A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Myelodysplastic Syndromes

In brief

The main aim of this study is to find out how well elritercept works in lowering the need for RBC transfusions. Other aims are to learn how well elritercept works in reducing the need for RBC transfusions over longer periods of time or in adults with high transfusion needs. The study will also check on how safe elritercept is and how well it is tolerated.

Key facts

Study ID: NCT06499285
Run by: Takeda
People needed: 225
Starts: 2025-05-06
Expected to finish: 2032-05-01
Last updated by the study team: 2026-05-13

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information and/or protected personal data in accordance with national and local study participant data protections and privacy regulations.
Male or female greater than or equal to (≥)18 years of age at the time of signing informed consent.
Diagnosis of MDS with or without RS (as determined in an evaluable bone marrow aspirate, read by an independent central reader to confirm diagnosis at Screening) according to the World Health Organization 2016 classification that meets the International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.
Transfusion dependence assessed in the 16 weeks immediately preceding randomization in two 8-week blocks, classified as either:
a. Low-transfusion burden (LTB), defined as 4 to 7 red blood cells (RBC) units per 16 weeks; or b. High-transfusion burden (HTB), defined as ≥8 RBC units per 16 weeks; and c. For all participants: i. Only transfusion events for a pretransfusion hemoglobin (Hgb) lesser than (<)10 grams per deciliter (g/dL) are counted toward eligibility; ii. At least 1 transfusion event in each 8-week period and a minimum of 2 transfusion events separated by ≥7 days within the 16-week period immediately preceding randomization; and iii. No consecutive 56-day period can be RBC transfusion-free during the 16-week period immediately preceding randomization.
Refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) treatment (discontinued ≥4 weeks before randomization), or unlikely to respond to ESA treatment, defined as follows:
a. Refractory to prior ESA treatment: documentation of nonresponse or a response that was no longer maintained with a prior ESA-containing regimen, either as a single agent or combination (e.g., with granulocyte colony-stimulating factor [G-CSF]); ESA regimen must have been either: i. Recombinant human erythropoietin (EPO) ≥40,000 international units per week (IU/week) for ≥8 doses or equivalent; or ii. Darbepoetin alpha ≥500 micrograms (μg) every 3 weeks for ≥4 doses or equivalent.
b. Intolerant to prior ESA treatment: documentation of discontinuation of a prior ESA-containing regimen, either as a single agent or combination (e.g., with G-CSF), at any time after introduction due to intolerance or an AE.
c. Unlikely to respond to ESA treatment: low chance of response to ESA based on an endogenous serum EPO level greater than (>)200 units per liter (U/L).
Less than 5% blasts in an evaluable bone marrow aspirate collected at Screening, read by an independent central reader.
Eastern Cooperative Oncology Group performance status of 0 to 2.
Females of childbearing potential and sexually active males must agree to use highly effective methods of contraception.
In the opinion of the Investigator, the participant is able and willing to comply with the requirements of the protocol (e.g., all study procedures, return for follow-up visits).

You may not qualify if…

Del(5q) MDS or therapy-related (secondary) MDS.
Anemia due to any other known cause (e.g., thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and/or folate).
Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks before randomization.
Clinically significant cardiovascular disease defined as:
New York Heart Association heart disease class III or IV;
Fridericia corrected QT (QTcF) interval >500 milliseconds during Screening;
Presence of uncontrolled hypertension defined as mean systolic blood pressure ≥160 millimeters of mercury (mm Hg) or diastolic blood pressure ≥100 mm Hg during Screening; or
Uncontrolled arrhythmia, myocardial infarction, or unstable angina within 6 months before Screening.
Known ejection fraction <35%, confirmed by a local echocardiogram performed during Screening, or a previously performed echocardiogram if collected within 6 months before Screening.
Child-Pugh class C hepatic impairment.
Stroke, deep vein thrombosis, or pulmonary embolism within 6 months before Screening.
Any known history of acute myeloid leukemia (AML).
Prior history of malignancies, other than MDS, unless participant has been free of the disease (including completion of any treatment, including maintenance, for prior malignancy) for ≥ 5 years. However, participants with a history or concurrent diagnosis of the following conditions are allowed if not requiring systemic therapy:
Basal or squamous cell carcinoma of the skin;
Carcinoma in situ of the cervix;
Carcinoma in situ of the breast; and/or
Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, node, metastasis [TNM] clinical staging system).
History of solid organ or bone marrow transplantation.
Active infection requiring intravenous treatment (e.g., antibiotics, antifungals, or antivirals) within 28 days, or oral treatment within 14 days before randomization.
History of or known active chronic infection with HIV, hepatitis B virus (HBV), or hepatitis C virus (HCV). Participants without known positive history of HIV, HBV, and/or HCV do not require further testing, unless testing is mandated per local guidelines.
Body mass index ≥ 40 kilograms per meter square (kg/m\^2).
Major surgery within 28 days before randomization.
History of allergy/anaphylaxis to investigational medicinal product (IMP) excipients (refer to the current elritercept IB for a list of excipients) or recombinant proteins.
Prior use of elritercept, luspatercept, or sotatercept.
Prior use of hypomethylating agents (HMAs), isocitrate dehydrogenase inhibitor, lenalidomide, imetelstat, or immunosuppressive therapy given for treatment of MDS.

Where it is running

Flinders Medical Centre — Adelaide, South Australia, Australia (enrolling)
Porto Alegre Clinical Hospital (HCPA) — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
Townsville University Hospital — Douglas, Queensland, Australia (enrolling)
Royal Adelaide Hospital — Adelaide, South Australia, Australia (enrolling)
St Vincent's Hospital (Melbourne) Ltd — Fitzroy, Victoria, Australia (enrolling)
Peter MacCallum Cancer Center — Parkville, Victoria, Australia (enrolling)
Norton Cancer Institute — Louisville, Kentucky, United States (enrolling)
Smilow Cancer Hospital at Yale-New Haven — New Haven, Connecticut, United States (enrolling)
Los Angeles Cancer Network — Glendale, California, United States (enrolling)
Comprehensive Cancer Centers of Nevada — Henderson, Nevada, United States (enrolling)
Box Hill Hospital — Box Hill, Victoria, Australia (enrolling)
City of Hope — Duarte, California, United States (enrolling)
UC San Diego Moores Cancer Center — La Jolla, California, United States (enrolling)
Gabrail Cancer Center Research — Canton, Ohio, United States (enrolling)
Cleveland Clinic - Cleveland — Cleveland, Ohio, United States (enrolling)
Tennessee Cancer Specialists — Knoxville, Tennessee, United States (enrolling)
Maryland Oncology Hematolofy — Columbia, Maryland, United States (enrolling)
Vanderbilt University Medical Center — Nashville, Tennessee, United States (enrolling)
University of Miami Hospital and Clinics — Miami, Florida, United States (enrolling)
U.T. MD Anderson Cancer Center, Division of Cancer Medicine — Houston, Texas, United States (enrolling)
Texas Oncology Gulf Coast — The Woodlands, Texas, United States (enrolling)
Tranquil Research — Webster, Texas, United States (enrolling)
Moffitt Cancer Center — Tampa, Florida, United States (enrolling)
Westmead Hospital — Sydney, New South Wales, Australia (enrolling)
Hospital Mae de Deus, Clinical Research Unit - Cancer Institute — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)

Full record on ClinicalTrials.gov

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