Myelodysplastic Syndromes trial readouts
registry pull 2026-08-19Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in Myelodysplastic Syndromes. They are the sponsor's own estimated windows, revised as a study runs — not announcement dates. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Upcoming readout windows
next 180 days · by month4 studies across 3 companies, in the next 180 days.
October 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| BMY | Bristol-Myers Squibb | Asia Myelodysplastic Syndrome (MDS) RegistryNCT07355478observationalParticipant age | — | Not yet recruiting | 2026-10-31 | in 72 d | 2026-02-13 |
November 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| MGNX | MacroGenics | A Study of MGD024 in Patients With Relapsed or Refractory Hematologic MalignanciesNCT05362773Number of severe side effects in patients receiving MGD024 | Phase 1 | Active, not recruiting | Nov 2026 | ≤ 102 d | 2026-07-28 |
January 2027 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| CGEM | Cullinan Therapeutics Inc. | CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)NCT05143996Number of treatment emergent events (TEAEs) | Phase 1 | Recruiting | Jan 2027 | ≤ 164 d | 2026-01-16 |
February 2027 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| BMY | Bristol-Myers Squibb | Study of BMS-986497 (ORM-6151) as a Monotherapy, in Double and Triple Combination With Azacitidine and Venetoclax in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic SyndromeNCT06419634Incidence of dose-limiting toxicities (DLTs) | Phase 1 | Recruiting | 2027-02-01 | in 165 d | 2026-04-08 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Every registered study in Myelodysplastic Syndromes
most recently updated first119 studies match. Showing the first 100 of 119 studies — page through, or narrow the filters.
| Ticker | Name | Market cap | Study | Phase | Status | Interventions | Conditions | Enrollment | Start | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| ABBV | AbbVie | $457.5B | A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)NCT02942290AUCt for Azacitidine | Phase 1 | Completed | Drug: Azacitidine, Venetoclax | Myelodysplastic Syndromes (MDS) | 134 | 2017-01-12 | 2026-04-15actual | — | 2026-08-12 |
| IRON | Disc Medicine, Inc | $3.1B | Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and AnemiaNCT05320198Safety and Tolerability of DISC-0974 (Phase 1b only) | Phase 1/2 | Recruiting | Drug: DISC-0974 | Myelofibrosis; Anemia, Anemia, Myelofibrosis, Myelofibrosis Due to and Following Polycythemia Vera, Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Myelodysplastic Syndromes | 150 | 2022-06-06 | May 2027 | ≤ 284 d | 2026-08-12 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)NCT05490446Phase 2a: Proportion of Participants With Hemoglobin (Hb) Response | Phase 2 | Active, not recruiting | Drug: Tebapivat | Myelodysplastic Syndromes | 87 | 2022-11-07 | 2026-03-17actual | — | 2026-07-30 |
| MGNX | MacroGenics | $261M | A Study of MGD024 in Patients With Relapsed or Refractory Hematologic MalignanciesNCT05362773Number of severe side effects in patients receiving MGD024 | Phase 1 | Active, not recruiting | Drug: MGD024 | Leukemia, Acute Myeloid, Myelodysplastic Syndromes, Classical Hodgkin Lymphoma, Leukemia, B-cell, Leukemia, Hairy Cell, Mastocytosis, Aggressive Systemic, Blastic Plasmacytoid Dendritic Cell Neoplasm, Chronic Myeloid Leukemia | 130 | 2022-07-13 | Nov 2026 | ≤ 102 d | 2026-07-28 |
| KYNB | Kyntra Bio | $28M | A Study to Investigate the Efficacy and Safety of Roxadustat (FG-4592) for Treating Anemia in Participants With Myelodysplastic Syndromes (MDS)NCT07722988Percentage of Participants With Red Blood Cell-transfusion Independence (RBC-TI) Over Any Consecutive 56-day Period | Phase 3 | Not yet recruiting | Drug: Roxadustat, Placebo | Myelodysplastic Syndromes | 201 | Apr 2027 | Sep 2030 | ≤ 1,502 d | 2026-07-23 |
| PFE | Seagen, a wholly owned subsidiary of Pfizer | $155.3B | A Safety Study of SEA-CD70 in Patients With Myeloid MalignanciesNCT04227847Number of participants with adverse events (AEs) | Phase 1 | Recruiting | Drug: SEA-CD70, azacitidine, Venetoclax | Myelodysplastic Syndrome, Acute Myeloid Leukemia | 178 | 2020-08-07 | 2027-07-04 | in 318 d | 2026-07-22 |
| LLY | Eli Lilly and Company | $1.15T | Study of Oral LY3410738 in Patients With Advanced Hematologic Malignancies With IDH1 or IDH2 MutationsNCT04603001To determine the maximum tolerated dose (MTD)/recommended Phase 2 dose (RP2D) | Phase 1 | Active, not recruiting | Drug: LY3410738, Venetoclax, Azacitidine | Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Chronic Myelomonocytic Leukemia (CMML), Myeloproliferative Neoplasms (MPNs) | 260 | 2020-11-19 | 2023-07-03actual | — | 2026-07-22 |
| TCRX | TScan Therapeutics, Inc. | $107M | A Study of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell TransplantationNCT07702578Relapse-free survival (RFS) | Phase 3 | Recruiting | Drug: TSC-101 · Other: Control | AML, MDS | 310 | 2026-06-18 | Jun 2029 | ≤ 1,045 d | 2026-07-22 |
| TCRX | TScan Therapeutics, Inc. | $107M | A Phase 1/3 Study of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell TransplantationNCT05473910Occurrence of dose limiting toxicities | Phase 1 | Recruiting | Drug: SOC + TSC-101, SOC + TSC-100 · Other: Control | AML, MDS | 310 | 2022-11-01 | 2026-06-01actual | — | 2026-07-21 |
| GERN | Geron Corporation | $983M | Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)NCT02598661results2025-02-14Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants) | Phase 2/3 | Active, not recruiting | Drug: Imetelstat Sodium, Placebo | Myelodysplastic Syndromes | 289 | 2016-01-12 | 2023-10-13actual | — | 2026-07-17 |
