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 |
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Recruitment Status :
Recruiting
First Posted : July 20, 2021
Last Update Posted : November 1, 2023
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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.
| Condition or disease | Intervention/treatment |
|---|---|
| Leukemia Myelodysplastic Syndromes Myeloproliferative Syndrome | Genetic: Genomic profiling |
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] |
| 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 |
| Group/Cohort | Intervention/treatment |
|---|---|
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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
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Genetic: Genomic profiling
Genomic profiling using CLIA assay |
- 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.
- 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.
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.
| Ages Eligible for Study: | up to 30 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- Age: birth to < 30 years of age
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Diagnosis:
-- Patient with acute leukemia, chronic leukemia, MDS/AML, myelodysplastic syndrome or myeloproliferative syndromes. Disease can be newly diagnosed or relapsed/refractory.
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Pathology Criteria:
-- Histologic confirmation of leukemia or myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPS) at the time of diagnosis or recurrence
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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
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
| Contact: Yana Pikman, MD | (617) 632-4754 | yana_pikman@dfci.harvard.edu |
| United States, Connecticut | |
| Connecticut Children's Medical Center | Recruiting |
| Hartford, Connecticut, United States, 06106 | |
| Contact: Natalie Bezler, MD Nbezler@connecticutchildrens.org | |
| United States, Maine | |
| Maine Medical Center | Recruiting |
| Portland, Maine, United States, 04102 | |
| Contact: Sei Sze, MD Sei.Sze@mainehealth.org | |
| United States, Massachusetts | |
| Boston Children's Hospital | Active, not recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Dana Farber Cancer Institute | Active, not recruiting |
| Boston, Massachusetts, United States, 02215 | |
| United States, New Hampshire | |
| Dartmouth-Hitchcock | Recruiting |
| Lebanon, New Hampshire, United States, 03756 | |
| Contact: Angela Ricci, MD angela.m.ricci@hitchcock.org | |
| United States, New York | |
| Albany Medical Center | Recruiting |
| Albany, New York, United States, 12208 | |
| Contact: Jessica Geaney, MD geaneyj@amc.edu | |
| United States, Rhode Island | |
| Lifespan Cancer Institute | Recruiting |
| Providence, Rhode Island, United States, 02903 | |
| Contact: Jennifer Welch, MD jwelch@lifespan.org | |
| United States, Vermont | |
| University of Vermont Medical Center | Recruiting |
| Burlington, Vermont, United States, 05401 | |
| Contact: Jessica Heath, MD Jessica.Heath@uvmhealth.org | |
| Principal Investigator: | Yana Pikman, MD | Dana-Farber Cancer Institute |
| 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: | November 1, 2023 |
| Last Verified: | October 2023 |
| 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 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Leukemia Myelodysplastic Syndromes Myeloproliferative syndrome (MPS) |
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Preleukemia Hematologic Neoplasms Myelodysplastic Syndromes Syndrome Disease Pathologic Processes |
Neoplasms Hematologic Diseases Bone Marrow Diseases Precancerous Conditions Neoplasms by Site |