symptom severity at its worst as recalled \& the seventh captured MF-related inactivity in the 7 days prior to the clinic visit assessment. All 7 items asked subjects to record their answers on an 11-point numeric rating scale (NRS) (0=Absent, 10=Worst Imaginable). The first 6 items of the instrument focus on MF symptoms \& are summed to create a Total Symptom score, defined as the sum of the 6 individual symptom scores other than the inactivity score (each with 0-10 point scale) collected on the same week. The total symptom scale ranges from 0 -60 where higher score indicates a worse level of the condition.

Japanese

Korean

Chinese

Taiwanese

Race/Ethnicity, Customized

Full Analysis Set (FAS): comprised of all patients who received at least one dose of ruxolitinib.

Study Director

Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24

Week 36

Week 48

Week 72

Week 96

Week 120

Week 144

Week 168

Week 192

Week 216

Week 240

at any time point

Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Each Scheduled Time Point - Best Response

12 weeks

24 weeks

36 weeks

48 weeks

60 weeks

72 weeks

84 weeks

96 weeks

120 weeks

144 weeks

168 weeks

192 weeks

216 weeks

240 weeks

Kaplan Meier Estimates of Duration of Response of at Least ≥ 35% Reduction From Baseline in Spleen Volume Per Kaplan Meier Estimates

Func./QOL scales: Global Health Status/QOL

Func./QOL scales: Physical Functioning

Func./QOL scales:Role Functioning

Func./QOL scales: Emotional Functioning

Func./QOL scales: Cognitive Functioning

Func./QOL scales: Social Functioning

Symptom and Other items: Fatigue

Symptom & Other items: Nausea and Vomiting

Symptom and Other items: Pain

Symptom and Other items: Dyspnea

Symptom and Other items: Insomnia

Symptom and Other items: Appetite Loss

Symptom and Other items: Constipation

Symptom and Other items: Diarrhea

Symptom & Other items: Fin. difficulties

Change in EORTC QLQ-C30 Scores From Baseline in at Week 24

Change in Total Symptom Score From Baseline at Week 24 as Measured by Seven-day Modified MFSAF v2.0

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis	NCT02087059	Primary Myelofi...	Ruxolitinib	18 Years -	Novartis
Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF	NCT01392443	Primary Myelofi... Post-Polycythem... Post-Essential ...	Ruxolitinib	18 Years - 65 Years	Novartis

Spots Global Cancer Trial Database for primary myelofibrosis (mf)