A Phase II, Prospective, Open-Label Study to Evaluate the Efficacy and Safety of Dapagliflozin for Anemia in Patients With Lower-Risk Myelodysplastic Syndromes

Official 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.

Eligibility Requirements

• Minimum Age: 18 Years

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 37 participants

Study Arms

• Dapagliflozin (EXPERIMENTAL)
  Participants receive dapagliflozin 10 mg orally once daily for 24 weeks.

Interventions

• DRUG: Dapagliflozin (10mg Tab) — Dapagliflozin 10 mg administered orally once daily for 24 weeks.

Primary Outcomes

• Hemoglobin Response Rate (Within 24 weeks)

Secondary Outcomes

• 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)

Eligibility Criteria

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Contact Information

• Seug yun Yoon, MD, PhD — CONTACT
  Phone: +82-10-9267-2281
  Email: ysy6496@schmc.ac.kr

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