| ONC | BeOne Medicines | $545.6B | A Study of BGB-11417 in Participants With Myeloid MalignanciesNCT04771130Part 1 And 2: Number Of Participants Experiencing Dose-limiting Toxicities (DLTs) | Phase 1/2 | Recruiting | Drug: BGB-11417, Azacitidine, Posaconazole, BGB-11417, BGB-11417 | Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasm | 260 | 2021-05-24 | 2028-02-08 | in 537 d | 2026-07-13 |
| SNY | Sanofi | — | First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)NCT05086315results2026-07-06Dose Escalation: Number of Participants With Dose Limiting Toxicities (DLTs) | Phase 1/2 | Terminatedsponsor's stated reason: Early discontinuation based on strategic sponsor decision not driven by any safety concerns. | Drug: SAR443579 | Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia | 101 | 2021-12-08 | 2025-06-13actual | — | 2026-07-06 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemiaNCT07681440observationalParticipant age | — | Terminatedsponsor's stated reason: Insufficient enrollment | Drug: Luspatercept | Myelodysplastic Syndromes, Beta-thalassemia | 30 | 2023-11-16 | 2025-05-01actual | — | 2026-07-02 |
| BMY | Bristol-Myers Squibb | $134.9B | ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood TransfusionsNCT05949684Number of participants with lower-risk non-transfusion dependent myelodysplastic syndromes (NTD-MDS) who converted to Transfusion Dependence (TD) during any continuous 16-week interval within the 96-week treatment period | Phase 3 | Active, not recruiting | Biological: Luspatercept, Epoetin Alfa | Myelodysplastic Syndromes | 402 | 2023-10-24 | 2028-03-24 | in 582 d | 2026-06-30 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome ParticipantsNCT06045689Number of participants who achieve red blood cell transfusion independence (RBC-TI) for 8 weeks with a simultaneous mean hemoglobin (Hb) increase of ≥ 1 g/dL from Week 1 to Week 24 | Phase 3 | Active, not recruiting | Drug: Luspatercept | Myelodysplastic Syndromes | 106 | 2023-10-05 | 2025-10-01actual | — | 2026-06-15 |
| BMY | Bristol-Myers Squibb | $134.9B | Patient Reported Outcomes and Patient Voice Among Patients Diagnosed With Low Risk Myelodysplastic Syndrome (LR-MDS) or Unexplained Anemia In JapanNCT07008820observationalParticipant quality of life as assessed by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQ C30) patient reported outcome questionnaire | — | Completed | Other: Health-related quality of life questionnaires, Qualitative interviews | Myelodysplastic Syndrome (MDS) | 56 | 2025-05-13 | 2026-02-24actual | — | 2026-05-26 |
| CDNA | CareDx | $2.4B | Forecasting Relapse Outcomes With AlloHeme-based Risk Detection in Post-Allo-HCT AML/MDS Patients (FORWARD)NCT07607327observationalTo evaluate AlloHeme test performance in predicting post-transplant relapse (by FDA criteria) or any cytogenetic/molecular evidence of disease resulting in unplanned treatment intervention in AML and MDS patients. | — | Not yet recruiting | Diagnostic test: AlloHeme | AML (Acute Myelogenous Leukemia), MDS (Myelodysplastic Syndrome), CMML | 400 | 2026-08-31 | 2029-12-31 | in 1,229 d | 2026-05-26 |
| BMY | Bristol-Myers Squibb | $134.9B | Treatment Patterns and Outcomes in Patients With Lower-risk Myelodysplastic Syndromes Treated With Luspatercept in ChinaNCT07073690observationalParticipant red blood cell transfusion independence | — | Completed | Drug: Luspatercept | Myelodysplastic Syndromes | 88 | 2025-04-18 | 2026-05-12actual | — | 2026-05-19 |
| ABBV | AbbVie | $457.5B | Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic SyndromeNCT04401748Overall survival (OS) | Phase 3 | Active, not recruiting | Drug: Venetoclax, Azacitidine, Placebo | Myelodysplastic Syndrome (MDS) | 531 | 2020-09-10 | Jul 2027 | ≤ 345 d | 2026-05-06 |
| SLS | Sellas Life Sciences Group | $2.6B | Expanded Access Program (EAP) for Galinpepimut-S (GPS) in Patients Diagnosed With AML or MDSNCT05593185expanded access | — | Available | Biological: Galinpepimut-S | Acute Myeloid Leukemia, Myelodysplastic Syndromes | — | — | — | — | 2026-04-24 |
| BMY | Bristol-Myers Squibb | $134.9B | Study of BMS-986497 (ORM-6151) as a Monotherapy, in Double and Triple Combination With Azacitidine and Venetoclax in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic SyndromeNCT06419634Incidence of dose-limiting toxicities (DLTs) | Phase 1 | Recruiting | Drug: BMS-986497, Azacitidine, Venetoclax | Acute Myeloid Leukemia, Myelodysplastic Syndrome | 105 | 2024-05-29 | 2027-02-01 | in 165 d | 2026-04-08 |
| INCY | Incyte Corporation | $24.7B | Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid NeoplasmsNCT04279847Number of treatment-emergent adverse events | Phase 1 | Active, not recruiting | Drug: INCB057643, Ruxolitinib | Myelofibrosis, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasm Overlap Syndrome, Myeloproliferative Neoplasm, Relapsed or Refractory Primary Myelofibrosis, Secondary Myelofibrosis (Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis), ET (Essential Thrombocythemia) | 140 | 2021-02-23 | 2027-04-30 | in 253 d | 2026-04-01 |
| INMB | Inmune Bio, Inc. | $59M | A Phase I Open-Label Dose Escalation Study of Intravenous INKmune In Patients With MDS or AMLNCT05933070Primary Objective 1 | Phase 1 | Terminatedsponsor's stated reason: Slow/low Enrollment | Biological: INKmune | Cancer, MDS-EB, Myelodysplastic Syndromes, Acute Myeloid Leukemia, AML | 2 | 2020-06-15 | 2024-03-28actual | — | 2026-03-30 |
