Dapagliflozin for Anemia in Lower-Risk Myelodysplastic Syndromes

NCT07516847 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: DAPA-MDS-01
• Study Type: interventional
• Target: 37
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study is a prospective, single-arm, phase II clinical trial designed to evaluate the efficacy and safety of dapagliflozin in improving anemia in patients with lower-risk myelodysplastic syndromes (MDS). Anemia is the most common clinical problem in patients with lower-risk MDS and often leads to fatigue, reduced quality of life, and the need for repeated blood transfusions. Current treatment options, including erythropoiesis-stimulating agents and other therapies, are not effective in all patients, and additional treatment options are needed. Dapagliflozin is a sodium-glucose cotransporter-2 (SGLT2) inhibitor that is widely used for the treatment of diabetes, heart failure, and chronic kidney disease. Previous studies have shown that SGLT2 inhibitors can increase hemoglobin levels, possibly by stimulating erythropoiesis. In this study, eligible patients will receive dapagliflozin 10 mg orally once daily for 24 weeks. The primary objective is to evaluate the hemoglobin response rate during the study period. Secondary objectives include changes in hemoglobin levels, transfusion requirements, and safety outcomes. This study aims to explore whether dapagliflozin can serve as a potential treatment option for anemia in patients with lower-risk MDS.

Conditions

Myelodysplastic Syndromes (MDS); Anemia

Keywords

Myelodysplastic Syndromes; Anemia; SGLT2 inhibitor; Dapagliflozin; Lower-risk MDS

Outcome Measures

Primary Outcomes (1)

• Hemoglobin Response Rate

  Proportion of patients achieving a hemoglobin increase of ≥1.0 g/dL from baseline, sustained for at least 8 weeks, in the absence of red blood cell transfusion.

  Within 24 weeks

Secondary Outcomes (5)

• Change in Hemoglobin Level

  Up to 24 weeks

• Proportion of Patients With Hemoglobin Increase ≥1.5 g/dL

  Up to 24 weeks

• Change in Red Blood Cell Transfusion Requirement

  Up to 24 weeks

• Duration of Hemoglobin Response

  Up to 24 weeks

• Incidence of Adverse Events

  Up to 24 weeks

Study Arms (1)

Dapagliflozin

EXPERIMENTAL

Participants receive dapagliflozin 10 mg orally once daily for 24 weeks.

Drug: Dapagliflozin (10mg Tab)

Interventions

Dapagliflozin (10mg Tab) DRUG

Dapagliflozin 10 mg administered orally once daily for 24 weeks.

Also known as: Forxiga

Dapagliflozin

Eligibility Criteria

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

You may qualify if:

• Adults aged ≥18 years
• Diagnosis of myelodysplastic syndromes (MDS) according to WHO or ICC criteria
• Revised International Prognostic Scoring System (IPSS-R) very low, low, or intermediate risk
• Hemoglobin ≤10 g/dL at screening
• Transfusion independent or low transfusion burden (Defined as ≤2 units of red blood cell transfusion within 8 weeks prior to enrollment)
• If receiving erythropoiesis-stimulating agents (ESA) or other anemia-directed therapy, on a stable dose for at least 8 weeks prior to enrollment
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2
• Absolute neutrophil count (ANC) ≥0.75 ×10⁹/L
• Platelet count ≥50 ×10⁹/L
• Adequate organ function:
• Creatinine clearance ≥30 mL/min AST or ALT ≤3 × upper limit of normal

You may not qualify if:

• IPSS-R intermediate-high or high-risk MDS
• Transformation to acute myeloid leukemia or ≥20% blasts
• Initiation or dose change of MDS- or anemia-directed therapy (e.g., ESA, luspatercept, hypomethylating agents) within 8 weeks prior to screening
• Red blood cell transfusion \>2 units within 8 weeks prior to enrollment
• Current use of SGLT2 inhibitors or history of serious adverse reaction to SGLT2 inhibitors
• Uncontrolled diabetes mellitus (e.g., HbA1c \>10%) or history of diabetic ketoacidosis
• Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m²
• Active or uncontrolled infection
• Absolute neutrophil count (ANC) \<0.75 ×10⁹/L or platelet count \<50 ×10⁹/L
• Pregnant or breastfeeding women
• Any condition that, in the investigator's judgment, would make participation inappropriate

Sponsors & Collaborators

• Seug yun Yoon, MD: lead
• Boryung Pharmaceutical Co., Ltd: collaborator

Related Publications (2)

• Mazer CD, Hare GMT, Connelly PW, Gilbert RE, Shehata N, Quan A, Teoh H, Leiter LA, Zinman B, Juni P, Zuo F, Mistry N, Thorpe KE, Goldenberg RM, Yan AT, Connelly KA, Verma S. Effect of Empagliflozin on Erythropoietin Levels, Iron Stores, and Red Blood Cell Morphology in Patients With Type 2 Diabetes Mellitus and Coronary Artery Disease. Circulation. 2020 Feb 25;141(8):704-707. doi: 10.1161/CIRCULATIONAHA.119.044235. Epub 2019 Nov 11. No abstract available.

  PMID: 31707794 RESULT

• Alsugair A, Fathima S, Aperna F, Gangat N, Tefferi A. Sodium-glucose co-transporter-2 inhibitor treatmentassociated changes in hemoglobin level in anemic patients with myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms. Haematologica. 2025 Jun 1;110(6):1422-1425. doi: 10.3324/haematol.2024.287032. Epub 2025 Jan 23. No abstract available.

  PMID: 39844769 RESULT

MeSH Terms

Conditions

Myelodysplastic Syndromes; Anemia

Interventions

dapagliflozin

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Seug yun Yoon, MD, PhD

CONTACT

+82-10-9267-2281; ysy6496@schmc.ac.kr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor, Division of Hematology-Oncology

Model Details: This is a single-arm, open-label, phase II study in which all participants receive dapagliflozin 10 mg once daily for 24 weeks.

Study Record Dates

• First Submitted: April 1, 2026
• First Posted: April 8, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): September 1, 2029
• Last Updated: April 8, 2026

Record last verified: 2026-04