NCT04419649RecruitingPhase 2

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

A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)

Source: ClinicalTrials.gov · Last updated 19 Oct 2026

Brief summary (registry, in English)

The main aim of this study is to learn how safe elritercept is and how well adults with anemia associated with lower-risk MDS tolerate treatment with different doses of elritercept. Other aims are to learn how safe elritercept is by looking at how many participants have MDS that worsens during the study and learn about the effects of elritercept on anemia linked to MDS. The study will also look to learn how elritercept affects the production of healthy RBCs.

Status (registry): Recruiting
Phase (registry): Phase 2
Study type: Interventional
Enrollment: 210 (estimated)
First submitted: 3 Jun 2020
Start date: 19 Aug 2020
Primary completion date: 30 Oct 2029
Lead sponsor: Takeda
Diseases: Myelodysplastic syndromes
NCT number: NCT04419649
EU CT number: 2023-507469-24-00
EudraCT number: 2021-001838-19

What you commit to

Study type: Interventional · Phase 2
Planned participants: 210
From 19 Aug 2020 to the primary completion date, 30 Oct 2029
45 sites in 8 countries
Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local study participant privacy regulations.
Male or female ≥ 18 years of age, at the time of signing informed consent.
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).
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).
- Part 1 Inclusion Criteria
- Participants are eligible to be included in Part 1 of the study only if all the following criteria apply:
Diagnosis of MDS according to WHO classification that meets International Prognostic Scoring System-Revised (IPSS-R) classification of very low, low, or intermediate risk disease.
Less than (\<)5 percent (%) blasts in bone marrow during the Pretreatment Period.
Peripheral blood white blood cell (WBC) count \<13,000/microliter (μL) during the Pretreatment Period.
Anemia defined as:
- In non-transfused participants, having received no RBC transfusions within 8 weeks, Hgb concentration ≤ 10.0 g/dL during the Pretreatment Period. OR
- In LTB participants, having received 1 to 3 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks of the Pretreatment Period. OR
- In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks of the Pretreatment Period.
- Part 1 Extension - Abbreviated Inclusion Criteria
- Participants from Part 1 are eligible to be included in Part 1 Extension of the study only if all the following criteria apply:
Previously completed 4 cycles of elritercept in Part 1 with no dose-limiting toxicities (DLTs).
Participant has the potential to benefit from administration of elritercept, in the opinion of the Investigator.
\< 5% blasts in bone marrow.
Peripheral WBC count \< 13,000/μL during the 28 days prior to cycle 5 day 1 (C5D1).
- Part 2 Inclusion Criteria
- Participants are eligible to be included in Part 2 of the study only if all the following criteria apply:
Cohort A:
- Diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease.
- ring sideroblast (RS)-positive as defined by WHO 2016 criteria.
- Requiring at least 2 units of RBC transfusions in the preceding 8 weeks before cycle 1 day 1 (C1D1).
Cohort B:
- Diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease.
- Non-RS as defined by WHO 2016 criteria.
- Requiring at least 2 units of RBC transfusions in the 8 weeks before C1D1.
Cohort C:
- Diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease.
- Has anemia, defined by Hgb ≤ 10 g/dL during the Pretreatment Period, and received no RBC transfusion in the 8 weeks before C1D1.
Cohort D:
- Diagnosis of CMML according to WHO 2016 classification.
- Has anemia, defined by Hgb ≤ 10 g/dL during the Pretreatment Period, and received no RBC transfusion in the 8 weeks before C1D1. OR
- Received at least 2 units of RBC transfusions for anemia in the 8 weeks before C1D1.
Cohort E:
- Diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease.
- Requiring ≥ 2 units of RBC transfusions in the preceding 8 weeks before C1D1.
