Phase 2
Completed N=45
Alternative Dosing Strategy of Ruxolitinib in Patients With Myelofibrosis
Myelofibrosis · Post-Polycythemia Vera Myelofibrosis · Post-Essential Thrombocythemia Myelofibrosis
Source: ClinicalTrials.gov NCT01445769 ↗
Enrolled (actual)
45
Serious AEs
4.4%
Results posted
Sep 2014
Primary outcomePrimary: Mean Percentage Change From Baseline in Spleen Volume at Week 24 — -14.9 Percentage change
Summary
The purpose of this study was to evaluate the effect of an alternative dosing strategy of ruxolitinib in subjects with primary myelofibrosis (PMF), post-polycythemia vera-myelofibrosis (PPV-MF) and post essential thrombocythemia-myelofibrosis (PET-MF) in order to minimize the development of anemia and thrombocytopenia.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Percentage Change From Baseline in Spleen Volume at Week 24 |
-14.9 | — |
| PRIMARY Median Percent Change From Baseline in Spleen Volume at Week 24 |
-17.3 | — |
| SECONDARY Mean Percentage Change From Baseline in the Total Symptom Score at Week 24 |
-34.3 | — |
| SECONDARY Median Percent Change From Baseline in the Total Symptom Score at Week 24 |
-45.6 | — |
| SECONDARY Percentage of Participants With a ≥ 35% Reduction From Baseline in Spleen Volume at Week 24 |
15.6 | — |
| SECONDARY Percentage of Participants With a ≥ 10% Reduction From Baseline in Spleen Volume at Week 24 |
57.8 | — |
| SECONDARY Percentage of Participants With a ≥ 50% Improvement From Baseline in Total Symptom Score at Week 24 |
40.0 | — |
| SECONDARY Mean Percentage Change From Baseline in Palpable Spleen Length at Week 24 |
-47.6 | — |
| SECONDARY Median Percent Change From Baseline in Palpable Spleen Length at Week 24 |
-39.8 | — |
| SECONDARY Percentage of Participants With a ≥ 50% Improvement From Baseline in Their Transfusion Status or With New Transfusion Independence Status for Those Participants Who Were Transfusion Dependent at Baseline |
20.0 | — |
| SECONDARY Percentage of Participants With Clinically Notable Anemia |
33.3; 27.3; 27.9 | — |
| SECONDARY Mean Percentage Change in Abdominal Symptom Scores at Week 24. |
-33.2 | — |
| SECONDARY Median Percentage Change in Abdominal Symptom Scores at Week 24. |
-50.5 | — |
| SECONDARY Number of Participants With Grade 3 or Grade 4 Adverse Events |
9; 1; 1; 1; 1; 1 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) as confirmed by bone marrow biopsy.
- Must score at least 2 points on the Dynamic International Prognostic Scoring System (DIPSS) scale for prognostic risk factors.
- Peripheral blast count < 5% at both Screening and Baseline hematology assessments.
- Must discontinue all drugs used to treat underlying myelofibrosis (MF) disease no later than Day -1 (the day prior to starting ruxolitinib).
- Must have hemoglobin value ≥ 6.5 g/dL and be willing to receive blood transfusions.
- Platelet count ≥ 100*10^9/L.
- Must have a palpable spleen.
Exclusion Criteria
- Inadequate liver or bone marrow reserves, end stage renal disease on dialysis, clinically significant concurrent infections requiring therapy, or unstable cardiac function.
- Invasive malignancies over the previous 5 years (except treated early stage carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, and completely resected papillary thyroid and follicular thyroid cancers).
- Splenic irradiation within 6 months prior to receiving the first dose of study medication.
- Life expectancy less than 6 months.
Data sourced from ClinicalTrials.gov (NCT01445769). 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. Informational only — not medical advice.