The National Myelodysplastic Syndromes (MDS) Study

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Observational
Eligible Ages	18 Years and Over
Gender	All

More Inclusion & Exclusion Criteria

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)

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT02775383
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Mikkael Sekeres, MD, MSAmy DeZern, MD, MHS
Principal Investigator Affiliation	University of MiamiJohns Hopkins University
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	NIH
Overall Status	Recruiting
Countries	Israel, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Myelodysplastic Syndromes (MDS)
Study Website:	View Trial Website

Additional Details

Multi-center, prospective cohort study enrolling patients from centers in the National Cancer Institute (NCI) National Clinical Trials Network (NCTN) and NCI Community Oncology Research Program (NCORP). The accrual period is 5+ years. After central pathology review of registered participants, approximately 2,000 cases of MDS or MDS/MPN overlap disorders, and 500 cases of ICUS will be identified for the longitudinal study cohort and up to 1000 cases will be identified for the cross-sectional cohort. No more than 3500 total participants will be registered. Participants in the longitudinal cohort may be followed for life. The goal of the National MDS Study is to establish a publicly available resource to facilitate the study of MDS natural history. This will be accomplished through: 1) Creation of a multi-institutional, longitudinal collection of consistently processed and clinically well-annotated blood and tissue specimens collected prospectively from participants with MDS and participants with idiopathic cytopenia of undetermined significance (ICUS); and 2) Support for investigator-initiated studies of MDS that will have high-impact for MDS patients, including basic science, clinical, health outcomes and epidemiological research.

Arms & Interventions

Arms

: Longitudinal

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

: Cross-sectional

Participants who do not have MDS, MDS/MPN overlap disorder, or ICUS and have the baseline visit only

Interventions

Other: - Therapeutic

Observational Study

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Kaiser Permanente-Anaheim, Anaheim, California

Status

Recruiting

Address

Kaiser Permanente-Anaheim

Anaheim, California, 92806

Site Contact

Desiree Goldstein

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