NCT04968834

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
300

participants targeted

Target at P75+ for all trials

Timeline
86mo left

Started Jun 2021

Longer than P75 for all trials

Geographic Reach
1 country

8 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress41%
Jun 2021Jun 2033

Study Start

First participant enrolled

June 11, 2021

Completed
28 days until next milestone

First Submitted

Initial submission to the registry

July 9, 2021

Completed
11 days until next milestone

First Posted

Study publicly available on registry

July 20, 2021

Completed
11.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2033

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2033

Last Updated

June 12, 2025

Status Verified

June 1, 2025

Enrollment Period

12 years

First QC Date

July 9, 2021

Last Update Submit

June 9, 2025

Conditions

Keywords

LeukemiaMyelodysplastic SyndromesMyeloproliferative syndrome (MPS)

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (1)

  • Number of Patients Enrolled for Genomic Profiling-New Diagnosis

    3 Years

Study Arms (1)

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

Interventions

Genomic profiling using CLIA assay

GENOMIC PROFILING AND SPECIMEN BANKING REGISTRATION ARM

Eligibility Criteria

AgeUp to 30 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-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.

You may qualify if:

  • 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)

You may not qualify if:

  • \- 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

RECRUITING

Maine Medical Center

Portland, Maine, 04102, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

ACTIVE NOT RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

ACTIVE NOT RECRUITING

Dartmouth-Hitchcock

Lebanon, New Hampshire, 03756, United States

RECRUITING

Albany Medical Center

Albany, New York, 12208, United States

RECRUITING

Lifespan Cancer Institute

Providence, Rhode Island, 02903, United States

RECRUITING

University of Vermont Medical Center

Burlington, Vermont, 05401, United States

RECRUITING

MeSH Terms

Conditions

LeukemiaMyelodysplastic Syndromes

Interventions

Genetic Profile

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Diseases

Intervention Hierarchy (Ancestors)

Genetic BackgroundGenetic Phenomena

Study Officials

  • Yana Pikman, MD

    Dana-Farber Cancer Institute

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

July 9, 2021

First Posted

July 20, 2021

Study Start

June 11, 2021

Primary Completion (Estimated)

June 1, 2033

Study Completion (Estimated)

June 1, 2033

Last Updated

June 12, 2025

Record last verified: 2025-06

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
Data can be shared no earlier than 1 year following the date of publication
Access Criteria
Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu

Locations