Phase 3
N=267
Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence
Primary Myelofibrosis · MPN-associated Myelofibrosis
Bottom Line
View on ClinicalTrials.gov: NCT01178281 ↗Enrolled (actual)
267
Serious AEs
39.6%
Results posted
Mar 2014
Primary outcome: Primary: Percentage of Participants Who Achieved RBC-Transfusion Independence — 17.3; 16.7 percentage of participants — p=1.000
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Pomalidomide 0.5 mg (Drug); Placebo (Drug); Pomalidomide (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Celgene
- Primary completion
- Jan 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants Who Achieved RBC-Transfusion Independence |
17.3; 16.7 | 1.000 |
| PRIMARY China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days |
1 | — |
| SECONDARY Overall Survival |
24.2; 26.2 | 0.929 |
| SECONDARY Duration of RBC-Transfusion Independence |
NA; 5.8 | — |
| SECONDARY Time to RBC-Transfusion Independence |
6.9; 2.4 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAE) |
164; 81; 12; 90; 32; 3 | — |
| SECONDARY Healthcare Resource Utilization |
— | — |
| SECONDARY Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score |
-0.0385; -0.0298; -0.0202; 0.0766 | — |
| SECONDARY Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale |
2.0; -1.4; 2.9; 0.3 | — |
| SECONDARY Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score |
-2.1; 4.3; 6.2; 11.9 | — |
Summary
The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis (global study) and in reversing anemia in Chinese with MPN-associated myelofibrosis and severe anemia not receiving RBC-transfusions (China extension study only)
Eligibility Criteria
Inclusion Criteria
- Age ≥ 18 years
- Myeloproliferative-neoplasm (MPN)-associated myelofibrosis
- RBC-transfusion-dependence (global study):
- Average RBC-transfusion frequency ≥ 2 units/28 days over at least the 84 days immediately prior to randomization. There must be no interval > 42 days without ≥ 1 RBC-transfusion.
- Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in
determining eligibility.
- RBC-transfusions due to bleeding are not scored in determining eligibility.
- RBC-transfusions due to chemotherapy-induced anemia are not scored in determining eligibility.
- Severe anemia (China-specific extension):
- ≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment.
- No RBC-transfusion within 6 months prior to enrollment.
- Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in the China-specific extension.
- Bone marrow biopsy within 6 months (global study only).
- Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
- Agree to follow pregnancy precautions as required by the protocol.
- Agree to receive counseling related to teratogenic and other risks of pomalidomide.
- Agree not to donate blood or semen.
Exclusion Criteria
- Prior blood cell or bone marrow allotransplant.
- Use of drugs to treat MPN-associated myelofibrosis ≤ 30 days before starting study drug.
- Treatment with erythropoietin or androgenic steroids ≤ 84 days before starting study drug.
- Anemia due to reasons other than MPN-associated myelofibrosis.
- Pregnant or lactating females.
- More than 10% blasts by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks
- Prior history of malignancies, other than the disease being studied, unless the subject has been free of the malignancy for ≥ 5 years with the following exceptions:
- Carcinoma in situ of the cervix
- Carcinoma in situ of the breast
- Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system)
- Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections.
- Prior treatment with pomalidomide.
- Allergic reaction or rash after treatment with thalidomide or lenalidomide
- Any of the following laboratory abnormalities:
- Neutrophils 3.0 x upper limit of normal (ULN)
- Total bilirubin ≥ 4 x ULN;
- Uncontrolled hyperthyroidism or hypothyroidism.
- Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting study drug
- Clinically-important heart disease within the past 6 months
Data sourced from ClinicalTrials.gov (NCT01178281). 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.