A Phase 3 Study of Elritercept Versus Epoetin Alfa in ESA‑Naïve Adults with Lower‑Risk Myelodysplastic Syndrome Requiring Red Blood Cell Transfusions

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What is this study about?

The study focuses on adults who have Myelodysplastic syndromes and are experiencing anemia, a condition where the blood does not carry enough oxygen. Two medicines are being compared: the investigational drug elritercept and the approved medicine epoetin alfa. Both are given by injection under the skin to help the body produce more red blood cells.

The purpose of the study is to compare the effectiveness and safety of the two treatments in reducing the need for red blood cell transfusions. Participants are randomly assigned to receive either elritercept or epoetin alfa and will be treated for up to 24 weeks. During this time, they will have regular clinic visits where blood samples are taken to check hemoglobin levels, which indicate how well the blood is carrying oxygen, and doctors will record whether transfusions are still needed.

The study involves a series of visits scheduled roughly every few weeks. At each visit, a healthcare professional will administer the injection, perform a quick blood test, and ask about any side effects. The overall goal is to see if participants can stay without transfusions for a sustained period while their hemoglobin improves.

1 enrollment and consent

after joining the trial, a written informed consent form is signed, confirming understanding of the study procedures and potential risks.

2 baseline assessments

initial medical history, physical examination, and laboratory tests are performed.

baseline hemoglobin level is measured and the need for red blood cell transfusions is documented.

3 randomization

the participant is assigned by the study system to receive either elritercept (test drug) or epoetin alfa (comparator).

4 start of treatment (cycle 1 day 1)

on the first day of the treatment cycle, the assigned medication is administered by subcutaneous injection.

elritercept is given at a dose of 5 mg per kilogram of body weight; the dose is expressed in milligram(s) per kilogram.

epoetin alfa is given at a dose of 80 000 iu (international units); iu stands for international unit, a measure of biological activity.

5 regular drug administration

the study drug continues to be given by subcutaneous injection according to the schedule defined in the trial protocol, typically on a weekly basis, for the duration of the 24‑week treatment period.

6 monitoring visits and laboratory tests

the participant attends scheduled clinic visits where blood samples are drawn to measure hemoglobin level and assess safety parameters.

records of any red blood cell transfusions received are kept and reviewed at each visit.

7 assessment of red blood cell transfusion independence

throughout weeks 1 to 24, the study evaluates whether the participant achieves red blood cell transfusion independence, defined as at least twelve consecutive weeks without receiving a transfusion while the average hemoglobin increase from baseline is 1.5 g/dl or greater.

8 final evaluation at week 24

at the end of the 24‑week period, a comprehensive assessment is performed, including the final hemoglobin measurement, review of transfusion history, and documentation of any adverse events.

9 study completion and follow‑up

after the final evaluation, the participant completes the trial and may be offered a follow‑up visit to monitor long‑term safety, as outlined in the protocol.

Who Can Join the Study?

You must be 18 years old or older when you sign the informed consent form.
You must be able to understand why the study is being done and any possible risks, and you must voluntarily sign the informed consent form allowing the use of your health information.
You must have a documented diagnosis of myelodysplastic syndrome (MDS) that is classified as very low, low, or intermediate risk according to the International Prognostic Scoring System – Revised (IPSS‑R). This diagnosis must be confirmed by a central laboratory before randomization, and blood counts (hemoglobin, platelets, neutrophils) must be taken at least 14 days after any red blood cell transfusion or 7 days after any platelet transfusion.
Your bone marrow sample taken at screening must show less than 5% blasts (immature cells), and this must be confirmed by a central pathology reviewer.
Your natural blood level of erythropoietin (EPO) must be less than 500 units per liter, measured from a blood sample taken at least 14 days after any red blood cell transfusion.
You must need red blood cell transfusion, defined as receiving 2 to 6 units of packed red blood cells (pRBCs) within any 8‑week period for at least the 8 weeks immediately before randomization. At the time of each transfusion, your hemoglobin (Hgb) must have been 9.0 g/dL or lower (if you had symptoms of anemia) or 7.0 g/dL or lower (if you had no symptoms). Transfusions given for surgery, infections, or bleeding do not count toward this requirement.
Your most recent hemoglobin (Hgb) level after the last transfusion before randomization must be less than 11.0 g/dL.
Your overall health status must be rated as 0, 1, or 2 on the Eastern Cooperative Oncology Group (ECOG) performance scale, which measures how well you can perform daily activities.

Who Cannot Join the Study?

