Protocol for Genomically Profiling, Collecting, Archiving and Distributing Blood and Bone Marrow Specimens from Children and Young Adults with Hematologic Malignancy

Recruiting

• Conditions: Myelodysplastic Syndromes; Leukemia; Myeloproliferative Syndrome
• Interventions: Genetic: Genomic profiling

• Registration Number: NCT04968834
• Lead Sponsor: Dana-Farber Cancer Institute

Brief Summary

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.

Detailed Description

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

Study Timeline

• Study Start: 2021-06-11
• Study First Submitted: 2021-07-09
• Study First Submitted QC: 2021-07-09
• Study First Posted: 2021-07-20
• Status Verified: 2024-05
• Last Update Submitted: 2024-11-04
• Last Update Posted: 2024-11-05
• Primary Completion: 2033-06
• Study Completion: 2033-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
GENOMIC PROFILING AND SPECIMEN BANKING REGISTRATION ARM	Genomic profiling	The research study procedures include screening for eligibility, reviewing and signing this consent form, collecting patient information and clinical data, obtaining previously collected bone marrow and blood samples, and completing a brief optional Household Survey. Bone marrow and blood samples may also be collected in the future as part of your routine clinical procedures

Primary Outcome Measures

Name	Time	Method
Number of Patients Enrolled for Genomic Profiling-Pediatric Leukemia	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 Outcome Measures

Name	Time	Method
Number of Patients Enrolled for Genomic Profiling-New Diagnosis	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.

Trial Locations

Locations (8)

Dana Farber Cancer Institute; 🇺🇸 Boston, Massachusetts, United States

Connecticut Children's Medical Center; 🇺🇸 Hartford, Connecticut, United States

University of Vermont Medical Center; 🇺🇸 Burlington, Vermont, United States

Maine Medical Center; 🇺🇸 Portland, Maine, United States

Dartmouth-Hitchcock; 🇺🇸 Lebanon, New Hampshire, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Albany Medical Center; 🇺🇸 Albany, New York, United States

Lifespan Cancer Institute; 🇺🇸 Providence, Rhode Island, United States