LeukemiaNCT04968834
Genomically Profiling, Collecting, Archiving and Distributing Hematologic Malignancy Specimens
An observational study of Leukemia and Myelodysplastic Syndromes, run by Dana-Farber Cancer Institute at 8 sites in United States.
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. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes, Myeloproliferative Syndrome
- What is being tested?
- Genomic profiling
- How many people
- 300 participants
- Where
- 8 sitesUnited States
- Study dates
- 11 Jun 2021to Jun 2033
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 6 recruiting
- Recruiting
Albany Medical Center
Albany, New York, 12208
Jessica Geaney, MD, geaneyj@amc.edu
- Active, not recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
- Active, not recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
- Recruiting
University of Vermont Medical Center
Burlington, Vermont, 05401
Jessica Heath, MD, Jessica.Heath@uvmhealth.org
- Recruiting
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Natalie Bezler, MD, Nbezler@connecticutchildrens.org
- Recruiting
Dartmouth-Hitchcock
Lebanon, New Hampshire, 03756
Angela Ricci, MD, angela.m.ricci@hitchcock.org
- Recruiting
Maine Medical Center
Portland, Maine, 04102
Sei Sze, MD, Sei.Sze@mainehealth.org
- Recruiting
Lifespan Cancer Institute
Providence, Rhode Island, 02903
Jennifer Welch, MD, jwelch@lifespan.org
About this study
Official title: Protocol For Genomically Profiling, Collecting, Archiving and Distributing Blood and Bone Marrow Specimens From Children and Young Adults With Hematologic Malignancy
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.
Read the 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
- Being tested
- Genomic profiling
- Keywords
- Leukemia, Myelodysplastic Syndromes, Myeloproliferative syndrome (MPS)
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genomic profilingGenetic
Genomic profiling using CLIA assay
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Genetic: Genomic profiling |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 Years
Secondary outcomes (1)
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.
Measured: 3 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2021
- Study start11 Jun 2021
- Primary completionJun 2033 (estimated)
- Study completionJun 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Charles H. Hood Foundation, Rally Foundation for Childhood Cancer Research
- Responsible party
- Principal investigator, Yana Pikman, MD, Dana-Farber Cancer Institute
- Investigators
- Yana Pikman, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04968834
- Sponsor's protocol ID
- 20-302
- Status last verified
- Jul 2026
- First submitted
- 9 Jul 2021
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protocol For Genomically Profiling, Collecting, Archiving and Distributing Blood and Bone Marrow Specimens From Children and Young Adults With Hematologic Malignancy
- ClinicalTrials.gov ID
- NCT04968834
- Acronym / sponsor ID
- 20-302
- Keywords
- Leukemia, Myelodysplastic Syndromes, Myeloproliferative syndrome (MPS)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Charles H. Hood Foundation, Rally Foundation for Childhood Cancer Research
- Study start
- 11 Jun 2021 (actual)
- Primary completion
- Jun 2033 (estimated)
- Study completion
- Jun 2033 (estimated)
- First posted
- 20 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GENOMIC PROFILING AND SPECIMEN BANKING REGISTRATION ARM | – | Genetic: Genomic profilingThe 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 |
Who may qualify
- Ages
- Up to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (1 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Genomic profilingGenomic profiling using CLIA assay | Genetic | GENOMIC PROFILING AND SPECIMEN BANKING REGISTRATION ARM |
Primary outcome
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.
1 secondary outcome
Number of Patients Enrolled for Genomic Profiling-New Diagnosis
Time frame: 3 Years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Yana Pikman, MD principal investigator
Dana-Farber Cancer Institute
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia and Myelodysplastic Syndromes, run by Dana-Farber Cancer Institute at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yana Pikman, MD ((617) 632-4754, yana_pikman@dfci.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients Enrolled for Genomic Profiling-Pediatric Leukemia (time frame: 3 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2026.