Genomic profiling of blood and bone marrow samples from children and young adults with blood cancers
Official title: Protocol For Genomically Profiling, Collecting, Archiving and Distributing Blood and Bone Marrow Specimens From Children and Young Adults With Hematologic Malignancy
Run by Dana-Farber Cancer Institute · NCT04968834 · Data updated 2026-11-12
This study is testing how analyzing the genes in blood and bone marrow samples from kids and young adults with blood cancers can help doctors make better treatment decisions.
Quick facts
| Study type | Observational |
|---|---|
| Ages | Any age to 30 Years |
| Sex | All |
| Enrollment | 300 (estimated) |
| Sponsor | Dana-Farber Cancer Institute Academic / other |
| Locations | 8 sites (Hartford, Connecticut and 7 other locations) |
| Status | Recruiting |
| Started | June 11, 2021 |
| Main results expected | June 2033 |
| Study ends | June 2033 |
| Registry record updated | July 16, 2026 |
| Trial ID | NCT04968834 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include children and young adults under 30 years old with newly diagnosed or relapsed/refractory leukemia or related syndromes.
✕Probably not a fit
Patients without sufficient specimens for genomic profiling or those not undergoing planned clinical evaluations may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study focuses on genomic profiling and the collection of blood and bone marrow specimens from pediatric patients diagnosed with leukemia, myelodysplastic syndromes, or myeloproliferative syndromes. Participants will have their samples analyzed to understand the genetic factors contributing to their diseases, which may influence treatment decisions. The study aims to enroll approximately 100 patients annually across eight medical centers in the United States, utilizing samples obtained during clinical procedures. The genomic data collected will be shared with treating oncologists to potentially guide therapy.
Why it matters
Potential benefit: If successful, this study could lead to more personalized and effective treatment strategies for children and young adults with hematologic malignancies.
How similar studies have gone: Other studies utilizing genomic profiling in hematologic malignancies have shown promise, suggesting that this approach may yield valuable insights.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age: birth to \< 30 years of age * Diagnosis: \-- Patient with acute leukemia, chronic leukemia, MDS/AML, myelodysplastic syndrome or myeloproliferative syndromes. Disease can be newly diagnosed or relapsed/refractory. * Pathology Criteria: \-- Histologic confirmation of leukemia or myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPS) at the time of diagnosis or recurrence * Specimen Criteria: * Sufficient sample available for genomic profiling OR bone marrow aspirate/blood draw planned for clinical care which is anticipated to allow collection of minimum specimen for testing (See Section 6.1 for description of specimen requirements) Exclusion Criteria: \- Insufficient leukemia or MDS specimen available for profiling from diagnosis or recurrence (See Section 6.1); or bone marrow evaluations NOT planned for clinical care; or peripheral blast percentage \<20%, or clinical blood draw not planned
Where it's running
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Connecticut Children's Medical Center
Hartford, Connecticut, United States Recruiting -
Maine Medical Center
Portland, Maine, United States Recruiting -
Dartmouth-Hitchcock
Lebanon, New Hampshire, United States Recruiting -
Albany Medical Center
Albany, New York, United States Recruiting -
Lifespan Cancer Institute
Providence, Rhode Island, United States Recruiting -
University of Vermont Medical Center
Burlington, Vermont, United States Recruiting
Show 2 more locations
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Boston Children's Hospital
Boston, Massachusetts, United States Active not recruiting -
Dana Farber Cancer Institute
Boston, Massachusetts, United States Active not recruiting
Study contacts
- Principal investigator: Yana Pikman, MD — Dana-Farber Cancer Institute
- Study coordinator: Yana Pikman, MD
- Email: yana_pikman@dfci.harvard.edu
- Phone: (617) 632-4754
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.