An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions
The purpose of this Phase 3 study is to evaluate the efficacy and safety of Luspatercept
compared with placebo in subjects with myeloproliferative neoplasm (MPN)-associated
Myelofibrosis (MF) and anemia on concomitant Janus kinase 2 (JAK2) inhibitor therapy and who
require red blood cell count (RBC) transfusions.
The study is divided into Screening Period, a Treatment Phase (consisting of a Blinded Core
Treatment Period, a Day 169 Response Assessment, a Blinded Extension Treatment Period, and an
Open-label Extension Treatment Period), and a Posttreatment Follow-up Period.
Permitted Concomitant Medications and Procedures
Subjects are receiving a JAK2 inhibitor for the treatment of MPN-associated MF that is
approved in the country where the study is being conducted. JAK2 inhibitors are to be
used according to their respective label and as prescribed as part of the subject's
standard-of-care therapy as prescribed by their physician prior to study entry.
Best supportive care (BSC) includes, but is not limited to, treatment with transfusions
(eg, RBC, platelet, whole blood), ICTs, antibiotic, antiviral and/or antifungal therapy,
and nutritional support as needed.
Granulocyte colony-stimulating factors (ie, G-CSF, granulocyte macrophage
colony-stimulating factor [GM-CSF]) are allowed only in cases of neutropenic fever or as
clinically indicated per product label.
Prophylactic antithrombotic therapy is permitted.
Thrombopoietin and platelet transfusions are permitted.
Treatment with systemic corticosteroids is permitted for nonhematological conditions
providing the subject is receiving a constant dose equivalent to ≤ 10 mg prednisone
during the study.
Administration of attenuated vaccines (eg, influenza vaccine) is allowed if clinically
indicated per Investigator discretion.
Iron chelation therapy (ICT) is to be used according to the product label. If the label
permits, the ICT dose should be stable during at least the first 24 weeks of IP.
Initiation of ICT while within the first 24 weeks of IP should be clinically indicated
to treat an AE.
Prohibited Concomitant Medications
The following concomitant medications are specifically excluded during the course of the
Cytotoxic, chemotherapeutic, targeted, or investigational agents/therapies (excluding
JAK2 inhibitor therapy)
Azacitidine, decitabine, or other hypomethylating agents
Lenalidomide, thalidomide, and pomalidomide
Erythropoietin stimulating agents (ESAs) and other RBC hematopoietic growth factors (eg,
Hydroxyurea or other alkylating agents
Androgens (unless given to treat hypogonadism)
Oral retinoids (topical retinoids are permitted)
Systemic corticosteroids at a dose equivalent to > 10 mg prednisone
Investigational products for the treatment of MPN-associated MF
Myeloproliferative Disorders, Myelofibrosis, Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Anemia
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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