You have received any of the listed study drugs (such as epoetin alfa, darbepoetin, certain growth‑factor medicines, lenalidomide, hypomethylating agents, luspatercept, sotatercept, imetelstat, elritercept, immunosuppressive therapy, iron‑chelation drugs, androgens, high‑dose steroids, or any other investigational drug for MDS) within the time limits described; these medicines must not have been used recently unless a special exception was approved.
You have had a solid‑organ transplant (like a kidney or liver) or a bone‑marrow transplant in the past.
You currently have an infection that needed intravenous (through a vein) antibiotics in the past 28 days or oral antibiotics in the past 14 days.
You test positive for HIV, or you have an active hepatitis B or hepatitis C infection.
Your body‑mass index (BMI) is 40 kg/m² or higher (this indicates severe obesity).
You have had major surgery within the past 28 days.
You have had a new seizure or seizures that are not well controlled in the past 12 weeks.
You are allergic to the study drug or any of its ingredients, or you have had a severe allergic reaction (anaphylaxis) to similar medicines.
You have a history of pure red cell aplasia (a condition where the body stops making red blood cells) or antibodies that attack erythropoietin (a hormone that stimulates red blood cell production).
Your laboratory test results show any of the following:
- Very low white‑blood‑cell count (ANC) below 500 cells per microliter.
- Platelet count less than 50,000 or higher than 450,000 per microliter.
- Liver enzymes (AST or ALT) three times higher than the normal upper limit.
- Total bilirubin (a waste product from red blood cells) two times higher than normal.
- Kidney function measured by eGFR less than 30 mL/min/1.73 m² (indicates severe kidney disease).
- Ferritin (iron storage protein) 50 µg/L or lower.
- Folate (vitamin B9) 2.0 ng/mL or lower.
- Vitamin B12 200 pg/mL or lower.
You are currently taking part in another interventional clinical trial or have used another investigational medicine within a period equal to five times that drug’s half‑life.
You have a type of MDS that is classified as del(5q) cytogenetic abnormality, unclassifiable MDS, or secondary MDS according to the WHO 2016 guidelines.
You are unwilling or, in the investigator’s judgment, unable to follow the study’s rules and visits.
You are of childbearing potential and will not use at least one highly effective form of contraception from the start of the study until at least 60 days after the last dose.
If you are a male participant with a partner who could become pregnant, you will not use a barrier method (such as a condom) throughout the study and for at least 60 days after the last dose.
You are pregnant, have a positive pregnancy test during screening, or are breastfeeding and do not agree to stop breastfeeding for the required time.
In France, you are under court protection, not covered by social security, or otherwise legally protected as an adult under French law.
You have a known diagnosis of acute myeloid leukemia (AML).
Your anemia is caused by another known problem such as thalassemia, thyroid disease, iron, vitamin B12, vitamin B6, zinc or folate deficiency, autoimmune or hereditary hemolytic anemia, significant bleeding, or drug‑induced anemia.
You have serious heart disease, defined as any of the following:
- New York Heart Association (NYHA) class III or IV heart failure (moderate to severe limitation of physical activity).
- QTc interval (a measure of heart rhythm on an ECG) longer than 500 milliseconds.
- Uncontrolled irregular heartbeat, recent heart attack, or unstable chest pain within the past 6 months.
Your heart’s ejection fraction (the percentage of blood the heart pumps out with each beat) is less than 35 %.
You have had a blood‑clot‑related event (stroke, transient ischemic attack, deep‑vein thrombosis, pulmonary embolism, arterial clot, etc.) within the past 6 months (superficial vein inflammation is allowed).
You have a history of other cancers (besides MDS) unless you have been cancer‑free for at least 3 years after completing all treatment; certain skin cancers, in‑situ cervical or breast cancers, and early‑stage prostate cancer are permitted if not requiring systemic therapy.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Institut Gustave Roussy	Villejuif	France
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Oslo Universitetssykehus HF	Oslo	Norway
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
IRCCS Humanitas Research Hospital	Rozzano	Italy
Hospital Universitario De Salamanca	Salamanca	Spain
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Katholieke Universiteit te Leuven	Leuven	Belgium
CHU Grenoble Alpes	La Tronche	France
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA	Rome	Italy

