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

NCT04419649 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: KER050-MD-2012023-507469-24-00
• Study Type: interventional
• Target: 160
• Locations: 8 countries
• Sites: 47

Brief Summary

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.

Conditions

Myelodysplastic Syndromes; Cytopenia

Keywords

Transfusion; Drug Therapy; Elritercept; TAK-226; KER-050

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  An AE is defined as any untoward medical occurrence, in a clinical study participant administered a medicinal product, that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not it is related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that, at any dose: results in death, is life threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a medically important event.

  From treatment initiation to end of study (up to 11.2 years)

Secondary Outcomes (12)

• Number of Participants with Progression to Higher Risk MDS or Acute Myeloid Leukemia (AML)

  From study day 1 through end of study (up to 11.2 years)

• Percentage of Participants with Low Transfusion Burden (LTB) and High Transfusion Burden (HTB) who Achieve RBC Transfusion Independence (TI)

  From study day 1 through end of study (up to 11.2 years)

• Percentage of Participants who Achieve Hematologic Improvement Erythroid (HI-E) Response Based on Modified 2006 International Working Group (IWG)

  From study day 1 to end of study (up to 11.2 years)

• Percentage of Participants who Achieve Overall Erythroid Response

  Up to approximately 11.2 years

• Percentage of Participants who Achieve Erythropoietic Improvement

  Up to approximately 11.2 years

Study Arms (13)

Part 1: Elritercept Cohort 1

EXPERIMENTAL

Participants will be administered elritercept at 0.75 milligrams per kilogram (mg/kg), subcutaneous (SC) injection, every 4 weeks on Day 1 of each cycle for up to 4 cycles (each cycle = 28 days). Following the above dose regimen, participants will continue to receive elritercept, administered SC, on Day 1, every 4 weeks up to 24 cycles (each cycle = 28 days).

Drug: Elritercept

Part 1: Elritercept Cohort 2

EXPERIMENTAL

Participants will be administered elritercept at 1.5 mg/kg, SC injection, every 4 weeks on Day 1 of each cycle for up to 4 cycles (each cycle = 28 days). Following the above dose regimen, participants will continue to receive elritercept, administered SC, on Day 1, every 4 weeks up to 24 cycles (each cycle = 28 days).

Drug: Elritercept

Part 1: Elritercept Cohort 3

EXPERIMENTAL

Participants will be administered elritercept at 2.5 mg/kg, SC injection, every 4 weeks on Day 1 of each cycle for up to 4 cycles (each cycle = 28 days). Following the above dose regimen, participants will continue to receive elritercept, administered SC, on Day 1, every 4 weeks up to 24 cycles (each cycle = 28 days).

Drug: Elritercept

Part 1: Elritercept Cohort 4

EXPERIMENTAL

Participants will be administered elritercept at 3.75 mg/kg, SC injection, every 4 weeks on Day 1 of each cycle for up to 4 cycles (each cycle = 28 days). Following the above dose regimen, participants will continue to receive elritercept, administered SC, on Day 1, every 4 weeks up to 24 cycles (each cycle = 28 days).

Drug: Elritercept

Part 1: Elritercept Cohort 5

EXPERIMENTAL

Participants will be administered elritercept at 5.0 mg/kg, SC injection, every 4 weeks on Day 1 of each cycle for up to 4 cycles (each cycle = 28 days). Following the above dose regimen, participants will continue to receive elritercept, administered SC, on Day 1, every 4 weeks up to 24 cycles(each cycle = 28 days).

Drug: Elritercept

Part 2: Elritercept Dose Confirmation Cohort A

EXPERIMENTAL

Participants with ring sideroblasts (RS)-positive Myelodysplastic syndrome (MDS) who are requiring red blood cell (RBC) transfusions will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Part 2: Elritercept Dose Confirmation Cohort B

EXPERIMENTAL

Participants with non-RS MDS who are requiring RBC transfusions will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Part 2: Elritercept Dose Confirmation Cohort C

EXPERIMENTAL

Participants who are non-transfused with either RS-positive MDS or non-RS MDS will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Experimental: Part 2: Elritercept Dose Confirmation Cohort D

EXPERIMENTAL

Participants with chronic myelomonocytic leukemia (CMML) and anemia will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Part 2: Elritercept Dose Confirmation Cohort E

EXPERIMENTAL

Participants with MDS (either RS-positive or non-RS) who are requiring RBC transfusions, have iron-overload, and are receiving iron chelation therapy will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Part 2: Elritercept Dose Confirmation Cohort F

EXPERIMENTAL

Participants with MDS (either RS-positive or non-RS) who are requiring RBC transfusions, have iron-overload, and are not receiving iron chelation therapy will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Part 2: Elritercept Dose Confirmation Cohort G

EXPERIMENTAL

Participants with MDS (either RS-positive or non-RS) who require RBC transfusions and have either relapsed, become refractory to, or intolerant to frontline luspatercept treatment will be administered Elritercept at 3.75 mg/kg, SC injection, on Day 1, every 4 weeks for up to 24 cycles (each cycle = 28 days). The dose can be modified based on participant's response.

Drug: Elritercept

Long-term Extension Cohort

EXPERIMENTAL

Participants from Part 1 and 2 cohorts who may have potential benefit from continued elritercept treatment, in the opinion of the Investigator, may elect to continue in the LTE at the same dose they were being administered in Part 1 and 2, SC injection, on day 1, every 4 weeks until end of treatment (EOT) (approximately 122 months).

