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

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.

Study Overview

• Status: Recruiting
• Conditions: Myelodysplastic Syndromes; Leukemia; Myeloproliferative Syndrome
• Intervention / Treatment: Genetic: Genomic profiling

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 Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

• Study Contact: Name: Yana Pikman, MD; Phone Number: (617) 632-4754; Email: yana_pikman@dfci.harvard.edu

Study Locations

• United States
  • Connecticut
    • Hartford, Connecticut, United States, 06106
      • Recruiting
      • Connecticut Children's Medical Center
      • Contact: Natalie Bezler, MD; Email: Nbezler@connecticutchildrens.org
  • Maine
    • Portland, Maine, United States, 04102
      • Recruiting
      • Maine Medical Center
      • Contact: Sei Sze, MD; Email: Sei.Sze@mainehealth.org
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Active, not recruiting
      • Dana Farber Cancer Institute
    • Boston, Massachusetts, United States, 02115
      • Active, not recruiting
      • Boston Children's Hospital
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Recruiting
      • Dartmouth-Hitchcock
      • Contact: Angela Ricci, MD; Email: angela.m.ricci@hitchcock.org
  • New York
    • Albany, New York, United States, 12208
      • Recruiting
      • Albany Medical Center
      • Contact: Jessica Geaney, MD; Email: geaneyj@amc.edu
  • Rhode Island
    • Providence, Rhode Island, United States, 02903
      • Recruiting
      • Lifespan Cancer Institute
      • Contact: Jennifer Welch, MD; Email: jwelch@lifespan.org
  • Vermont
    • Burlington, Vermont, United States, 05401
      • Recruiting
      • University of Vermont Medical Center
      • Contact: Jessica Heath, MD; Email: Jessica.Heath@uvmhealth.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 28 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

- birth to < 30 years of age. Patient with acute leukemia, chronic leukemia, MDS/AML, myelodysplastic syndrome or myeloproliferative syndromes. Disease can be newly diagnosed or relapsed/refractory.

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

GENOMIC PROFILING AND SPECIMEN BANKING REGISTRATION ARM; 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

Genetic: Genomic profiling; Genomic profiling using CLIA assay

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients Enrolled for Genomic Profiling-Pediatric Leukemia	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.	3 Years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients Enrolled for Genomic Profiling-New Diagnosis	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.	3 Years

Collaborators and Investigators

• Sponsor: Dana-Farber Cancer Institute
• Collaborators: Charles H. Hood Foundation
• Investigators: Principal Investigator: Yana Pikman, MD, Dana-Farber Cancer Institute

Study record dates

Study Major Dates

• Study Start (Actual): June 11, 2021
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: July 9, 2021
• First Submitted That Met QC Criteria: July 9, 2021
• First Posted (Actual): July 20, 2021

Study Record Updates

• Last Update Posted (Actual): November 1, 2023
• Last Update Submitted That Met QC Criteria: October 31, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Leukemia; Myelodysplastic Syndromes; Myeloproliferative syndrome (MPS)
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Neoplasms by Site; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Hematologic Neoplasms; Preleukemia
• Other Study ID Numbers: 20-302

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to Sponsor Investigator or designee. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
• IPD Sharing Time Frame: Data can be shared no earlier than 1 year following the date of publication
• IPD Sharing Access Criteria: Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No