Clinical trial · Observational
The National Myelodysplastic Syndromes (MDS) Study
The National Myelodysplastic Syndromes Natural History Study
NCT02775383CI-TRIAL-00102304MDScompletedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Participants will be followed long term. Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myelodysplastic Syndromes (MDS) | Myelodysplastic Syndrome | ALIAS | 0.85 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Therapeutic | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- label
- Longitudinal
- description
- Participants with MDS, MDS/MPN overlap disorder, AML \<30% blasts without core binding factor or acute promyelocytic leukemia, ICUS, or at risk based on select karyotypic or genetic abnormalities
- interventionNames
- Other: Therapeutic
- label
- Cross-sectional
- description
- Participants who do not have MDS, MDS/MPN overlap disorder, or ICUS and have the baseline visit only
- interventionNames
- Other: Therapeutic
Primary outcomes (1)
- measure
- Number of patients dying
- timeFrame
- Through study completion, an average of 3 years
- description
- Deaths will be reported on study case report forms
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Suspected (e.g., persistent unexplained cytopenia, circulating peripheral blasts etc.) MDS or MDS/MPN overlap disorders and undergoing diagnostic work-up with planned bone marrow assessments, or diagnosed with de novo or therapy-related MDS within 12 months of enrollment per the World Health Organization (WHO) criteria1 and undergoing clinical evaluation and planned bone marrow assessments to confirm MDS or to evaluate disease status * Bone marrow aspirate expected to be performed within 1 week of registration, and in all cases must be performed no later than 4 weeks after enrollment * Age 18 or older * If anemic without prior MDS diagnosis, B12 level, serum folate, mean corpuscular volume (MCV), red cell distribution width (RDW), ferritin, and iron studies (Iron, total iron-binding capacity (TIBC) test, and percent saturation) must be performed within prior 6 months. Exclusion Criteria: * Prior treatment for MDS at entry and through the time of the entry bone marrow aspirate * Treatment with hematopoietic growth factors in prior 6 months * Diagnosis of a solid tumor or hematologic malignancy within two years prior to enrollment except for in situ cancer of the skin (basal or squamous cell), cervix, bladder, breast, or prostate * Treatment with radiation therapy in the two years prior to registration * Non-hormonal treatment for malignancy within the two years prior to registration * Established hereditary bone marrow failure syndrome * Known primary diagnosis of aplastic anemia, classical paroxysmal nocturnal hemoglobinuria, amegakaryocytic thrombocytopenic purpura, or large granular lymphocyte leukemia * Enrolled in the Connect® MDS/AML Disease Registry (NCT01688011)
References
Publications (4)
- DERIVEDGillis N, Colin-Leitzinger C, Tang YH, Otterstatter M, Sherman S, Zhang L, Moscinski LC, Walker ME, Painter JS, Abel GA, Al Baghdadi T, Deeg HJ, Foran JM, Gore SD, Harrington AM, Kroft SH, Liu JJ, Saber W, Virani S, Bejar R, Lindsley RC, Padron E, Walter MJ, Komrokji R, DeZern AE, Sekeres MA. Clinical, Genetic, and Pathologic Variability in Myelodysplastic Syndromes and Precursor Conditions Across Race, Ethnicity, and Sex. Am J Hematol. 2026 Jun;101(6):1407-1420. doi: 10.1002/ajh.70279. Epub 2026 Mar 29. PMID 41906220
- DERIVEDDeZern AE, Gillis N, Otterstatter M, Abel G, Padron E, Deeg HJ, Al Baghdadi T, Liu J, Xie Z, Zhang L, Moscinski L, Kroft S, Harrington A, Foran J, Komrokji R, Starczynowski D, Gore S, Saber W, Bejar R, Lindsley RC, Sherman S, Lee C, DiFronzo N, Walter M, Sekeres MA. A novel approach to defining progression in MDS and precursor myeloid conditions in the MDS Natural History Study. Blood Adv. 2026 Apr 28;10(8):2743-2753. doi: 10.1182/bloodadvances.2025018770. PMID 41671442
- DERIVEDAbel GA, Hebert D, Lee C, Rollison D, Gillis N, Komrokji R, Foran JM, Liu JJ, Al Baghdadi T, Deeg J, Gore S, Saber W, Wilson S, Otterstatter M, Thompson J, Borchert C, Padron E, DeZern A, Cella D, Sekeres MA. Health-related quality of life and vulnerability among people with myelodysplastic syndromes: a US national study. Blood Adv. 2023 Jul 25;7(14):3506-3515. doi: 10.1182/bloodadvances.2022009000. PMID 37146263
- DERIVEDDeZern AE, Goll JB, Lindsley RC, Bejar R, Wilson SH, Hebert D, Deeg J, Zhang L, Gore S, Al Baghdadi T, Maciejewski J, Liu J, Padron E, Komrojki R, Saber W, Abel G, Kroft SH, Harrington A, Grimes T, Reed H, Fulton RS, DiFronzo NL, Gillis N, Sekeres MA, Walter MJ. Utility of targeted gene sequencing to differentiate myeloid malignancies from other cytopenic conditions. Blood Adv. 2023 Jul 25;7(14):3749-3759. doi: 10.1182/bloodadvances.2022008578. PMID 36947201