Myelodysplastic Syndromes (MDS)Phase 2DAPA-MDS1NCT07516847
Dapagliflozin for Anemia in Lower-Risk Myelodysplastic Syndromes
A Phase 2 study testing Dapagliflozin (10mg Tab) in people with Myelodysplastic Syndromes (MDS) and Anemia, run by Seug yun Yoon, MD.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes (MDS), Anemia
- What is being tested?
- Dapagliflozin (10mg Tab)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- No sites listed yet
- Study dates
- Sep 2026to Sep 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
Read more
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.
Read the 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.
- Conditions
- Myelodysplastic Syndromes (MDS), Anemia
- Being tested
- Dapagliflozin (10mg Tab)
- Keywords
- Myelodysplastic Syndromes, Anemia, SGLT2 inhibitor, Dapagliflozin, Lower-risk MDS
- Medical subject headings
- Myelodysplastic Syndromes, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dapagliflozin (10mg Tab)Drug
Also called Forxiga
Dapagliflozin 10 mg administered orally once daily for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Dapagliflozin | Experimental | Participants receive dapagliflozin 10 mg orally once daily for 24 weeks. Receives: Drug: Dapagliflozin (10mg Tab) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Within 24 weeks
Secondary outcomes (5)
Change in Hemoglobin Level
Mean change in hemoglobin level from baseline during the study period.
Measured: Up to 24 weeks
Proportion of Patients With Hemoglobin Increase ≥1.5 g/dL
Proportion of patients achieving a hemoglobin increase of ≥1.5 g/dL from baseline.
Measured: Up to 24 weeks
Change in Red Blood Cell Transfusion Requirement
Change in red blood cell transfusion requirement compared to baseline.
Measured: Up to 24 weeks
Duration of Hemoglobin Response
Duration from first documented hemoglobin response to loss of response.
Measured: Up to 24 weeks
Incidence of Adverse Events
Incidence and severity of adverse events assessed according to CTCAE criteria.
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2026
- Study startSep 2026 (estimated)
- Primary completionJan 2029 (estimated)
- Study completionSep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic Syndromes (MDS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a single-arm, open-label, phase II study in which all participants receive dapagliflozin 10 mg once daily for 24 weeks.
- Masking
- None (open label)
- Enrolment
- 37 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Seug yun Yoon, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Boryung Pharmaceutical Co., Ltd
- Responsible party
- Sponsor-investigator, Seug yun Yoon, MD, Soonchunhyang University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07516847
- Sponsor's protocol ID
- DAPA-MDS-01
- Acronym
- DAPA-MDS1
- Status last verified
- Apr 2026
- First submitted
- 1 Apr 2026
- Last updated
- 8 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 31707794Alsugair 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.
PubMed 39844769
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Prospective, Open-Label Study to Evaluate the Efficacy and Safety of Dapagliflozin for Anemia in Patients With Lower-Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT07516847
- Acronym / sponsor ID
- DAPA-MDS1 / DAPA-MDS-01
- Conditions
- Myelodysplastic Syndromes (MDS), Anemia
- Keywords
- Myelodysplastic Syndromes, Anemia, SGLT2 inhibitor, Dapagliflozin, Lower-risk MDS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 37 participants (estimated)
- Lead sponsor
- Seug yun Yoon, MDUniversity, hospital or other
- Collaborators
- Boryung Pharmaceutical Co., Ltd
- Study start
- Sep 2026 (estimated)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Sep 2029 (estimated)
- First posted
- 8 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a single-arm, open-label, phase II study in which all participants receive dapagliflozin 10 mg once daily for 24 weeks.
| Arm | Type | Receives |
|---|---|---|
| Dapagliflozin | Experimental | Drug: Dapagliflozin (10mg Tab)Participants receive dapagliflozin 10 mg orally once daily for 24 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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)
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dapagliflozin (10mg Tab)Also: ForxigaDapagliflozin 10 mg administered orally once daily for 24 weeks. | Drug | Dapagliflozin |
Primary outcome
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.
5 secondary outcomes
Change in Hemoglobin Level
Time frame: Up to 24 weeks
Proportion of Patients With Hemoglobin Increase ≥1.5 g/dL
Time frame: Up to 24 weeks
Change in Red Blood Cell Transfusion Requirement
Time frame: Up to 24 weeks
Duration of Hemoglobin Response
Time frame: Up to 24 weeks
Incidence of Adverse Events
Time frame: Up to 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2026.
- Results publication: PubMed 31707794Mazer 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.
- Results publication: PubMed 39844769Alsugair 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dapagliflozin (10mg Tab) in people with Myelodysplastic Syndromes (MDS) and Anemia, run by Seug yun Yoon, MD.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Seug yun Yoon, MD, PhD (+82-10-9267-2281, ysy6496@schmc.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hemoglobin Response Rate (time frame: Within 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07516847, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2026.