Phase 3
N=51
A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis
Primary Myelofibrosis (MF)
Bottom Line
View on ClinicalTrials.gov: NCT02087059 ↗Enrolled (actual)
51
Serious AEs
23.5%
Results posted
Jul 2016
Primary outcome: Primary: Number of Participants With Adverse Events as a Measure of Safety and Tolerability — 50; 46; 12; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Ruxolitinib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Mar 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events as a Measure of Safety and Tolerability |
50; 46; 12; 5; 36; 33 | — |
| SECONDARY Charge in Spleen Size From Baseline at Specified Week |
12; 14; 14; 11; 14; 13 | — |
| SECONDARY Charge in Spleen Size From Baseline up to the Specified Week |
18; 22; 23; 26; 26; 26 | — |
| SECONDARY Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time |
2.9; 0.6; 1.8; 0.6; 4.4; 1.4 | — |
| SECONDARY Summary of Summary of EORTC QLQ-C30 Responses by Time |
79.35; 86.67; 77.78; 83.72; 85.46; 92.05 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- ≥18 years of age
- Diagnosis of PMF, PPV-MF, or PET-MF, regardless of JAK2 mutational status. The diagnostic of PMF will be according to the World Health Organization (WHO) criteria (Thiele et al., 2008) and PPV-MF and PET-MF according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria (Barosi et al., 2008).
- At least one risk factors provided in the definition of IWG-MRT (Cervantes et al., 2009; classified as intermediate risk-1, intermediate risk-2, or high risk)
- Patients with intermediate risk-1 (patients who have only one of the IMG-MRT risk factors indicated above ) must have palpable splenomegaly with a length of ≥5 cm from the costal margin to the point of the greatest spleen protrusion.
- Proportion of blasts in peripheral blood 2.5-fold ULN
- Creatinine >2.0 mg/dL
- Clinically significant infection by bacteria, fungus, mycobacteria, parasite, or virus (screening and enrollment postponed until completion of antibiotic treatment in patients with an acute bacterial infection that requires antibiotic use)
- Active hepatitis A, B, or C or HIV infection defined by a positive IgM-HA Ab test [hepatitis A virus antibody (immunoglobulin M [IgM])], HBs Ag test (hepatitis B surface antigen), HCV Ab test (hepatitis C virus antibody), or HIV Ab (human immunodeficiency virus antibody) at screening.
- History of malignancy within the previous 3 years, except for early-stage squamous cell carcinoma and basal cell carcinoma.
- History of serious congenital or acquired hemorrhagic disease
- Previous platelet count <25, 000/μL or absolute neutrophil count <500/μL, except for patients currently undergoing treatment for a myeloproliferative neoplasm or cytotoxic therapy for any other reason.
- Splenic irradiation within 12 months before screening
- Administration of hematopoietic growth factor receptor agonists (erythropoietin, granulocyte colony stimulating factor, romiplostim, eltrombopag) within 14 days before screening or 28 days before treatment initiation.
- Currently receiving another investigational drug, or received another investigational drug within 30 days before the start of treatment.
- History of myocardial infarction or acute coronary syndrome within 6 months before screening
- Poorly controlled or unstable angina at present
- Rapid or paroxysmal atrial fibrillation at present
- Active alcohol or drug addiction that could hinder the patient's ability to comply with the study's requirements
- Pregnant or currently breastfeeding woman
- Women of childbearing potential or men with reproductive ability who are unwilling to take appropriate contraception measures
- Patient with any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol
- History of hypersensitivity to the study drug or a drug with a similar chemical structure
Data sourced from ClinicalTrials.gov (NCT02087059). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.