| CRIS | Curis, Inc. | $22M | Dose Escalation/ Expansion Study of CA-4948 as Monotherapy in Patients With Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)NCT04278768Determine Maximum Tolerated Dose (MTD) of emavusertib (CA-4948) monotherapy (Phase 1) | Phase 1/2 | Suspendedsponsor's stated reason: Dosing completed in all cohorts. Planning underway for potential additional patient cohorts. | Drug: Emavusertib, Venetoclax, Emavusertib | Acute Myelogenous Leukemia, Myelodysplastic Syndrome | 366 | 2020-07-06 | 2026-04-01 | — | 2026-03-19 |
| CDNA | CareDx | $2.4B | Assessment of Chimerism and Relapse Post Bone Marrow/Hematopoietic Cell Transplant (HCT) Using AlloHeme TestNCT04635384observationalTo determine the association between increased mixed chimerism (iMC) detected by AlloHeme and post-transplant relapse in patients with acute leukemia and myelodysplastic syndrome. | — | Active, not recruiting | — | Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome | 307 | 2021-06-30 | 2026-05-31 | — | 2026-03-18 |
| STTK | Shattuck Labs, Inc. | $748M | Phase 1 Study of Shattuck Labs (SL)-172154 in Subjects With MDS or AMLNCT05275439results2026-03-18Safety Profile of SL-172154 Monotherapy or in Combination With Azacitidine | Phase 1 | Terminatedsponsor's stated reason: Development discontinued | Drug: SL-172154, Azacitidine (AZA), Venetoclax | Acute Myeloid Leukemia, Myelodysplastic Syndromes | 106 | 2022-03-17 | 2025-02-06actual | — | 2026-03-18 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)NCT05469737Number of participants with Adverse Events (AEs) evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria v.5.0 | Phase 2/3 | Active, not recruiting | Drug: Oral Azacitidine, Placebo for Oral Azacitidine | Myelodysplastic Syndromes | 230 | 2022-12-14 | 2028-07-31 | in 711 d | 2026-02-25 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United StatesNCT06851065observationalParticipant baseline demographics | — | Completed | Drug: Luspatercept, Erythropoiesis-stimulating agents | Myelodysplastic Syndromes | 418 | 2024-08-22 | 2025-03-10actual | — | 2026-02-17 |
| BMY | Bristol-Myers Squibb | $134.9B | Asia Myelodysplastic Syndrome (MDS) RegistryNCT07355478observationalParticipant age | — | Not yet recruiting | Drug: Prescribed treatments for myelodysplastic syndromes (MDS) | Myelodysplastic Syndromes (MDS) | 500 | 2026-03-26 | 2026-10-31 | in 72 d | 2026-02-13 |
| PRLD | Prelude Therapeutics | $459M | A Study of PRT2527 as Monotherapy and in Combination With Zanubrutinib or Venetoclax in Participants With R/R Hematologic MalignanciesNCT05665530Dose limiting toxicity (DLT) of PRT2527 | Phase 1 | Completed | Drug: PRT2527, Zanubrutinib, Venetoclax | Aggressive B-Cell Non-Hodgkin's Lymphoma (NHL), Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Mantle Cell Lymphoma (MCL), Richter's Syndrome, T-cell Lymphoma, Diffuse Large B-cell Lymphoma (DLBCL), Marginal Zone Lymphoma, Myeloid Malignancies, Acute Myeloid Leukemia (AML), Chronic Myelomonocytic Leukemia (CMML), Myelodysplastic Syndrome (MDS), MDS/Myeloproliferative Neoplasm (MPN) Overlap Syndrome | 86 | 2023-09-12 | 2025-06-12actual | — | 2026-01-26 |
| CGEM | Cullinan Therapeutics Inc. | $1.3B | CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)NCT05143996Number of treatment emergent events (TEAEs) | Phase 1 | Recruiting | Drug: CLN-049 | Relapsed/Refractory Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) | 60 | 2021-11-18 | Jan 2027 | ≤ 164 d | 2026-01-16 |
| MRKR | Marker Therapeutics, Inc. | $19M | Safety of MT-401-OTS in Patients With Relapsed AML or MDSNCT06552416Safety of MT-401-OTS | Phase 1 | Recruiting | Drug: MT-401-OTS | Acute Myeloid Leukemia, in Relapse, MDS | 40 | 2025-06-16 | Sep 2029 | ≤ 1,137 d | 2025-12-16 |
| INCY | Incyte Corporation | $24.7B | To Assess the Safety and Tolerability of INCB000928 in Participants With Myelodysplastic Syndromes or Multiple MyelomaNCT04582539results2025-10-22Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | Phase 1/2 | Terminatedsponsor's stated reason: Strategic Business Decision | Drug: INCB000928 | Myelodysplastic Syndromes, Multiple Myeloma, Anemia | 22 | 2021-08-19 | 2024-08-15actual | — | 2025-10-22 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Assess the Effect of CC-95251 in Participants With Acute Myeloid Leukemia and Myelodysplastic SyndromesNCT05168202results2025-09-25Number of Participants With DLTs | Phase 1 | Terminatedsponsor's stated reason: Business objectives have changed | Drug: CC-95251, Azacitidine, Venetoclax | Leukemia, Myeloid, Acute, Myelodysplastic Syndromes | 56 | 2022-01-19 | 2024-07-30actual | — | 2025-09-25 |
| TCRX | TScan Therapeutics, Inc. | $107M | A Long Term Follow-up Study of TScan TCR-T ProductsNCT06976736observationalIncidence of Treatment-Emergent Adverse Events (Safety and Tolerability) | — | Recruiting | Other: Observational | AML, ALL, MDS | 1,000 | 2025-09-09 | Sep 2040 | ≤ 5,155 d | 2025-09-10 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate Treatment Patterns, Outcomes, and Healthcare Resource Utilization in Patients With Lower-Risk Myelodysplastic Syndromes Treated With LuspaterceptNCT07075406observationalParticipant baseline demographics | — | Completed | Drug: Luspatercept | Myelodysplastic Syndromes | 250 | 2024-11-01 | 2025-08-01actual | — | 2025-08-22 |
| VOR | Vor Biopharma | $1.4B | Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDSNCT04849910Incidence of neutrophil engraftment | Phase 1/2 | Terminatedsponsor's stated reason: Lack of Funding | Biological: VOR33 · Drug: Mylotarg | Leukemia, Myeloid, Acute, Myelodysplastic Syndromes | 67 | 2021-12-16 | 2025-05-28actual | — | 2025-07-29 |
