Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04968834
Recruitment Status : Recruiting
First Posted : July 20, 2021
Last Update Posted : December 6, 2021
Sponsor:
Collaborator:
Charles H. Hood Foundation
Information provided by (Responsible Party):
Yana Pikman, MD, 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.


Condition or disease Intervention/treatment
Leukemia Myelodysplastic Syndromes Myeloproliferative Syndrome 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

Layout table for study information
Study Type : Observational [Patient Registry]
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: Protocol For Genomically Profiling, Collecting, Archiving And Distributing Blood And Bone Marrow Specimens From Children And Young Adults With Hematologic Malignancy
Actual Study Start Date : June 11, 2021
Estimated Primary Completion Date : June 2025
Estimated Study Completion Date : June 2025

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  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 Outcome Measures :
  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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   up to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
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.
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04968834


Contacts
Layout table for location contacts
Contact: Yana Pikman, MD (617) 632-4754 yana_pikman@dfci.harvard.edu

Locations
Layout table for location information
United States, Massachusetts
Childrens Hospital Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Yana Pikman, MD    617-632-4754    yana_pikman@dfci.harvard.edu   
Principal Investigator: Yana Pikman, MD         
Principal Investigator: Jessica Pollard, MD         
Dana Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
Contact: Yana Pikman, MD    617-632-4754    YPIKMAN@PARTNERS.ORG   
Principal Investigator: Yana Pikman, MD         
Principal Investigator: Jessica Pollard, MD         
United States, New Hampshire
Dartmouth-Hitchcock Recruiting
Lebanon, New Hampshire, United States, 03756
Contact: Bonnie Lau, MD    603-650-5000      
Principal Investigator: Bonnie Lau, MD         
Sponsors and Collaborators
Dana-Farber Cancer Institute
Charles H. Hood Foundation
Investigators
Layout table for investigator information
Principal Investigator: Yana Pikman, MD Dana-Farber Cancer Institute
Layout table for additonal information
Responsible Party: Yana Pikman, MD, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT04968834    
Other Study ID Numbers: 20-302
First Posted: July 20, 2021    Key Record Dates
Last Update Posted: December 6, 2021
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (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

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Yana Pikman, MD, Dana-Farber Cancer Institute:
Leukemia
Myelodysplastic Syndromes
Myeloproliferative syndrome (MPS)
Additional relevant MeSH terms:
Layout table for MeSH terms
Preleukemia
Hematologic Neoplasms
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms by Site