Other Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Algemeen Ziekenhuis Delta	Roeselare	Belgium
Lietuvos sveikatos mokslu universiteto ligonine Kauno klinikos	Kaunas	Lithuania
Mtz Clinical Research Powered By Pratia	Warsaw	Poland
Komarom-Esztergom Varmegyei Szent Borbala Korhaz	Tatabanya	Hungary
Gesellschaft Zur Forderung Des Wissenschaftlich Medizinischen Erkenntnisgewinns In Der Hamatologie Und Oncologie	Munster	Germany
Azienda Ospedaliera di Padova	Padua	Italy
Hippokration Hospital	Athens	Greece
Pratia Hematologia Sp. z o.o.	Katowice	Poland
University Of Debrecen	Debrecen	Hungary
University General Hospital Of Ioannina	Ioannina	Greece
Virgen del Rocío University Hospital	Sevilla	Spain
Ospedale San Raffaele S.r.l.	Milan	Italy
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Istituto Di Candiolo Fondazione Del Piemonte Per Loncologia IRCCS	Candiolo	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Vilniaus universiteto ligonine Santaros klinikos VšĮ	Vilnius	Lithuania
Pratia S.A.	Skorzewo	Poland
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca	Cluj Napoca	Romania
Centre Hospitalier Universitaire De Nice	Nice	France
University General Hospital Of Alexandroupoli	Alexandroupoli	Greece
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Hopital Beaujon	Clichy	France
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur	Namur	Belgium
University Hospital Galway	Galway	Ireland
Sykehuset I Vestfold HF	Tonsberg	Norway
Specialized Hospital For Active Treatment Of Hematological Diseases EAD	Sofia	Bulgaria
Hospital Universitario Virgen De La Victoria	Malaga	Spain
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara	Chaidari	Greece
Praxis am Volkspark Berlin	Berlin	Germany
Myhkyvvsszzafqwlgewhgseoxz Hblkxsabbygpzsel	Halle (Saale)	Germany
Cqij Unorwiwssz Htkzgtvz	Cork	Ireland
Ipwshmqy Cnwhsm Drfauuoqdtgnrzuyw	L'hospitalet De Llobregat	Spain
Dbq Pqgmkf Grxrptbm Anaocsxmsz Fgv Iutdmtjoff Puahvoid Fsi Majvtvw Ajc Feq Ckqezgpn Hbjwkuurau Eodq	Plovdiv	Bulgaria
Lqflu Gjlcudr Hcnsibdc Ox Acfsgq	Athens	Greece
Avblwta Sxt z oqlc	Poznan	Poland
Azoljunvq Unk	Amsterdam	The Netherlands
Hpbxx Baofow Hq	Bergen	Norway
Kpqgnzkx Bcjdfpua Gaek	Bayreuth	Germany
Aawxpll Ugacs Sqrzongzu Ltjdlk Dg Bhqhvgx	Bologna	Italy
Uczcochzmg Dcabc Sbvaf Dx Rywu Ls Sjiriwme	Rome	Italy
Hqbgycwg Vxjs dkrtufub	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not yet recruiting	30.05.2026
Bulgaria	Not yet recruiting	30.05.2026
France	Not yet recruiting	30.05.2026
Germany	Not yet recruiting	30.05.2026
Greece	Not yet recruiting	30.05.2026
Hungary	Not yet recruiting	30.05.2026
Ireland	Not yet recruiting	30.05.2026
Italy	Not yet recruiting	30.05.2026
Lithuania	Not yet recruiting	30.05.2026
Norway	Not yet recruiting	30.05.2026
Poland	Recruiting	30.05.2026
Romania	Not yet recruiting	30.05.2026
Spain	Not yet recruiting	30.05.2026
The Netherlands	Not yet recruiting	30.05.2026

Trial locations

Investigated drugs:

Erythropoietin (epoetin alfa) is a medication that helps the bone marrow make more red blood cells. In this study it is used as the standard treatment (comparator) to see how well the new drug works. Participants receive it by injection under the skin. The goal is to reduce the need for blood transfusions by raising the level of hemoglobin in the blood.

Elritercept is an experimental drug being tested for the first time in this trial. It is given as a solution for injection under the skin. The study is looking at whether Elritercept can help patients with certain low‑risk blood disorders become independent from red blood cell transfusions for at least 12 weeks, while also increasing their hemoglobin by at least 1.5 g/dL. It is being compared directly to epoetin alfa to evaluate its effectiveness and safety.

Myelodysplastic syndromes (MDS) – Myelodysplastic syndromes are a group of disorders where the bone marrow makes too few and abnormally formed blood cells. The condition often starts with a slow decline in red blood cells, causing fatigue and weakness. Over months, white blood cells and platelets may also decrease, leading to more frequent infections or easy bruising. The marrow cells appear irregular and do not mature properly. The disease may remain relatively stable for some time or gradually become more severe as blood cell production worsens.

Trial ID:

2025-523544-12-00

Protocol code:

TAK-226-3001

Trial Phase:

Therapeutic confirmatory (Phase III)

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A Phase 3 Study of Elritercept Versus Epoetin Alfa in ESA‑Naïve Adults with Lower‑Risk Myelodysplastic Syndrome Requiring Red Blood Cell Transfusions

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?