A Study of TAK-226 for Transfusion-Dependent Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes

NCT07319845 | Not Yet Recruiting Phase 2

• 1 other identifier: TAK-226-2001
• Study Type: interventional
• Target: 27
• Locations: 0 countries
• Sites: N/A

Brief Summary

The main aim of the study is to evaluate how TAK-226 improves symptoms of transfusion-dependent anemia in Japanese patients with lower-risk myelodysplastic syndromes. The study consists of Screening Period (up to 6 weeks), Treatment Period , Safety Follow-Up Period (8 weeks), and Long-Term Follow-Up Period (5 years from the first dose of the study drug or 3 years after the last dose, whichever is longer). Participants of this study will be administered TAK-226 during Treatment Period. Subsequently, the participants will be monitored for side effects related to the study treatment during Safety Follow-Up Period and Long-Term Follow-Up Period. The approximate duration of participation for a participant is up to approximately 6 years. During the study period, participants will visit the study clinic/hospital multiple times as per the study schedule. During Treatment Period, the participants will come to the clinic/hospital approximately every two to four weeks.

Conditions

Myelodysplastic Syndromes

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Achieving Transfusion Independence (TI) for Greater Than or Equal to 8 Weeks from Baseline through Week 24

  Transfusion independence is defined as the absence of any red blood cells (RBC) transfusions in a period of at least 8 weeks after the first dose of the study treatment through week 24.

  Baseline, Up to Week 24

Secondary Outcomes (8)

• Percentage of Participants Achieving TI for Greater Than or Equal to 24 Weeks from Baseline through Week 48

  Baseline, Up to Week 48

• Percentage of Participants with High Transfusion Burden (HTB) Achieving TI for Greater Than or Equal to 8 Weeks from Baseline through Week 24

  Baseline, Up to Week 24

• Percentage of Participants Achieving Mean Haemoglobin (Hgb) Increase of Greater Than or Equal to 1.5 grams per deciliter (g/dL) for Greater Than or Equal to 8 Weeks from Baseline through Week 24

  Baseline, Up to Week 24

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

  Up to approximately 6 years

• Change from Baseline in Clinical Laboratory Values, Vital Signs, and Electrocardiograms (ECGs)

  From the time of signing the informed consent form through safety follow-up, approximately 16 months

Study Arms (1)

TAK-226

EXPERIMENTAL

Participants will receive the study drug, TAK-226, administered subcutaneously every four weeks (Q4W), up to 48 weeks or until the end of for approximately one year during Treatment Period.

Drug: TAK-226

Interventions

TAK-226 DRUG

TAK-226 subcutaneous injection

Also known as: Elritercept, KER-050

TAK-226

Eligibility Criteria

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

You may qualify if:

• Participants or their legally authorized representative must be willing and able to sign the ICF and to adhere to the protocol requirements.
• Japanese adult male or female participant ≥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 collected at Screening to confirm diagnosis) according to WHO 2016 classification that meets the IPSS-R classification of very low-, low-, or intermediate-risk MDS.
• Note: Due to expected impacts of transfusion, Hgb values from blood samples collected within 14 days following an RBC transfusion and platelet count obtained within 7 days following a platelet transfusion cannot be used to evaluate IPSS-R for eligibility.
• Transfusion dependence assessed in the 16 weeks immediately preceding enrollment in two 8-week blocks classified as either:
• a. LTB, defined as 4 to 7 RBC units per 16 weeks; or b. HTB, defined as ≥8 RBC units per 16 weeks; and c. For all participants: i. Only transfusion events for a pretransfusion Hgb \<10 g/dL are counted toward eligibility; ii. At least 1 transfusion event in each 8-week block and a minimum of 2 transfusion events separated by ≥7 days within the 16-week period immediately preceding enrollment; and iii. No consecutive 56-day period can be RBC transfusion-free during the 16 week period immediately preceding enrollment.
• Note: Only transfusions for the disease under study will be counted towards classification for LTB or HTB participants. Transfusions for intercurrent diseases (bleeding, surgical procedure, infection, etc.) are not considered.
• Refractory or intolerant to prior ESA treatment (discontinued ≥4 weeks before enrollment), 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 (eg, with granulocyte colony-stimulating factor \[G-CSF\]); ESA regimen must have been either: i. Recombinant human EPO ≥40,000 IU/week for ≥8 doses or equivalent; or ii. Darbepoetin alpha ≥500 mcrg 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 (eg, 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 \>200 U/L.
• Note: Due to expected impacts of transfusion on EPO levels, blood samples collected within the 14 days following an RBC transfusion or within 7 days following a platelet transfusion cannot be used to evaluate serum EPO level for eligibility.
• Less than 5% blasts in an evaluable bone marrow aspirate collected at Screening.
• ECOG performance status of 0 to 2.
• Women of childbearing potential (WOCBP), defined as a sexually mature woman who has not undergone surgical sterilization or who has not been naturally postmenopausal for at least 12 consecutive months must
• Agree to use 1 highly effective method of contraception and 1 additional effective (barrier) method at the same time, from the time of signing the informed consent through 60 days after the last dose of study drug; or

You may not qualify if:

• Medical History
• Del(5q) MDS or therapy-related (secondary) MDS.
• Anemia due to any other known cause (eg, 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 enrollment.
• 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 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.
• 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 the 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;

Sponsors & Collaborators

• Takeda: lead

MeSH Terms

Conditions

Myelodysplastic Syndromes

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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2025
• First Posted: January 6, 2026
• Study Start: February 6, 2026
• Primary Completion (Estimated): June 26, 2028
• Study Completion (Estimated): January 10, 2033
• Last Updated: January 6, 2026

Record last verified: 2025-12

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.