- Receipt of ≥ 20 units of RBC in transfusion over the participant's lifetime.
- Serum ferritin \> 1000 nanograms per milliliter (ng/mL) on ≥ 2 assessments in the preceding 8 weeks before C1D1.
- Treated with stable dose of iron chelation therapy for ≥ 8 weeks prior to C1D1.
Cohort F:
- Diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease.
- Requiring ≥ 2 units of RBC transfusions in the preceding 8 weeks before C1D1.
- Receipt of ≥ 20 units of RBC in transfusion over the participant's lifetime.
- Serum ferritin \> 1000 ng/mL on ≥ 2 assessments in the preceding 8 weeks before C1D1.
- Not treated with iron chelation therapy in the preceding 8 weeks before C1D1 and not eligible to initiate iron chelation therapy in the opinion of the Investigator and in accordance with local treatment guidelines for initiation of iron chelation therapy.
Cohort G:
- Diagnosis of MDS according to WHO 2016 classification that meets IPSS-R classification of very low, low, or intermediate risk disease.
- RS-positive as defined by WHO 2016 criteria OR non-RS as defined by WHO 2016 criteria.
- Relapsed, refractory, or intolerant to luspatercept treatment
- Relapsed is defined as documentation of response to luspatercept therapy followed by subsequent development of a need for transfusion(s), whether the transfusion need develops during luspatercept treatment or after luspatercept has been discontinued.
- Refractory is defined as documentation of no response with luspatercept ≥ 1 mg/kg administered for ≥ 12 weeks duration.
- Intolerant is defined as documentation of discontinuation of luspatercept therapy due to intolerance including, discontinuation because of any adverse event (serious or nonserious) at any time after treatment initiation.
- Has anemia, defined as either of the following:
- Has received at least 1 unit of RBC transfusions for anemia in the 8 weeks before C1D1; OR
- Hgb ≤ 10 g/dL, and received no RBC transfusion in the 8 weeks before C1D1
- Erythropoietin (EPO) \< 500 international units per liter (U/L) at Baseline.
- Last dose of luspatercept is ≥ 3 weeks from C1D1.
\< 5% blasts in bone marrow assessed by bone marrow aspirate during the Pretreatment Period.
- Part 1 Exclusion Criteria
- Participants are excluded from Part 1 of the study if any of the following criteria apply.
- Medical History
Diagnosis of MDS with deletion of chromosome 5q (Del5q).
Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and/or antifungals for neutropenia are allowed.
Presence of uncontrolled heart disease or New York Heart Association (NYHA) Class III or IV heart failure.
History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.
History of stroke, deep venous thrombosis (DVT), or arterial embolism within 6 months prior to C1D1.
Major surgery within 28 days prior to C1D1. Participants must be completely recovered from any previous surgery prior to C1D1.
Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B virus (HBV), or active infectious 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.
Any malignancy other than MDS that has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C1D1. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
History of solid organ or hematological transplantation.
Presence of uncontrolled hypertension, defined as systolic blood pressure (BP) ≥ 150 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.
Body mass index (BMI) ≥ 40 kilograms per meter square (kg/m\^2) during the Pretreatment Period.
History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the investigational medicinal product (IMP).
- Treatment History
Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
Treatment with ESA within 56 days prior to C1D1.
Prior or concurrent chronic treatment with granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF).
Iron chelation therapy if initiated within 8 weeks prior to C1D1.
Vitamin B12 therapy initiated within 8 weeks prior to C1D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.
Treatment with another investigational drug or device or approved therapy for investigational use ≤ 28 days prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.