| FHTX | Foghorn Therapeutics Inc. | $401M | FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic MalignanciesNCT04891757Incidence of AEs, dose-limiting toxicities (DLTs), serious AEs (SAEs), AEs leading to discontinuation, and adverse events of special interest (AESIs); safety laboratory assessments | Phase 1 | Active, not recruiting | Drug: FHD-286, Low Dose Cytarabine, Decitabine | Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Relapsed Myelodysplastic Syndromes, Refractory Myelodysplastic Syndromes, Relapsed Chronic Myelomonocytic Leukemia, Refractory Chronic Myelomonocytic Leukemia | 144 | 2021-06-14 | Sep 2025 | — | 2025-07-10 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate Treatment Patterns and Effectiveness of LuspaterceptNCT06971185observationalParticipant baseline demographics | — | Active, not recruiting | Drug: Luspatercept, Erythropoiesis-stimulating agent (ESA) | Myelodysplastic Syndromes (MDS) | 430 | 2024-11-22 | 2025-08-01 | — | 2025-05-14 |
| APRE | Aprea Therapeutics | $8M | APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)NCT03745716results2022-07-12Complete Response Rate (CR) | Phase 3 | Completed | Drug: APR-246 + azacitidine, Azacitidine | MDS | 154 | 2019-01-11 | 2020-11-27actual | — | 2025-03-18 |
| APRE | Aprea Therapeutics | $8M | APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS)NCT04638309results2025-03-07To Investigate the "Number of Participants With Treatment Emergent Adverse Events From Treatment With APR-548 as Monotherapy and in Combination With Azacitidine | Phase 1 | Terminatedsponsor's stated reason: Sponsor decision | Drug: APR-548 + Azacitidine | MDS, Myelodysplastic Syndromes | 4 | 2021-09-20 | 2022-04-25actual | — | 2025-03-10 |
| APTOF | Aptose Biosciences Inc. | — | A Study of CG-806 in Patients With Relapsed or Refractory AML or Higher-Risk MDSNCT04477291Incidence of treatment-emergent adverse events of CG-806 | Phase 1 | Terminatedsponsor's stated reason: Change in corporate strategy | Drug: CG-806 | Acute Myeloid Leukemia, Myelodysplastic Syndromes | 45 | 2020-10-06 | 2024-04-15actual | — | 2025-03-07 |
| NKTX | Nkarta, Inc. | $173M | NKX101, Intravenous Allogeneic CAR NK Cells, in Adults With AML or MDSNCT04623944Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | Phase 1 | Active, not recruiting | Biological: NKX101 - CAR NK cell therapy | Relapsed/Refractory AML, AML, Adult, MDS, Refractory Myelodysplastic Syndromes | 61 | 2020-09-21 | 2024-10-02actual | — | 2024-12-27 |
| PGEN | Precigen, Inc | $2.4B | PRGN-3006 Adoptive Cellular Therapy for CD33-Positive Relapsed or Refractory AML, MRD Positive AML or Higher Risk MDSNCT03927261Number of Participants who Experience Dose Limiting Toxicities (DLTs) | Phase 1 | Active, not recruiting | Drug: PRGN-3006 T Cells | Acute Myeloid Leukemia, Myelodysplastic Syndromes | 88 | 2019-05-20 | 2024-08-01actual | — | 2024-11-08 |
| BMY | Bristol-Myers Squibb | $134.9B | A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemiaNCT06073860observationalNumber of participants with adverse events (AEs) | — | Recruiting | Drug: Luspatercept | Myelodysplastic Syndrome, Beta Thalassemia | 104 | 2024-03-25 | 2027-12-21 | in 488 d | 2024-11-06 |
| 4503.T | Astellas Pharma Inc | $26.0B | Cytomegalovirus (CMV) Vaccine in Donors and Recipients Undergoing Allogeneic Hematopoietic Cell Transplant (HCT)NCT00285259Safety of CMV immunotherapeutic vaccine in donors and recipients undergoing HCT | Phase 2 | Completed | Biological: VCL-CB01 · Other: Phosphate-buffered Saline (PBS) | Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Acute Myelogenous Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Myelodysplastic Syndrome | 112 | 2006-01-31 | 2009-11-30actual | — | 2024-10-21 |
| AAPG | Ascentage Pharma Group Inc. | — | A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.NCT04275518Dose Limiting Toxicities (DLT) | Phase 1 | Recruiting | Drug: APG-115, Azacitidine, Cytarabine | Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS) | 102 | 2020-07-06 | 2025-12-31 | — | 2024-10-15 |
| BMY | Bristol-Myers Squibb | $134.9B | A Retrospective Study to Describe Real-World Treatment Patterns and Clinical Outcomes Among Patients With Myelodysplastic SyndromesNCT06581055observationalParticipant treatment patterns: Type of treatment received | — | Withdrawnsponsor's stated reason: Sponsor decision | Drug: Luspatercept, Epoetin Alfa | Myelodysplastic Syndromes (MDS) | 0 | 2024-11-30 | 2025-03-30 | — | 2024-10-09 |
| AMGN | Amgen | $229.9B | A Phase 1 Study of AMG 330 in Subjects With Myeloid MalignanciesNCT02520427results2024-07-31Number of Participants Who Experienced a Dose-limiting Toxicity (DLT) | Phase 1 | Terminatedsponsor's stated reason: Amgen strategic business decision to cancel following portfolio review, re-prioritization. | Drug: AMG 330 | Relapsed/Refractory AML, Minimal Residual Disease Positive AML, Myelodysplastic Syndrome | 95 | 2015-08-31 | 2022-01-09actual | — | 2024-07-31 |
| KURA | Kura Oncology, Inc. | $1.1B | Tipifarnib in Subjects With Myelodysplastic SyndromesNCT02779777results2024-07-31Objective Response Rate (ORR) | Phase 2 | Terminatedsponsor's stated reason: Lack of efficacy | Drug: Tipifarnib | Myelodysplastic Syndromes | 16 | 2016-06-01 | 2018-08-28actual | — | 2024-07-31 |
| GILD | Gilead Sciences | $177.9B | Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS)NCT04313881results2024-03-21Percentage of Participants With Complete Remission (CR) | Phase 3 | Terminatedsponsor's stated reason: Study discontinued due to futility based on a planned analysis | Drug: Magrolimab, Azacitidine, Placebo | Myelodysplastic Syndromes | 539 | 2020-09-09 | 2023-09-13actual | — | 2024-06-27 |
