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

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ClinicalTrials.gov Identifier: NCT04968834
Recruitment Status : Recruiting
First Posted : July 20, 2021
Last Update Posted : May 7, 2024
Sponsor:
Collaborator:
Charles H. Hood Foundation
Information provided by (Responsible Party):
Yana Pikman, MD, Dana-Farber Cancer Institute

Tracking Information
First Submitted Date July 9, 2021
First Posted Date July 20, 2021
Last Update Posted Date May 7, 2024
Actual Study Start Date June 11, 2021
Estimated Primary Completion Date June 2033   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 9, 2021)		 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.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: July 9, 2021)		 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.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Protocol For Genomically Profiling, Collecting, Archiving And Distributing Blood And Bone Marrow Specimens From Children And Young Adults With Hematologic Malignancy
Official Title Protocol For Genomically Profiling, Collecting, Archiving And Distributing Blood And Bone Marrow Specimens From Children And Young Adults With Hematologic Malignancy
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 Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 5 Years
Biospecimen Not Provided
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.
Condition
  • Leukemia
  • Myelodysplastic Syndromes
  • Myeloproliferative Syndrome
Intervention Genetic: Genomic profiling
Genomic profiling using CLIA assay
Study Groups/Cohorts 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
Intervention: Genetic: Genomic profiling
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 9, 2021)		 300
Original Estimated Enrollment Same as current
Estimated Study Completion Date June 2033
Estimated Primary Completion Date June 2033   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 30 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Yana Pikman, MD (617) 632-4754 yana_pikman@dfci.harvard.edu
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04968834
Other Study ID Numbers 20-302
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
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
Supporting Materials: 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
Current Responsible Party Yana Pikman, MD, Dana-Farber Cancer Institute
Original Responsible Party Same as current
Current Study Sponsor Dana-Farber Cancer Institute
Original Study Sponsor Same as current
Collaborators Charles H. Hood Foundation
Investigators
Principal Investigator: Yana Pikman, MD Dana-Farber Cancer Institute
PRS Account Dana-Farber Cancer Institute
Verification Date May 2024