- Laboratory Exclusions (during Pretreatment Period)
Platelet count \> 450 ✕ 10\^9/L or \< 30 ✕ 10\^9/L.
Transferrin saturation \< 15%.
Ferritin \< 50 nanograms per milliliter (ng/mL).
Folate \< 4.5 nanomoles per liter (nmol/L) (\< 2.0 ng/mL).
Vitamin B12 \< 148 picomoles per liter (pmol/L) (\< 200 picograms per milliliter \[pg/mL\]).
Estimated glomerular filtration rate (GFR) \< 30 milliliter per minute per 1.73 meter square (mL/min/1.73 m\^2), as determined by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation.
Positive for HIV.
- Miscellaneous
Pregnant or lactating females.
Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.
Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or contract research organization (CRO) employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
- Part 1 Extension - Exclusion Criteria
- Participants from Part 1 are excluded from Part 1 Extension of the study if any of the following criteria apply.
- Medical History
Discontinuation of IMP in Part 1 for any reason.
Has not completed a study visit in the past 12 months.
Active infection requiring parenteral antibiotic therapy within 28 days prior to C5D1 or oral antibiotics within 14 days of C5D1. Prophylactic antibiotics and/or antifungals for neutropenia are allowed.
Presence of uncontrolled heart disease or NYHA Class III or IV heart failure.
History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.
History of stroke, DVT, or arterial embolism within 6 months prior to C5D1.
Major surgery within 28 days prior to C5D1. Participants must be completely recovered from any previous surgery prior to C5D1.
Known positive for HIV, active infectious HBV, or active infectious HCV. Participants without known positive history of HIV, HBV, and/or HCV do not require further testing, unless testing is mandated per local guidelines.
Any malignancy other than MDS that has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C5D1.
History of solid organ or hematological transplantation.
Presence of uncontrolled hypertension, defined as systolic BP ≥ 150 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.
BMI ≥ 40 kg/m\^2 during the 28 days prior to C5D1.
- Treatment History
Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
Treatment with ESA within 56 days prior to C5D1.
Prior or concurrent chronic treatment with G-CSF or GM-CSF.
Iron chelation therapy if initiated within 8 weeks prior to C5D1.
Vitamin B12 with treatment initiated within 8 weeks prior to C5D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.
Treatment with another investigational drug or device or approved therapy for investigational use ≤ 28 days prior to C5D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C5D1, whichever is longer. Previous treatment with elritercept is acceptable.
- Laboratory Exclusions (during Abbreviated Pretreatment Period)
Platelet count \> 450 × 10\^9/L or \< 30 × 10\^9/L.
Transferrin saturation \< 15%.
Ferritin \< 50 ng/mL.
Folate \< 4.5 nmol/L (\< 2.0 ng/mL).
Vitamin B12 \< 148 pmol/L (\< 200 pg/mL).
Estimated GFR \< 30 mL/min/1.73 m\^2, as determined by the CKD-EPI equation.
- Miscellaneous
Pregnant or lactating females.
Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.
Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or CRO employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
- Part 2 Exclusion Criteria
- Participants are excluded from Part 2 of the study if any of the following criteria apply.
- Medical History
Diagnosis of MDS with Del5q.
Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
Active infection requiring parenteral antibiotic therapy within 28 days prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic antibiotics and/or antifungals for neutropenia are allowed.
Presence of the following cardiac conditions:
- Presence of uncontrolled heart disease or NYHA Class III or IV heart failure.
- QTcF (QT interval corrected by Fridericia's formula) \> 500 msec on the screening or C1D1 electrocardiogram (ECG; mean of 3 measurements).
- Uncontrolled clinically significant arrhythmia (participants with rate-controlled atrial fibrillation are not excluded).
- Acute myocardial infarction or unstable angina pectoris ≤ 6 months prior to C1D1.
Presence of uncontrolled hypertension, defined as systolic BP ≥ 160 mmHg or diastolic BP ≥ 100 mmHg despite adequate treatment.
History of stroke, DVT, or arterial embolism within 6 months prior to C1D1.
History of drug or alcohol abuse (as defined by the Investigator) within the past 2 years.
Major surgery within 28 days prior to C1D1. Participants must be completely recovered from any previous surgery prior to C1D1.
Any malignancy other than MDS or CMML that has not been in remission and/or has required major surgery or systemic therapy including radiation, chemotherapy, targeted therapy, or hormonal therapy within 1 year prior to C1D1.
History of solid organ or hematological transplantation.
Diagnosis of hemolytic anemia, active bleeding, hemoglobinopathies, or congenital disorders as a cause of the participant's anemia.
NCI-CTCAE Grade ≥ 2 bleeding events within the 3 months prior to C1D1.
Receipt of an RBC or platelet transfusion for any reason(s) or combination of reasons other than underlying MDS within the 16 weeks prior to C1D1. If a participant requires a transfusion for an unanticipated reason during the Pretreatment Period, a prolonged screening period may be considered after discussion with the Medical Monitor.
Known positive for HIV, active infectious HBV with positive viral load (HBV DNA), or active infectious HCV with positive viral load (HCV RNA). Participants without known positive history of HIV, HBV, and/or HCV do not require further testing, unless testing is mandated per local guidelines.
BMI ≥ 40 kg/m\^2.
History of severe allergic or anaphylactic reaction(s) or hypersensitivity to recombinant proteins or excipients in the IMP.
Diagnosis of cirrhosis, non-alcoholic steatohepatitis, alcoholic liver disease, hepatitis, or other liver disease (acute or chronic) meeting Child-Pugh C criteria for hepatic impairment. Participants with elevated liver enzymes are allowed if the liver enzyme elevation is suspected to be due to iron-overload or iron chelation, and other hepatic causes have been ruled out, in the opinion of the Investigator.
- Treatment History
Prior treatment with azacitidine, decitabine, lenalidomide, or sotatercept.
Prior treatment with luspatercept (Cohorts A, B, C, D, E, and F only).
Treatment with ESA within 8 weeks prior to C1D1.
Iron chelation therapy if initiated within 8 weeks prior to C1D1. Participants on stable doses of iron chelation therapy for ≥ 8 weeks are allowed.
Vitamin B12 and/or folate therapy initiated within 8 weeks prior to C1D1. Participants on stable replacement doses for ≥ 8 weeks and without concurrent vitamin B12 or folate deficiency are allowed.
Any need to receive a prohibited medication.
Treatment with another investigational drug or device or approved therapy for investigational use within 8 weeks prior to C1D1, or, if the half-life of the previous product is known, within 5 times the half-life prior to C1D1, whichever is longer.
- Laboratory Exclusions (during Pretreatment Period)
Platelet count \> 800 × 10\^9/L or \< 25 × 10\^9/L.
Ferritin \< 50 ng/mL.
Folate \< 4.5 nmol/L (\< 2.0 ng/mL).
Vitamin B12 \< 148 pmol/L (\< 200 pg/mL).
Estimated GFR \< 30 mL/min/1.73 m\^2 as determined by the CKD-EPI equation.
- Miscellaneous
Pregnant or lactating females.
Any other condition not specifically noted above which, in the opinion of the Investigator, would preclude the participant from participating in the study.
Participants who are investigational site staff members directly involved in the conduct of the trial and their immediate family members, site staff members otherwise supervised by the Investigator, or participants who are Sponsor or CRO employees directly involved in the conduct of the study. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
- For Cohort G ONLY:
Any luspatercept related AE Grade ≥ 3 that has not resolved to baseline or Grade ≤ 1.
History of allergy/anaphylaxis/hypersensitivity to luspatercept.
Prior treatment with imetelstat.

