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NCT02966353: REALISE

Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients.

Completed Phase 2 Results posted Last updated 30 April 2020
What this trial tests

Phase 2 trial testing ruxolitinib in Primary Myelofibrosis in 51 participants. Completed in 15 February 2019.

Timeline
31 March 2017
Primary endpoint
24 July 2018
15 February 2019

Quick facts

Lead sponsorNovartis Pharmaceuticals
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment51
Start date31 March 2017
Primary completion24 July 2018
Estimated completion15 February 2019
Sites20 locations across Italy, Japan, Russia, Greece, Belgium, Austria, Germany, Canada

Drugs / interventions tested

Conditions studied

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

18 and older, any sex, with Primary Myelofibrosis or Post-Polycythemia Vera-Myelofibrosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants With at Least 50% Reduction in Spleen Length From Baseline at Week 24 Primary · Baseline up to week 24

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.

GroupValue95% CI
All Subjects56.041.3 – 70.0
Percentage of Participants With at Least 50% Reduction in Spleen Length From Baseline at Week 48 Secondary · Baseline up to week 48

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.

GroupValue95% CI
All Subjects36.022.9 – 50.8
Percentage of Participants by Spleen Length Reduction or no Increase From Baseline Category at Week 24 and Week 48 Secondary · baseline, weeks 24 and 48

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.

Wk 24 ≤ -50% reduction n=43
GroupValue95% CI
All Subjects65.1
Wk 24 -50%, -25% reduction n=43
GroupValue95% CI
All Subjects11.6
Wk 24 -25%, -5% reduction n=43
GroupValue95% CI
All Subjects9.3
Wk 24 -5%, 5% reduction, increase n=43
GroupValue95% CI
All Subjects4.7
Wk 24 5%, 25% increase n=43
GroupValue95% CI
All Subjects9.3
Wk 24 25%, 50% increase n=43
GroupValue95% CI
All Subjects0
Wk 24 >50% increase n=43
GroupValue95% CI
All Subjects0
Wk 24 remained non-palpable n=43
GroupValue95% CI
All Subjects0
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 Secondary · Baseline and week 24

The 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 questio

MF-7 Total Symptom score
GroupValue95% CI
All Subjects51.135.8 – 66.3
Modified MFSAF v2.0 Total symptom score
GroupValue95% CI
All Subjects55.640.0 – 70.4
Patient Global Impression of Change (PGIC) at Week 24 and Week 48 Secondary · Baseline up to week 48

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.

Week 24 n=41 Very much improved
GroupValue95% CI
All Subjects5
Week 24 n=41 Much improved
GroupValue95% CI
All Subjects20
Week 24 n=41 Minimally improved
GroupValue95% CI
All Subjects9
Week 24 n=41 No change
GroupValue95% CI
All Subjects6
Week 24 n=41 Minimally worse
GroupValue95% CI
All Subjects1
Week 24 n=41 Much worse
GroupValue95% CI
All Subjects0
Week 24 n=41 Very much worse
GroupValue95% CI
All Subjects0
Week 48 n=33 Very much improved
GroupValue95% CI
All Subjects5
Percentage of Participants Transfusion Independency From Baseline up to Week 96 Secondary · Baseline up to week 96

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.

Week 24 transfusion independent rate
GroupValue95% CI
All Subjects0
Week 24 transfusion responder rate
GroupValue95% CI
All Subjects44.4
Week 48 transfusion independent rate
GroupValue95% CI
All Subjects0
Week 48 transfusion responder rate
GroupValue95% CI
All Subjects66.7
Week 72 transfusion independent rate
GroupValue95% CI
All Subjects0
Week 72 transfusion responder rate
GroupValue95% CI
All Subjects66.7
Week 96 transfusion independent rate
GroupValue95% CI
All Subjects0
Week 96 transfusion responder rate
GroupValue95% CI
All Subjects66.7

Adverse events — posted to ClinicalTrials.gov

Time frame: 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. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

All Patients
Serious: 17/51 (33%)
Deaths: 7/51

Serious adverse events (27 terms)

