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Home › Conditions › MDS

MDS

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for MDS

20 trials tracked for MDS: 3 in phase 3 or 4 and 1 with published results. The most-cited published study has 215 citations.

20Trials tracked
3Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 3 Phase 2 14 Phase 1 2 Other / NA 1
  1. Phase 3 Controlled Study of Post-transplant Azacitidine for Prevention of Acute Myelogenous Leukemia and Myelodysplastic Syndrome Relapse (VZ-AML-PI-0129) Completed · 215 cited
  2. Phase 3 Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) Completed
  3. Phase 3 APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS) Completed
  4. Phase 2 Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS) Completed
  5. Phase 2 Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC Completed
  6. Phase 2 Ganciclovir by Infusion and by Mouth in Treating Patients With Cytomegalovirus After Donor Bone Marrow Transplant Completed
Show 14 more trials
  1. Phase 2 Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases Completed
  2. Phase 2 Study of FF-10501-01 in Patients With Relapsed or Refractory Hematological Malignancies Completed
  3. Phase 2 Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating Agents Completed
  4. Phase 2 Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) Completed
  5. Phase 2 Allo vs Hypomethylating/Best Supportive Care in MDS (BMTCTN1102) Completed
  6. Phase 2 SGI-110 in Participants With Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML) Completed
  7. Phase 2 Fludarabine and Cytarabine in Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS) Completed
  8. Phase 2 Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS) Completed
  9. Phase 2 Allogeneic Transplantation From Related Haploidentical Donors Completed
  10. Phase 2 Safety Study of Bone Marrow Transplant Using Mismatched Tissue Followed by Chemotherapy Completed
  11. Phase 2 Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS Completed
  12. Phase 1 Pharmacokinetics and Safety Study of Azacitidine in Cancer Patients With and Without Impaired Renal Function Completed
  13. Phase 1 A Phase I Study of AC220 in Patients With Relapsed/Refractory Acute Myeloid Leukemia Regardless of FLT3 Status Completed
  14. N/A Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS) Completed

Showing the 20 most-cited and recently-updated of 20 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

MDS: what the trials found

Established evidence for MDS includes the use of azacitidine [1, 19]. In a Phase 3 trial, the addition of APR-246 to azacitidine was evaluated 2.

Darbepoetin alfa demonstrated significant clinical improvements in red blood cell (RBC) management and erythroid response. Specifically, patients receiving Darbepoetin alfa showed a significantly lower percentage of participants requiring at least one RBC transfusion compared to the control group (36.1% vs 59.2%, p=0.008) 3. Furthermore, there was a significant increase in the percentage of participants achieving an erythroid response based on IWG 2006 criteria (14.7% vs 0.0%, p=0.017) 3.

Regarding bleeding management, studies comparing placebo groups showed that certain cohorts had significantly lower annualized rates of platelet transfusion events (748.9 vs 1013.5, p<0.001) and overall bleeding events (3459.9 vs 3786.4, p=0.026) 15.

Recent results — preliminary, needs further review

  • * FF-10501-01 (Phase 2) 4 * Guadecitabine (Phase 2); results showed varying rates of CR, PR, mCR, and HI in MDS patients (19%, 44%, 26%, and 33% respectively) 5 * Allogeneic Hematopoietic Cell Transplant (Phase 2); significantly higher leukemia-free survival was observed (35.8% vs 20.6%, p=0.0030) 6 * ASTX727 Dose Escalation (Phase 2) 7 * filgrastim (Phase 2); reported high chimerism rates at 6 months (98.1%) 8 * Busulfan (Phase 2) 9 * Ganciclovir (Phase 2) 10 * Cyclophosphamide (Phase 2); reported median days to neutrophil and platelet engraftment of 12 and 30 respectively 11 * Anti-Thymocyte Globulin (Phase 2) 12 * Prednisone (Phase 2) 13 * Cytarabine (Phase 2) 14 * Romiplostim (Phase 2) 17 * AC220 (Phase 1) 18 * Romiplostim (Phase N/A) 20

For the clinician treating this condition

  • Darbepoetin alfa is associated with a significant reduction in the need for RBC transfusions and an improved erythroid response rate compared to standard care 3.
  • Azacitidine remains an established intervention in the management of MDS [1, 19].
  • Evidence regarding bleeding event rates varies across studies, but specific cohorts showed significantly lower annualized platelet transfusion and overall bleeding events 15.

AI synthesis of 17 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
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This retrospective cohort study of 16 Chinese patients describes main clinical features and treatment responses in VEXAS syndrome.
A specific genetic change called a UBA1 variant was found in 81 percent of patients in China with blood disorders and inflammation affecting…
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