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (45)

Sites in registry order. Pick your city on the disease page to sort them by distance.

Site	City	Country	Site status
Royal Adelaide Hospital Coordinator of this site? Confirm its status	Adelaide	Australia	Recruiting
Border Medical Oncology Research Coordinator of this site? Confirm its status	Albury	Australia	Recruiting
Flinders Medical Centre Coordinator of this site? Confirm its status	Bedford Park	Australia	Recruiting
Boxhill Hospital Coordinator of this site? Confirm its status	Box Hill	Australia	Recruiting
Townsville University Hospital Coordinator of this site? Confirm its status	Douglas	Australia	Completed
University Hospital Geelong Coordinator of this site? Confirm its status	Geelong	Australia	Recruiting
Austin Health Coordinator of this site? Confirm its status	Heidelberg	Australia	Recruiting
St Vincent's Hospital Melbourne Coordinator of this site? Confirm its status	Melbourne	Australia	Recruiting
Royal Melbourne Hospital Coordinator of this site? Confirm its status	Melbourne	Australia	Recruiting
Tweed Hospital Coordinator of this site? Confirm its status	Tweed Heads	Australia	Recruiting
Ballarat Oncology & Haematology Service Coordinator of this site? Confirm its status	Wendouree	Australia	Completed
Westmead Hospital Coordinator of this site? Confirm its status	Westmead	Australia	Recruiting
Fakultni nemocnice Kralovske Vinohrady Coordinator of this site? Confirm its status	Prague	Czechia	Active, not recruiting
Klinikum Bayreuth GmbH Coordinator of this site? Confirm its status	Bayreuth	Germany	Active, not recruiting
Charite Universitaetsmedizin Berlin KöR Coordinator of this site? Confirm its status	Berlin	Germany	Active, not recruiting
Praxis am Volkspark Coordinator of this site? Confirm its status	Berlin	Germany	Active, not recruiting
Universitaetsklinikum Bonn AöR Coordinator of this site? Confirm its status	Bonn	Germany	Active, not recruiting
Marien Hospital Duesseldorf GmbH Coordinator of this site? Confirm its status	Duesseldorf	Germany	Active, not recruiting
Klinikum Esslingen GmbH Coordinator of this site? Confirm its status	Esslingen am Neckar	Germany	Active, not recruiting
Martin-Luther-Universitaet Halle-Wittenberg Coordinator of this site? Confirm its status	Halle (Saale)	Germany	Active, not recruiting
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR Coordinator of this site? Confirm its status	Mainz	Germany	Active, not recruiting
Hospital Universitari Vall d'Hebron Coordinator of this site? Confirm its status	Barcelona	Spain	Recruiting
Institut Catala D'oncologia Coordinator of this site? Confirm its status	L'Hospitalet de Llobregat	Spain	Recruiting
Hospital Universitario Central De Asturias Coordinator of this site? Confirm its status	Oviedo	Spain	Recruiting
Hospital Universitario De Salamanca Coordinator of this site? Confirm its status	Salamanca	Spain	Recruiting
Hospital Universitario Y Politecnico La Fe Coordinator of this site? Confirm its status	Valencia	Spain	Recruiting
Centre Hospitalier Universitaire de Nantes Coordinator of this site? Confirm its status	Nantes	France	Recruiting
Centre Hospitalier Universitaire de Nice Coordinator of this site? Confirm its status	Nice	France	Recruiting
Hopital Saint Louis Coordinator of this site? Confirm its status	Paris	France	Recruiting
Hopital Haut-Leveque Coordinator of this site? Confirm its status	Pessac	France	Recruiting
Hôpital NOVO Coordinator of this site? Confirm its status	Pontoise	France	Recruiting
Sheba Medical Center - Sheba Fund for Health Services and Research Coordinator of this site? Confirm its status	Ramat Gan	Israel	Recruiting
Sourasky Medical Center - Infrastructure and Health Services Fund of the Tel Aviv Medical Center Coordinator of this site? Confirm its status	Tel Aviv	Israel	Completed
Middlemore Hospital Coordinator of this site? Confirm its status	Auckland	New Zealand	Completed
The Center for Cancer and Blood Disorders (CCBD) - Bethesda Coordinator of this site? Confirm its status	Bethesda	United States	Recruiting
City of Hope National Medical Center Coordinator of this site? Confirm its status	Duarte	United States	Recruiting
Hackensack Medical Center Coordinator of this site? Confirm its status	Hackensack	United States	Recruiting
Genesis Cancer and Blood Institute Coordinator of this site? Confirm its status	Hot Springs	United States	Recruiting
Univ of Texas MD Anderson Cancer Center, Division of Cancer Medicine Coordinator of this site? Confirm its status	Houston	United States	Recruiting
Karmanos Cancer Institute at McLaren Greater Lansing Coordinator of this site? Confirm its status	Lansing	United States	Completed
Baptist Clinical Research Institute Coordinator of this site? Confirm its status	Memphis	United States	Recruiting
University of Miami School of Medicine Sylvester Comprehensive Cancer Center (SCCC) Coordinator of this site? Confirm its status	Miami	United States	Recruiting
University of Pittsburgh Medical Health Center Coordinator of this site? Confirm its status	Pittsburgh	United States	Completed
New York Cancer and Blood Coordinator of this site? Confirm its status	Shirley	United States	Recruiting
H. Lee Moffitt Cancer Center and Research Center Coordinator of this site? Confirm its status	Tampa	United States	Recruiting

This page is information, not medical advice. Discuss any trial with your doctor.