ReactionSystemAll Patients
AnaemiaBlood and lymphatic system disorders
Cardiac failureCardiac disorders
PneumoniaInfections and infestations
Haemolytic anaemiaBlood and lymphatic system disorders
PancytopeniaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
Aortic valve diseaseCardiac disorders
Atrial fibrillationCardiac disorders
Cardiac failure acuteCardiac disorders
Myocardial ischaemiaCardiac disorders
Abdominal painGastrointestinal disorders
Crohn's diseaseGastrointestinal disorders
Bile duct stoneHepatobiliary disorders
BronchitisInfections and infestations
Escherichia sepsisInfections and infestations
Escherichia urinary tract infectionInfections and infestations
GastroenteritisInfections and infestations
Lower respiratory tract infection bacterialInfections and infestations
Respiratory syncytial virus bronchitisInfections and infestations
SepsisInfections and infestations
Septic shockInfections and infestations
Staphylococcal bacteraemiaInfections and infestations
Acute myeloid leukaemiaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic encephalopathyNervous system disorders
NephrolithiasisRenal and urinary disorders
Other adverse events (27 terms — click to expand)

ReactionSystemAll Patients
AnaemiaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
DiarrhoeaGastrointestinal disorders
AstheniaGeneral disorders
Gamma-glutamyltransferase increasedInvestigations
FatigueGeneral disorders
Urinary tract infectionInfections and infestations
Alanine aminotransferase increasedInvestigations
Aspartate aminotransferase increasedInvestigations
PyrexiaGeneral disorders
Platelet count decreasedInvestigations
Muscle spasmsMusculoskeletal and connective tissue disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
PruritusSkin and subcutaneous tissue disorders
HaematomaVascular disorders
Abdominal painGastrointestinal disorders
Abdominal pain upperGastrointestinal disorders
ConstipationGastrointestinal disorders
Oedema peripheralGeneral disorders
BronchitisInfections and infestations
Oral herpesInfections and infestations
Blood alkaline phosphatase increasedInvestigations
Blood creatinine increasedInvestigations
ArthralgiaMusculoskeletal and connective tissue disorders
Muscular weaknessMusculoskeletal and connective tissue disorders
Acute kidney injuryRenal and urinary disorders
EpistaxisRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Anaemia, Cardiac failure, Pneumonia, Haemolytic anaemia, Pancytopenia, Thrombocytopenia, Aortic valve disease, Atrial fibrillation.

Data from ClinicalTrials.gov NCT02966353 adverse events section.

Sponsor's own description

This was a study of treatment with ruxolitinib in patients who presented with transfusion dependent or independent anemia. Starting dose was 10 mg BID. This dose was maintained for the first 12 weeks of the study and up-titrated thereafter unless the subject met criteria for dose hold or dose reduction

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Targeting fibrosis, mechanisms and cilinical trials.
    Zhao M, Wang L, Wang M, Zhou S, et al · · 2022 · cited 352× · PMID 35773269 · DOI 10.1038/s41392-022-01070-3
  2. Efficacy and safety of a novel dosing strategy for ruxolitinib in the treatment of patients with myelofibrosis and anemia: the REALISE phase 2 study.
    Cervantes F, Ross DM, Radinoff A, Palandri F, et al · · 2021 · cited 45× · PMID 34017073 · DOI 10.1038/s41375-021-01261-x
  3. Efficacy and tolerability of Janus kinase inhibitors in myelofibrosis: a systematic review and network meta-analysis.
    Sureau L, Orvain C, Ianotto JC, Ugo V, et al · · 2021 · cited 26× · PMID 34315858 · DOI 10.1038/s41408-021-00526-z
  4. How I individualize selection of JAK inhibitors for patients with myelofibrosis.
    Masarova L, Chifotides HT. · · 2025 · cited 8× · PMID 39357058 · DOI 10.1182/blood.2023022415
  5. Real World Management of Cytopenias and Infections in Patients With Myelofibrosis Treated With Ruxolitinib.
    Butler LA, Forsyth C, Harrison C, Perkins AC. · · 2025 · PMID 40123795 · DOI 10.1002/jha2.70007

Verify or expand the search:

Other trials of ruxolitinib

Trials testing the same drug.

Other recruiting trials for Primary Myelofibrosis

Currently open trials in the same condition.

Other Novartis Pharmaceuticals trials

Trials by the same sponsor.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02966353.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing