At a glance
ClinicalIndex Comparison Record- ✓Age ≥18 years at informed consent
- ✓Confirmed MPN-associated myelofibrosis (PMF, post-PV MF, or post-ET MF) by bone marrow biopsy per WHO 2016 criteria
- ✓Anemia per cohort-specific criteria: Cohorts 1 & 3A require ≥3 hemoglobin levels ≤9.5 g/dL on different days (≥14 days apart, no >42 days between measurements) in 84 days pre-treatment with no RBC transfusions in that period; Cohorts 2 & 3B require 4–12 RBC units/84 days with hemoglobin <13 g/dL on C1D1
- ✓ECOG performance status ≤2
- ✕Use of hydroxyurea or other drugs affecting hematopoiesis with ongoing adverse events ≤112 days before enrollment (systemic corticosteroids allowed if stable/decreasing for ≥84 days and ≤10 mg prednisone equivalent for 28 days pre-enrollment)
- ✕Cohorts 1 & 2: JAK2 inhibitor use ≤112 days before enrollment or anticipated/substantial likelihood of ruxolitinib within first 168 days of study
- ✕Cohort 3: must be on stable ruxolitinib for ≥280 days without >2 consecutive weeks interruption and stable dose ≥112 days pre-enrollment
- ✕Use of ESAs or androgenic steroids ≤112 days before enrollment
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase-2 Study To Determine Efficacy and Safety of Luspatercept in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Anemia With or Without Red Blood Cell-Transfusion Dependence
In Brief
A Phase 2 clinical trial evaluating Luspatercept for Primary Myelofibrosis and Anemia. Completed, enrolled 95 participants across 47 sites in 4 countries.
Detailed Summary
This is a Phase 2, multicenter, open-label study to evaluate the efficacy and safety of luspatercept in subjects with MPN-associated myelofibrosis and anemia with and without RBC-transfusion dependence. The study is divided into a Screening Period, a Treatment Period (consisting of a Primary Phase, a Day 169 Disease Response Assessment, and an Extension Phase), followed by a Posttreatment Follow-up Period.
Study Details
Timeline
Interventions
Luspatercept is a recombinant fusion protein consisting of a modified form of the extracellular domain of the human active in receptor type IIB linked to the IgG1 Fc domain. Luspatercept, through a mechanism of action different from erythropoietin, works to correct ineffective erythropoiesis by promoting late-stage maturation of erythroblasts.