Evaluation of Myelodysplastic Syndromes in Adults and Children
Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS
This study is trying to learn more about myelodysplastic syndromes by collecting health information and samples from both patients with the condition and healthy volunteers.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1100 (estimated) |
| Ages | 1 Day to 120 Years |
| Sex | All |
| Sponsor | National Institutes of Health Clinical Center (CC) NIH |
| Locations | 1 site (Bethesda, Maryland) |
| Trial ID | NCT05350748 on ClinicalTrials.gov |
What this trial studies
This observational study aims to understand the natural history of myelodysplastic syndromes (MDS) and related conditions by collecting clinical data and biological samples from participants. Individuals with suspected or confirmed MDS, as well as healthy donors, will undergo medical history screenings, physical exams, and provide blood and urine samples. Participants will also complete surveys regarding their symptoms and undergo bone marrow biopsies to gather comprehensive information about the disease. The goal is to elucidate the mechanisms driving MDS and identify potential biomarkers for diagnosis and treatment response.
Who should consider this trial
Good fit: Ideal candidates include individuals of any age with suspected or confirmed myelodysplastic syndromes or related conditions.
Not a fit: Patients with conditions unrelated to myelodysplastic syndromes or those who do not meet the eligibility criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved understanding and treatment options for patients with myelodysplastic syndromes.
How similar studies have performed: While there have been studies on MDS, this approach focusing on the natural history and chronic inflammation is novel and may provide new insights.
Eligibility criteria
Show full inclusion / exclusion criteria
* INCLUSION CRITERIA - MDS Participants * Either sex, any age. * Histologically or cytologically suspected or confirmed myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN), MDS/myeloproliferative neoplasm with ringed sideroblasts and thrombocytosis (MDS/MPN-RS-T), myelodysplastic syndromes/myeloproliferative neoplasms unclassified (MDS/MPN-U), chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), secondary acute myeloid leukemia (sAML) with antecedent MDS or MDS/MPN, or participants who have precursor conditions that are associated with a risk of progression to MDS, including but not limited to clonal hematopoiesis of indeterminate potential (CHIP) and clonal cytopenia of unknown significance (CCUS). * Participants may have had any amount of prior therapy and may be receiving MDS-directed therapy at time of enrollment. * Participants must have an identified primary oncologist, hematologist or generalist outside of NIH who agrees to manage participant care and any diagnostic findings provided by this study. INCLUSION CRITERIA - Marrow Control Donor Participants * Either sex, and must be eligible for marrow donation per NIH Clinical Center requirements. * No history of hematological malignancies as listed as inclusion in 'Inclusion Criteria - MDS Participants' or current autoimmune disease. * Must be scheduled for bone marrow harvest for clinical application (e.g., marrow donation); or, if being evaluated for malignancy, have a clinical bone marrow aspirate scheduled (e.g., to rule out bone marrow involvement). INCLUSION CRITERIA - All Participants * Stated willingness to comply with all study procedures and availability for the duration of the study. * Ability of participant or parent/guardian to understand and the willingness to sign a written consent document. EXCLUSION CRITERIA - All Participants -Uncontrolled intercurrent illness, psychiatric illness, or other that would limit compliance with study requirements, or at the investigator s discretion.
Where this trial is running
Bethesda, Maryland
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)
Study contacts
- Principal investigator: Kathy L McGraw, Ph.D. — National Cancer Institute (NCI)
- Study coordinator: Ashley E Carpenter
- Email: carpentera@mail.nih.gov
- Phone: (240) 550-0492
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.