| GERN | Geron Corporation | $983M | Expanded Access for Treatment With ImetelstatNCT05937568expanded access | — | Approved for marketing | Drug: Imetelstat | Myelodysplastic Syndromes | — | — | — | — | 2024-06-24 |
| 4151.T | Kyowa Kirin, Inc. | $8.4B | An Ascending Dose Study of KW-2449 in Acute Leukemias, Myelodysplastic Syndromes, and Chronic Myelogenous LeukemiaNCT00346632results2016-08-12Safety of KW-2449 - Number of Participants With Treatment-related Adverse Events (TEAEs) as Assessed by NCI-CTCAE v3.0 | Phase 1 | Terminatedsponsor's stated reason: Terminated due to suboptimal dosing schedule | Drug: KW-2449 | Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Chronic Myelogenous Leukemia | 37 | Jun 2006 | Apr 2008actual | — | 2024-05-17 |
| 4151.T | Kyowa Kirin, Inc. | $8.4B | Study of KHK2823 in Patients With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)NCT02181699Number of Participants with Adverse Events as a Measure of Safety and Tolerability. | Phase 1 | Terminatedsponsor's stated reason: Failed treatment response | Drug: KHK2823 | Acute Myeloid Leukemia, Myelodysplastic Syndrome | 39 | Jun 2014 | 2019-05-10actual | — | 2024-04-29 |
| ABBV | AbbVie | $457.5B | Study to Evaluate Adverse Events and Movement of Lemzoparlimab in Body When Used Intravenously (IV) With Azacitidine Subcutaneously or IV and Venetoclax Orally in Participants With Acute Myeloid Leukemia and With Azacitidine With or Without Venetoclax in Participants With Myelodysplastic SyndromeNCT04912063Dose Limiting Toxicities (DLTs) of Lemzoparlimab (TJ011133) When Co-administered With Venetoclax and Azacitidine in Participants With Treatment-Naïve Acute Myeloid Leukemia (AML) Ineligible for Standard Induction Therapy | Phase 1 | Terminatedsponsor's stated reason: Strategic considerations | Drug: Lemzoparlimab, Azacitidine, Venetoclax | Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) | 40 | 2021-06-25 | 2023-05-09actual | — | 2024-02-26 |
| AAPG | Ascentage Pharma Group Inc. | — | A Study of APG-115 Alone or Combined With Azacitidine in Patients With AML, CMML, or MDSNCT04358393Maximum Tolerated Dose | Phase 1/2 | Unknown status | Drug: APG-115, 5-azacitidine | AML, Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, CMML, Myelodysplastic Syndromes, High-risk Myelodysplastic Syndrome, MDS | 69 | 2020-12-04 | 2024-12-30 | — | 2024-02-01 |
| PRLD | Prelude Therapeutics | $459M | PRT1419 as Monotherapy or in Combination With Azacitidine or Venetoclax in R/R Myeloid or B-cell MalignanciesNCT05107856Dose limiting toxicities (DLT) of PRT1419 | Phase 1 | Terminatedsponsor's stated reason: Company Decision | Drug: PRT1419, Azacitidine, Venetoclax | Acute Myeloid Leukemia, B-cell Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia, Chronic Myelomonocytic Leukemia, Follicular Lymphoma, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Small Lymphocytic Lymphoma | 21 | 2022-03-22 | 2024-01-19actual | — | 2024-01-31 |
| PFE | Pfizer | $155.3B | A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML PatientsNCT02367456results2021-03-05Number of Participants With Treatment-Emergent Adverse Events (TEAEs) in the Lead-in Cohort (LIC) | Phase 1 | Completed | Drug: PF-04449913 (Glasdegib), Azacitidine | Myelodysplastic Syndrome, Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia | 73 | 2015-04-28 | 2020-01-29actual | — | 2024-01-19 |
| SLN | Silence Therapeutics plc | $2.0B | A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic SyndromeNCT04718844Incidence of treatment-emergent adverse events | Phase 1 | Completed | Drug: SLN124, Placebo | Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome, Very-Low Risk Myelodysplastic Syndrome | 44 | 2021-04-14 | 2023-05-23actual | — | 2024-01-03 |
| PFE | Pfizer | $155.3B | Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMMLNCT04842604results2023-12-18Number of Participants With Treatment Emergent Adverse Event (AE) and Treatment Related AE | Phase 3 | Completed | Drug: Glasdegib, Azacitidine | Acute Myeloid Leukemia, Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia | 14 | 2021-05-17 | 2022-12-02actual | — | 2023-12-18 |
| 4523.T | Eisai Inc. | $8.7B | Escalation Study to Determine Bioavailability of a Single Oral Dose of Decitabine in Patients With Myelodysplastic Syndrome (MDS)NCT00941109Pharmacokinetic (PK) endpoints will be decitabine PK parameters: Tmax, Cmax, AUC0-inf, t1/2 and F. | Phase 1 | Completed | Drug: decitabine, decitabine, decitabine, decitabine | Myelodysplastic Syndrome | 12 | Dec 2009 | Sep 2011actual | — | 2023-06-18 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study of Azacitidine in Participants With International Prognostic Scoring System (IPSS) Intermediate-2 and High-risk Myelodysplastic Syndromes (MDS), or Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML)NCT05218902observationalIncidence of Adverse Events (AEs) | — | Completed | — | Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Acute | 290 | 2022-02-25 | 2023-03-20actual | — | 2023-05-31 |
| ABBV | AbbVie | $457.5B | A Study Evaluating Venetoclax Alone and in Combination With Azacitidine in Participants With Relapsed/Refractory Myelodysplastic Syndromes (MDS)NCT02966782AUCt for azacitidine | Phase 1 | Completed | Drug: venetoclax, azacitidine | Myelodysplastic Syndromes (MDS) | 70 | 2017-03-07 | 2023-04-05actual | — | 2023-05-09 |
