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NCT Number: NCT04968834

Genomically Profiling, Collecting, Archiving and Distributing Hematologic Malignancy Specimens

This research study is a genomic profiling and repository study for children and young adults who have leukemia, myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPS). Genes are the part of cells that contain the instructions which tell cells how to make the right proteins to grow and work. Genes are composed of DNA letters that spell out these instructions. Genomic profiling helps investigators understand why the disease develops and the instructions that led to its development. Understanding the genetic factors of the disease can also help investigator understand why the disease of some people can respond to certain therapies differently than others.

The genomic profiling will be performed using bone marrow and blood samples that either have already been obtained during a previous clinical procedure or will be obtained at the time of a scheduled clinical procedure. Studying the genetic information in the cells of these samples will provide information about the origin, progression, and treatment of leukemia and myeloproliferative syndromes and myelodysplastic syndrome. Storing the bone marrow and blood samples will allow for additional research and genomic assessments to be performed in the future.

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Key information

Conditions

Leukemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes Myeloproliferative Syndrome Neoplasms Neoplasms by Histologic Type

Age range

Up to 30 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Connecticut Children's Medical Center, Hartford, Connecticut, United States

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About this study

Pediatric patients with new diagnosis or relapsed/refractory acute leukemia, MDS/AML, chronic leukemia, myeloproliferative syndromes or myelodysplastic syndrome will be enrolled onto this study. At the time of enrollment, a sample of the leukemia will be submitted for genomic profiling using CLIA assay(s). This information will be returned to the treating oncologist. The study will collect follow up data on patient outcome and whether the genomic profiling influenced treatment.

It is expected that about 100 people each year will take part in this research study at 8 medical centers in the United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Genomic profiling

Genetic

Genomic profiling using CLIA assay

Primary outcomes

  1. Number of Patients Enrolled for Genomic Profiling-Pediatric Leukemia

    Time frame: 3 Years

    To perform genomic profiling of pediatric leukemia using clinical genomics platforms and return results to treating oncologist. This objective will be accomplished by enrolling patients and obtaining samples for sequencing and banking. The pathologist-interpreted genomic test results will be returned to the treating oncologist.

Secondary outcomes

  1. Number of Patients Enrolled for Genomic Profiling-New Diagnosis

    Time frame: 3 Years

    To collect and bank samples from pediatric patients with new diagnosis or relapsed/refractory acute and chronic leukemia, and myelodysplastic syndrome. This objective will be accomplished by enrolling patients and obtaining samples for sequencing and banking.

Study contacts

Contact information is provided by the study sponsor or research team.

Yana Pikman, MD

CONTACT

[email protected]

(617) 632-4754

Sponsors and collaborators

Lead sponsor

Dana-Farber Cancer Institute

Other

Collaborators

  • Charles H. Hood Foundation
  • Rally Foundation for Childhood Cancer Research

Registry information

Official study title

Protocol For Genomically Profiling, Collecting, Archiving and Distributing Blood and Bone Marrow Specimens From Children and Young Adults With Hematologic Malignancy

Important dates

Study start
2021
Primary completion
2033
Study completion
2033
First posted
Jul 20, 2021
Registry last updated
Jul 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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