A study to compare the efficacy & safety of Luspatercept vs. Epoetin Alfa for the treatment of anemia due to Myelodysplastic Syndrome in Erythropoiesis-stimulating agent-naive participants who are non-transfusion dependent
A Phase 3, open-label, randomized study to compare the efficacy and safety of Luspatercept (ACE-536) vs. Epoetin Alfa for the treatment of anemia due to revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate-risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-stimulating agent (ESA)-naive participants who are non-transfusion dependent (NTD).
About the study
Principal investigator
Dr. Heather Leitch
MD, PhD, FRCPC, Clinical Professor, Department of Medicine, UBC, Director, Hematology/Oncology Research, St. Paul’s Hospital, Hematologist, St. Paul’s Hospital
Area(s) of medical focus
Blood & Blood Disorders
Cancer & Tumor
Condition(s)
Blood cancers
Period of enrollment
2024-08-01 - 2029-12-31
Participating sites
St. Paul's Hospital
Purpose of trial
Luspatercept has recently become available for treatment of transfusion dependent anemia in adults with lower risk MDS who are not responsive to epoetin alfa or other erythropoiesis-stimulating agent (ESA) treatment. Epoetin alfa is commonly used for the treatment of anemia in transfusion and non-transfusion dependent adults with lower risk MDS. Luspatercept has not yet been approved for MDS.
The main purpose of this study is to see if the investigational drug luspatercept works to increase the hemoglobin of people with MDS when compared to epoetin alfa and therefore not become dependent on frequent red-blood cell transfusions, and additionally experience less recurrent infections.
The goal is to explore any changes in the hemoglobin level and red-blood cell transfusion requirements of patients with low, very-low or intermediate risk MDS.
Eligibility
Key Inclusion Criteria:
• Are at least 18 years old at the time of signing this ICF
• Are able to understand and voluntarily sign an informed consent form prior to any study-related assessments/procedures being conducted
• Are willing and able to follow the study visit schedule and other protocol requirements
• Are willing to avoid pregnancy or fathering children from the start of the study to 3 months after the last dose of study drug
• Have been diagnosed with MDS that meet Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk confirmed via bone marrow aspirate
Key Exclusion Criteria:
• Have certain types of MDS such as MDS associated with del(5q) cytogenetic abnormality, MDS unclassifiable or secondary MDS
• Have significant anemia due to other causes that are not MDS
• Are pregnant or breastfeeding
• Have bleeding disorders or prior history of malignancies, unless you have been free of the disease for more than 5 years
Contact information
Tathiana Ruiz, Study Coordinator
Kelsi Sorensen, Study Coordinator
Rachel Despotovic, Study Coordinator
Researcher
Co-Investigators
Dr. Lynda Foltz, Dr. Khaled Ramadan
Additional Information
Study Phase
Phase 3
Study Type
Drug