| KYNB | Kyntra Bio | $28M | Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)NCT03303066Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion | Phase 2/3 | Completed | Drug: FG-4592, Placebo | Anemia, Myelodysplastic Syndrome | 43 | 2018-06-06 | 2023-01-11actual | — | 2023-05-03 |
| AMGN | Amgen | $229.9B | Safety, Tolerability, Pharmacokinetics and Efficacy of AMG 397 in Subjects With Selected Relapsed or Refractory Hematological MalignanciesNCT03465540results2023-04-12Number of Participants Who Experienced a Dose-limiting Toxicity (DLT) | Phase 1 | Terminatedsponsor's stated reason: Strategic decisions | Drug: AMG 397, Dexamethasone, Azacitidine | Multiple Myeloma, Acute Myeloid Leukemia, Non-Hodgkins Lymphoma, Myelodysplastic Syndrome, AML, MDS, NHL | 24 | 2018-08-17 | 2019-07-25actual | — | 2023-04-12 |
| FATE | Fate Therapeutics | $307M | Study of ProTmune for Allogeneic HCT in Adult Patients With Hematologic MalignanciesNCT02743351results2023-02-08Percentage of Participants With Cumulative Incidence of Grade II to IV aGvHD Through Day +100 Based on Investigator Assessment | Phase 1/2 | Completed | Biological: ProTmune, Control Arm | Hematologic Malignancies, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Chronic Myelogenous Leukemia, Acute Graft-versus-host Disease | 96 | 2016-12-20 | 2020-12-04actual | — | 2023-02-08 |
| KPTI | Karyopharm Therapeutics Inc | $44M | Efficacy & Safety Study of KPT-330 in Erythropoietin-Refractory Lower-Risk Myelodysplastic Syndrome PatientsNCT02431351Hematologic improvement (based on the 2006 International Working Group (IWG) response criteria) | Phase 2 | Withdrawnsponsor's stated reason: Trial never opened. | Drug: Selinexor | Myelodysplastic Syndrome | 0 | Nov 2015 | Dec 2017 | — | 2023-01-26 |
| PRLD | Prelude Therapeutics | $459M | A Study of PRT1419 in Patients With Relapsed/Refractory Hematologic MalignanciesNCT04543305To describe dose limiting toxicities (DLT) of PRT1419 | Phase 1 | Completed | Drug: PRT1419 | Multiple Myeloma, Acute Myeloid Leukemia, Non Hodgkin Lymphoma, Myelodysplastic Syndromes | 16 | 2020-09-28 | 2022-03-21actual | — | 2022-11-15 |
| AMGN | Amgen | $229.9B | Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)NCT00614523results2013-08-13Number of Clinically Significant Bleeding Events | Phase 2 | Completed | Drug: Placebo · Biological: Romiplostim | MDS, Myelodysplastic Syndromes, Thrombocytopenia | 250 | 2008-07-21 | 2011-03-31actual | — | 2022-11-07 |
| TRAW | Traws Pharma, Inc. | $8M | Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMANCT02562443Overall survival of all randomized patients and overall survival of patients scored as IPSS-R very high risk. | Phase 3 | Terminatedsponsor's stated reason: Top line analysis indicated that the study had failed to achieve its primary endpoint. | Drug: rigosertib, Any approved or standard-of-care therapy, best supportive care (BSC), best supportive care (BSC) | Myelodysplastic Syndrome, MDS, Refractory Anemia With Excess Blasts, RAEB | 372 | 2015-12-02 | 2020-07-26actual | — | 2022-09-26 |
| 4568.T | Daiichi Sankyo | $31.7B | Safety, Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute Myelogenous Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS)NCT02319369results2021-10-11Number of Participants With Dose-Limiting Toxicities (DLTs) Following Administration of Milademetan Alone and In Combination With 5-Azacitidine (AZA) | Phase 1 | Terminatedsponsor's stated reason: This study was terminated based on a business decision by the Sponsor. | Drug: Milademetan, AZA, Milademetan | Acute Myelogenous Leukemia, Myelodysplastic Syndrome | 74 | 2014-11-25 | 2020-08-21actual | — | 2021-10-11 |
| GILD | Gilead Sciences | $177.9B | Phase I Study of Milatuzumab for Graft Versus Host DiseaseNCT01663766All patients administered any dose of study drug will be included in the evaluation of safety | Phase 1 | Terminatedsponsor's stated reason: PIs agreed no safety signals were shown, drug did not appear to lessen the risk of preventing GVHD no further patients would be enrolled | Drug: milatuzumab | GVHD (Acute or Chronic), Acute Myeloid or Lymphoblastic Leukemia (AML or ALL), Myelodysplastic Syndrome, Chronic Myelogenous Leukemia (CML), Multiple Myeloma (MM), Non-Hodgekin Lymphoma (NHL-both Follicular & Diffuse Large Cell), Chronic Lymphocytic Leukemia or Small Lymphocytic Leukemia (CLL or SLL) | 12 | Dec 2013 | Mar 2015actual | — | 2021-08-19 |
| JAZZ | Jazz Pharmaceuticals | $16.4B | A Trial to Evaluate the Potential Impact of Renal Impairment on the Pharmacokinetics and Safety of CPX-351NCT03555955Pharmacokinetics (PK) of CPX-351 | Phase 1 | Completed | Drug: CPX-351, CPX-351 | Hematologic Malignancy, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes | 21 | 2018-11-20 | 2021-05-26actual | — | 2021-07-22 |
| TRAW | Traws Pharma, Inc. | $8M | Oral Rigosertib in Low Risk MDS Patients Refractory to ESAsNCT01904682Hematologic Improvement | Phase 2 | Completed | Drug: Oral rigosertib | Myelodysplastic Syndromes | 45 | Jul 2013 | Dec 2020actual | — | 2021-06-16 |
| TRAW | Traws Pharma, Inc. | $8M | Phase II Part 2 Expansion of Oral Rigosertib in Combination With AzacitidineNCT01926587Dose escalation part of study: Number of patients in whom Dose a Limiting Toxicity (DLT) are observed | Phase 1/2 | Completed | Drug: oral rigosertib, Azacitidine | Myelodysplastic Syndrome, Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia | 45 | Aug 2013 | 2020-12-08actual | — | 2021-06-16 |
| CYAD.BR | Celyad Oncology SA | — | DEPLETHINK - LymphoDEPLEtion and THerapeutic Immunotherapy With NKR-2NCT03466320The occurrence of Dose-limiting toxicities (DLT) during the study treatment until 3 weeks after first NKR-2 study treatment administration. | Phase 1/2 | Completed | Biological: NKR-2 | AML, MDS | 21 | 2018-09-18 | 2021-02-01actual | — | 2021-05-04 |
