A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorTakeda
About this trial
The main aim of this study is to find out how well elritercept works to improve anemia in participants with myelofibrosis (MF) who are taking ruxolitinib when compared to placebo.
Other aims are to learn how elritercept improves anemia compared to placebo; to learn if elritercept reduces tiredness, improves symptoms related to MF, and helps participants do physical activities more easily. The study also aims to find out how elritercept affects the bone marrow, the spleen, and whether participants develop antibodies to the study drug.
The study will also check how safe elritercept is compared to placebo, and if elritercept stays safe over a long period of time. Participants will receive study treatment for at least 9 months (36 weeks). After this period, participants who received placebo will have the option to switch to elritercept.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Aged ≥18 years at the time of signing the informed consent form (ICF).
Able to understand the purpose and risks of the trial and voluntarily sign an ICF.
Diagnosed with primary myelofibrosis (PMF), post-essential thrombocythemia (post-ET MF) or post-polycythemia vera (post-PV MF) according to the 2022 WHO criteria (WHO Classification of Tumours Editorial Board 2024), confirmed by local pathology report.
Transfusion status as assessed in the 12 weeks immediately preceding randomization classified as Transfusion Dependent: 3 to 8 RBC units over 12 weeks.
Disqualifiers
Prior treatment with luspatercept, sotatercept, or other transforming growth factor beta inhibitors or activin receptor ligand traps.
Androgens (including danazol). Participants on stable androgen dosing for hypogonadism for ≥8 weeks are allowed.
erythropoiesis-stimulating agents.
granulocyte colony stimulating factor or granulocyte-macrophage colony stimulating factor.
Trial design
Design model
Parallel
Treatments tested in this trial
Placebo
DrugElritercept-matching placebo
Elritercept
DrugElritercept, SC, injection
Treatment groups
324 Participants
are divided into 2 treatment groupsGroup A: ElriterceptExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 2 interventions
Trial outcomes
Primary outcomes
1
Proportion of Participants Who Are Red Blood Cell-Transfusion Independent (RBC-TI) for Any Consecutive Greater Than or Equal to (≥) 12-Week Period During the 36-Week Double-Blinded Treatment Period
RBC-TI is defined as no RBC transfusions administered for the specified time period during study treatment.
Time frame
From Cycle 1 Day 1 through Week 36 (each cycle is 28 days)Secondary outcomes
1
Proportion of Participants Who Achieve ≥50 Percent (%) Reduction in RBC Transfusion Burden From Baseline Over Any Consecutive 12-Week Period
Time frame
From Cycle 1 Day 1 through Week 36 (each cycle is 28 days)2
Proportion of Participants Who Are RBC-TI for Any Consecutive ≥16-Week Period
RBC-TI is defined as no RBC transfusions administered for the specified time period during study treatment.
Time frame
From Cycle 1 Day 1 through Week 36 (each cycle is 28 days)3
Proportion of Participants Who Are RBC-TI for Any Consecutive ≥12-Week Period With Concurrent Mean Hemoglobin (Hgb) Increase ≥1.5 Grams per Deciliter (g/dL) From Baseline
RBC-TI is defined as no RBC transfusions administered for the specified time period during study treatment.
Time frame
From Cycle 1 Day 1 through Week 36 (each cycle is 28 days)4
Proportion of Participants Who Are RBC-TI for Any Consecutive ≥24-Week Period
RBC-TI is defined as no RBC transfusions administered for the specified time period during study treatment.
Time frame
From Cycle 1 Day 1 through Week 36 (each cycle is 28 days) Source ClinicalTrials.gov