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

Leukemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04968834
• Other study ID #: 20-302
• Status: Recruiting
• Start date: June 11, 2021
• Est. completion date: June 2033

Study information

• Verified date: May 2024
• Source: Dana-Farber Cancer Institute
• Contact: Yana Pikman, MD
• Phone: (617) 632-4754
• Email: yana_pikman[@]dfci.harvard.edu
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: June 2033
• Est. primary completion date: June 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 30 Years

Eligibility

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

Related Conditions & MeSH terms

• Hematologic Neoplasms
• Leukemia
• Myelodysplastic Syndromes
• Myeloproliferative Syndrome
• Preleukemia
• Syndrome

Intervention

Genetic:
• Genomic profiling
Genomic profiling using CLIA assay

Locations

Country	Name	City	State
United States	Albany Medical Center	Albany	New York
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Dana Farber Cancer Institute	Boston	Massachusetts
United States	University of Vermont Medical Center	Burlington	Vermont
United States	Connecticut Children's Medical Center	Hartford	Connecticut
United States	Dartmouth-Hitchcock	Lebanon	New Hampshire
United States	Maine Medical Center	Portland	Maine
United States	Lifespan Cancer Institute	Providence	Rhode Island

Sponsors (2)

Lead Sponsor	Collaborator
Dana-Farber Cancer Institute	Charles H. Hood Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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