| PFE | Array Biopharma, now a wholly owned subsidiary of Pfizer | $155.3B | A Study of ARRY-614 in Patients With Low or Intermediate-1 Risk Myelodysplastic SyndromeNCT00916227Characterize the safety profile of the study drug, in either the fasted or fed state, in terms of adverse events, clinical laboratory tests and electrocardiograms. | Phase 1 | Completed | Drug: ARRY-614, p38/Tie2 inhibitor; oral | Myelodysplastic Syndromes | 45 | Jun 2009 | Jan 2012actual | — | 2020-09-09 |
| PFE | Array Biopharma, now a wholly owned subsidiary of Pfizer | $155.3B | A Study of ARRY-614 in Patients With Low or Intermediate-1 Risk Myelodysplastic SyndromesNCT01496495Establish the maximum tolerated dose (MTD) of study drug. | Phase 1 | Completed | Drug: ARRY-614, p38/Tie2 inhibitor; oral | Myelodysplastic Syndromes | 71 | Jan 2012 | Dec 2014actual | — | 2020-09-09 |
| TRAW | Traws Pharma, Inc. | $8M | Three Dosing Schedules of Oral Rigosertib in MDS PatientsNCT02075034Micrograms of rigosertib per milliliter of plasma | Phase 1 | Withdrawn | Drug: rigosertib | Myelodysplastic Syndrome | 0 | May 2014 | Aug 2019 | — | 2020-07-01 |
| TRAW | Traws Pharma, Inc. | $8M | Randomized Study of ON 01910.Na in Refractory Myelodysplastic Syndrome Patients With Excess BlastsNCT01241500Overall survival | Phase 3 | Completed | Drug: ON 01910.Na | Myelodysplastic Syndromes, MDS, RAEB, Chronic Myelomonocytic Leukemia | 299 | Nov 2010 | 2018-10-03actual | — | 2020-06-30 |
| TRAW | Traws Pharma, Inc. | $8M | Efficacy and Safety of IV Rigosertib in MDS Patients With Excess Blasts Progressing After Azacitidine or DecitabineNCT01928537Relationship of bone marrow blast response and overall survival. | Phase 3 | Completed | Drug: rigosertib sodium | Myelodysplastic Syndromes, Refractory Anemia With Excess Blasts, Chronic Myelomonocytic Leukemia, Cytopenia | 67 | Aug 2013 | 2017-06-29actual | — | 2020-06-30 |
| CYAD.BR | Celyad Oncology SA | — | Study in Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Patients to Determine the Recommended Dose of CYAD-02NCT04167696Occurrence of Dose Limiting Toxicities as defined per protocol in order to define the final recommended dose. | Phase 1 | Recruiting | Biological: CYAD-02 · Drug: ENDOXAN, Fludara | Acute Myeloid Leukemia, Myelodysplastic Syndrome | 27 | 2019-11-25 | Dec 2021 | — | 2020-06-09 |
| BPTH | Bio-Path Holdings, Inc. | $2M | Clinical Trial of BP1001 (L-Grb-2 Antisense Oligonucleotide) in CML, AML, ALL & MDSNCT01159028Safety of BP1001 | Phase 1 | Completed | Drug: BP1001, BP1001 in combination with LDAC | Recurrent Adult Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Ph1 Positive CML | 60 | Jun 2010 | 2017-03-30actual | — | 2020-05-28 |
| BPTH | Bio-Path Holdings, Inc. | $2M | Clinical Trial of BP1001 (Liposomal Grb2 Antisense Oligonucleotide) in Combination With Dasatinib in Patients With Ph + CML Who Have Failed TKI, Ph+ AML, Ph+ MDSNCT02923986Dose Limiting Toxicity of BP1001 using non-hematologic and hematologic parameters per NCI CTCAE criteria | Phase 1/2 | Withdrawnsponsor's stated reason: No enrollment | Drug: BP1001 (varying dose), BP1001 (fixed dose), Dasatinib | Chronic Myelogenous Leukemia, Ph1-Positive, Acute Myeloid Leukemia, Myelodysplastic Syndrome | 0 | 2017-09-01 | 2020-05-27actual | — | 2020-05-28 |
| 4568.T | Daiichi Sankyo | $31.7B | A Phase I Study of AC220 in Patients With Relapsed/Refractory Acute Myeloid Leukemia Regardless of FLT3 StatusNCT00462761results2020-04-24Number of Participants With Treatment-Emergent Treatment Related Adverse Events Following Oral Administration of Quizartinib in Participants With Relapsed or Refractory Acute Myeloid Leukemia | Phase 1 | Completed | Drug: AC220 | Acute Myeloid Leukemia, Leukemia, Myelodysplastic Syndrome, AML, MDS | 76 | Jan 2007 | Mar 2009actual | — | 2020-05-11 |
| SLN | Silence Therapeutics plc | $2.0B | Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124NCT04176653# of participants with all AEs as assessed by CTCAE V4.0 included injection site reaction, will be measured from baseline to post dose follow up | Phase 1 | Withdrawnsponsor's stated reason: Study stopped prior to subject administered first dose, due to COVID-19 | Drug: SLN124 is a GalNAc conjugated double stranded fully modified siRNA. Sodium chloride 0.9% w/v is used as Placebo | Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome | 0 | 2019-08-20 | 2021-09-30 | — | 2020-04-27 |
| CYAD.BR | Celyad Oncology SA | — | EPITHINK: Epigenetic Drug Treatment and Therapeutic Immunotherapy With NKR-2NCT03612739The occurrence of Dose Limiting toxicities (DLT) during the study treatment until 6 months after initial treatment. | Phase 1 | Withdrawnsponsor's stated reason: Sponsor decision | Biological: NKR-2 CAR-T Cells | AML, Adult, Myelodysplastic Syndromes | 0 | Dec 2018 | May 2019 | — | 2019-10-29 |
| LLY | Eli Lilly and Company | $1.15T | A Study of Galunisertib in Participants With Myelodysplastic SyndromesNCT02008318results2019-03-07Percentage of Participants With Hematological Improvement (HI) | Phase 2/3 | Completed | Drug: Galunisertib, Placebo | Myelodysplastic Syndromes | 43 | Mar 2014 | Mar 2016actual | — | 2019-09-11 |
