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

Study Overview
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Brief Summary
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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
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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

Official Title
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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

Conditions
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Leukemia, Myelodysplastic Syndromes, Myeloproliferative Syndrome

Intervention / Treatment
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* Genetic: Genomic profiling


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 30 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| GENOMIC PROFILING AND SPECIMEN BANKING REGISTRATION ARM<br>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<br>* Genomic profiling using CLIA assay<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Leukemia, Myelodysplastic Syndromes, Myeloproliferative syndrome (MPS)