Drug: Elritercept

Interventions

Elritercept DRUG

Elritercept SC injection.

Also known as: KER-050, TAK-226

Experimental: Part 2: Elritercept Dose Confirmation Cohort D; Long-term Extension Cohort; Part 1: Elritercept Cohort 1; Part 1: Elritercept Cohort 2; Part 1: Elritercept Cohort 3; Part 1: Elritercept Cohort 4; Part 1: Elritercept Cohort 5; Part 2: Elritercept Dose Confirmation Cohort A; Part 2: Elritercept Dose Confirmation Cohort B; Part 2: Elritercept Dose Confirmation Cohort C; Part 2: Elritercept Dose Confirmation Cohort E; Part 2: Elritercept Dose Confirmation Cohort F; Part 2: Elritercept Dose Confirmation Cohort G

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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 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).
• 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 (\<)5percent (%) 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.
• In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks of the Pretreatment Period.
• 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Takeda: lead

Study Sites (47)

City of Hope National Medical Center

Duarte, California, 91010, United States

RECRUITING

University of Miami School of Medicine Sylvester Comprehensive Cancer Center (SCCC)

Miami, Florida, 33136, United States

RECRUITING

H. Lee Moffitt Cancer Center and Research Center

Tampa, Florida, 33612, United States

RECRUITING

Karmanos Cancer Institute at Mclaren Greater Lansing

Lansing, Michigan, 48910, United States

COMPLETED

University of Pittsburgh Medical Health Center

Pittsburgh, Pennsylvania, 15213, United States

COMPLETED

Border Medical Oncology Research

Albury, New South Wales, 2640, Australia

RECRUITING

Tweed Hospital

Tweed Heads, New South Wales, 2485, Australia

RECRUITING

Westmead Hospital

Westmead, New South Wales, 2145, Australia

RECRUITING

Townsville University Hospital

Douglas, Queensland, 4814, Australia

COMPLETED

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

RECRUITING

Flinders Medical Centre

Bedford Park, South Australia, 5042, Australia

RECRUITING

Boxhill Hospital

Box Hill, Victoria, 3128, Australia

RECRUITING

University Hospital Geelong

Geelong, Victoria, 3220, Australia

RECRUITING

Austin Health

Heidelberg, Victoria, 3084, Australia

RECRUITING

Royal Melbourne Hospital

Melbourne, Victoria, 3050, Australia

RECRUITING

St Vincent's Hospital Melbourne

Melbourne, Victoria, 3065, Australia

RECRUITING

Ballarat Oncology & Haematology Service

Wendouree, Victoria, 3355, Australia

COMPLETED

Fakultni nemocnice Brno

Brno, Czechia

COMPLETED

Fakultni nemocnice Kralovske Vinohrady

Prague, Czechia

RECRUITING

Vseobecna Fakultni Nemocnice Praha

Prague, Czechia

COMPLETED

CHU Angers - Hopital Hotel Dieu

Angers, France

COMPLETED

Centre Hospitalier de la Region dAnnecy

Épagny, France

COMPLETED

CHU de Nantes - Hotel Dieu

Nantes, France

RECRUITING

CHU Nice - Hopital de l'Archet 1

Nice, France

RECRUITING

Hopital Saint-Louis

Paris, France

RECRUITING

CH Rene-Dubos

Pontoise, France

RECRUITING

CHU de Bordeaux - Hopital Haut-Leveque

Talence, France

RECRUITING

Klinikum Bayreuth GmbH

Bayreuth, Germany

RECRUITING

Charite-Campus Benjamin Franklin

Berlin, Germany

NOT YET RECRUITING

Praxis am Volkspark Berlin

Berlin, Germany

NOT YET RECRUITING

University Hospital Bonn

Bonn, Germany

NOT YET RECRUITING

Marien Hospital Dusseldorf GMBH

Düsseldorf, Germany

RECRUITING

Universitaetsklinikum Duesseldorf AoeR

Düsseldorf, Germany

COMPLETED

Klinikum Esslingen GmbH

Esslingen am Neckar, Germany

RECRUITING

University Hospital Halle (Saale)

Halle, Germany

NOT YET RECRUITING

Universitaetsklinikum Leipzig AoeR

Leipzig, Germany

COMPLETED

Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz

Mainz, Germany

RECRUITING

Universitaetsmedizin Rostock

Rostock, Germany

COMPLETED

Sheba Medical Center - Sheba Fund for Health Services and Research

Ramat Gan, 52621, Israel

RECRUITING

Sourasky Medical Center - Infrastructure and Health Services Fund of the Tel Aviv Medical Center

Tel Aviv, 6423906, Israel

COMPLETED

Middlemore Hospital

Auckland, 2025, New Zealand

COMPLETED

Hospital Universitario Central de Asturias

Barcelona, Spain

RECRUITING

Hospital Universitario Vall d'Hebron

Barcelona, Spain

RECRUITING

ICO l'Hospitalet - Hospital Duran i Reynals

Barcelona, Spain

RECRUITING

Hospital Universitario de Salamanca

Salamanca, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Spain

COMPLETED

Hospital Universitari i Politecnic La Fe

Valencia, Spain

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes; Cytopenia

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Ascending dose study

Study Record Dates

• First Submitted: June 3, 2020
• First Posted: June 5, 2020
• Study Start: August 19, 2020
• Primary Completion (Estimated): October 30, 2029
• Study Completion (Estimated): October 30, 2031
• Last Updated: February 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

NZ (1); CZ (3); IL (2); FR (7); DE (11); AU (12); US (5); ES (6)