Dapagliflozin for Anemia in Lower-Risk Myelodysplastic Syndromes (DAPA-MDS1)

April 1, 2026 updated by: Seug yun Yoon, MD

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic disorders characterized by ineffective hematopoiesis and cytopenias. Among these, anemia is the most common and clinically significant manifestation in patients with lower-risk MDS, often leading to fatigue, decreased quality of life, and increased transfusion requirements.

Current treatment options for anemia in lower-risk MDS include erythropoiesis-stimulating agents (ESA) and other therapies such as luspatercept. However, these treatments are not universally effective, and access may be limited in certain settings. As a result, many patients remain transfusion-dependent or experience persistent anemia, highlighting the need for additional therapeutic options.

Sodium-glucose cotransporter-2 (SGLT2) inhibitors are widely used in the management of diabetes mellitus, heart failure, and chronic kidney disease. Multiple clinical studies have consistently demonstrated increases in hemoglobin and hematocrit levels in patients receiving SGLT2 inhibitors. The proposed mechanisms include increased erythropoietin production, modulation of iron metabolism, and reduction in plasma volume. These findings suggest a potential role of SGLT2 inhibitors in stimulating erythropoiesis.

Recent observational data have suggested that SGLT2 inhibitor therapy may improve hemoglobin levels in patients with myeloid neoplasms, including MDS. However, these findings are limited by small sample sizes and retrospective study designs, and prospective clinical data are lacking.

This study is designed as a prospective, single-arm, phase II clinical trial to evaluate the efficacy and safety of dapagliflozin in patients with lower-risk MDS and anemia. Participants will receive dapagliflozin 10 mg orally once daily for 24 weeks. The study will assess hemoglobin response, transfusion requirements, and safety outcomes over the study period.

The results of this study are expected to provide proof-of-concept evidence for the use of SGLT2 inhibitors as a potential therapeutic option for anemia in patients with lower-risk MDS and to support further clinical development in this setting.

Study Type

Interventional

Enrollment (Estimated)

37

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dapagliflozin
Participants receive dapagliflozin 10 mg orally once daily for 24 weeks.
Drug: Dapagliflozin (10mg Tab)
Dapagliflozin 10 mg administered orally once daily for 24 weeks.
Other Names:
  • Forxiga

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hemoglobin Response Rate
Time Frame: Within 24 weeks
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Hemoglobin Level
Time Frame: Up to 24 weeks
Mean change in hemoglobin level from baseline during the study period.
Up to 24 weeks
Proportion of Patients With Hemoglobin Increase ≥1.5 g/dL
Time Frame: Up to 24 weeks
Proportion of patients achieving a hemoglobin increase of ≥1.5 g/dL from baseline.
Up to 24 weeks
Change in Red Blood Cell Transfusion Requirement
Time Frame: Up to 24 weeks
Change in red blood cell transfusion requirement compared to baseline.
Up to 24 weeks
Duration of Hemoglobin Response
Time Frame: Up to 24 weeks
Duration from first documented hemoglobin response to loss of response.
Up to 24 weeks
Incidence of Adverse Events
Time Frame: Up to 24 weeks
Incidence and severity of adverse events assessed according to CTCAE criteria.
Up to 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Seug yun Yoon, MD

Collaborators

Boryung Pharmaceutical Co., Ltd

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

September 1, 2026

Primary Completion (Estimated)

January 1, 2029

Study Completion (Estimated)

September 1, 2029

Study Registration Dates

First Submitted

April 1, 2026

First Submitted That Met QC Criteria

April 1, 2026

First Posted (Actual)

April 8, 2026

Study Record Updates

Last Update Posted (Actual)

April 8, 2026

Last Update Submitted That Met QC Criteria

April 1, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DAPA-MDS-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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