| LIXT | Lixte Biotechnology Holdings, Inc. | $50M | A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)NCT03886662For Phase Ib - Number of patients with adverse events related to the study treatment as a measure of safety and tolerability of LB-100 study drug | Phase 1/2 | Recruiting | Drug: LB-100 | Myelodysplastic Syndromes | 47 | Apr 2019 | May 2021 | — | 2019-04-08 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | Study of Orally Administered AG-881 in Patients With Advanced Hematologic Malignancies With an IDH1 and/or IDH2 MutationNCT02492737Safety/tolerability; incidence of adverse events | Phase 1 | Completed | Drug: AG881 | Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, Hematologic Malignancies | 46 | 2015-08-07 | 2018-03-21actual | — | 2019-03-08 |
| GILD | Gilead Sciences | $177.9B | CAMELLIA: Anti-CD47 Antibody Therapy in Haematological MalignanciesNCT02678338Maximum tolerated dose (MTD) of Hu5F9-G4, graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 (Part A) | Phase 1 | Completed | Drug: Hu5F9-G4 | Acute Myeloid Leukemia, Myelodysplastic Syndrome | 20 | Nov 2015 | Nov 2018actual | — | 2019-02-21 |
| AMGN | Amgen | $229.9B | Darbepoetin Alfa MDS Companion ProtocolNCT02175277results2018-10-11Number of Participants With Treatment-emergent Adverse Events | Phase 3 | Completed | Drug: Darbepoetin Alfa | Myelodysplastic Syndrome (MDS) | 9 | 2014-06-12 | 2017-03-20actual | — | 2018-11-14 |
| AMGN | Amgen | $229.9B | Determination of Safe and Effective Dose of Romiplostim (AMG 531) in Subjects With Myelodysplastic Syndrome (MDS)Receiving Hypomethylating AgentsNCT00321711results2011-04-05Occurrence of a Clinically Significant Thrombocytopenic Event | Phase 2 | Completed | Drug: Placebo, Azacitidine, Decitabine · Biological: AMG 531 (Romiplostim) | MDS, Myelodysplastic Syndromes, Thrombocytopenia | 69 | 2006-10-01 | 2009-10-19actual | — | 2018-10-17 |
| CYAD.BR | Celyad Oncology SA | — | Safety Study of Chimeric Antigen Receptor Modified T-cells Targeting NKG2D-LigandsNCT02203825Safety and feasibility of intravenous administration of CM-CS1 T-cells. | Phase 1 | Completed | Biological: CM-CS1 T-cell infusion | Acute Myeloid Leukemia, Myelodysplastic Syndrome, Multiple Myeloma | 12 | Mar 2015 | Mar 2018actual | — | 2018-06-01 |
| JAZZ | Jazz Pharmaceuticals | $16.4B | Pharmacokinetic and Pharmacodynamic Assessment of Treatment With CPX-351 (Cytarabine: Daunorubicin) Liposome for Injection in Acute Leukemias and MDS Patients With Moderate Hepatic ImpairmentNCT02269579Total Drug Plasma PK | Phase 2 | Withdrawn | Drug: CPX-351 | Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS) | 0 | Apr 2015 | Jun 2017 | — | 2018-03-14 |
| INCY | Incyte Corporation | $24.7B | Randomized Open-Label Study of INCB047986 in Subjects With Primary Myelodysplastic Syndrome (MDS)NCT02093429Proportion of subjects who achieve a response for Hematologic Improvement in Erythrocytes (HI-E) during any 8-week period within the first 16-week treatment period with INCB047986 Monotherapy. | Phase 1/2 | Terminatedsponsor's stated reason: Business decision. | Drug: INCB047986 | MDS (Myelodysplastic Syndrome) | 6 | Jan 2014 | Sep 2014actual | — | 2018-01-17 |
| TRAW | Traws Pharma, Inc. | $8M | Phase I Study of ON 01910.Na in Refractory Leukemia or Myelodysplastic Syndrome (MDS)NCT00854646Safety data, including laboratory parameters and adverse events, will be collected for all patients in order to determine the qualitative and quantitative toxicity, and reversibility of toxicity, of ON 01910.Na. | Phase 1 | Completed | Drug: ON 01910.Na | Acute Myelocytic Leukemia, Acute Lymphocytic Leukemia, Chronic Myelocytic Leukemia, Chronic Lymphocytic Leukemia, Myelodysplastic Syndromes | 22 | Oct 2008 | Nov 2014actual | — | 2018-01-10 |
| AMGN | Amgen | $229.9B | Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)NCT00472290results2013-07-15Overall Summary of Adverse Events | Not applicable | Completed | Drug: Romiplostim (formerly AMG 531) | Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia | 72 | 2007-04-01 | 2011-07-18actual | — | 2017-12-29 |
| AMGN | Amgen | $229.9B | Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)NCT01362140results2016-12-05Percentage of Participants With at Least One Red Blood Cell (RBC) Transfusion During the Double-blind Treatment Period | Phase 3 | Completed | Drug: Darbepoetin alfa, Placebo | MDS | 147 | 2011-12-21 | 2015-02-11actual | — | 2017-12-19 |
| JAZZ | Jazz Pharmaceuticals | $16.4B | An Open Label Phase II Pharmacokinetic and Pharmacodynamic Assessment of the Potential for QTc Prolongation Following First Induction Treatment With CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in Acute Leukemias and MDS PatientsNCT02238925results2017-11-30Effect of CPX-351 on Cardiac Ventricular Repolarization (QTcF) | Phase 2 | Completed | Drug: CPX-351 | Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS) | 26 | Jul 2014 | May 2015actual | — | 2017-11-30 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
How Myelodysplastic Syndromes is matched — 8 exact registry spellings
A study is on this page when its ClinicalTrials.gov record names one of these conditions exactly (letter case aside). Nothing is matched by substring or stem, so a longer condition that merely contains one of these words is a different page. The list was curated by hand and last measured 2026-08-19; a spelling filed by only one study was not curated, so the counts here are a floor rather than the registry's own total for the disease.
- Myelodysplastic Syndromes
- Myelodysplastic Syndrome
- MDS
- Myelodysplastic Syndrome (MDS)
- Myelodysplastic Syndromes (MDS)
- Low Risk Myelodysplastic Syndrome
- MDS (Myelodysplastic Syndrome)
- Refractory Myelodysplastic Syndromes
Permalink · All conditions · Catalyst calendar · Every registered study · Search the whole pipeline for “Myelodysplastic Syndromes” (a text search, wider than this page)