Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome
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| ClinicalTrials.gov Identifier: NCT02670525 |
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Recruitment Status :
Active, not recruiting
First Posted : February 1, 2016
Results First Posted : July 18, 2023
Last Update Posted : November 1, 2023
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This research study is seeking to gain new knowledge about Recurrent, Refractory, or High Risk Leukemias in children and young adults.
This study is evaluating the use of specialized testing called leukemia profiling. Once the profiling is performed, the results are evaluated by an expert panel of physicians, scientists and pharmacists. This may result in a recommendation for a specific cancer therapy or a clinical trial called matched targeted therapy (MTT). The results of the leukemia profiling and, if applicable, the MTT recommendation will be communicated to the participant's primary oncologist.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Recurrent, Refractory, or High Risk Leukemias Matched Targeted Therapy | Genetic: Leukemia Profiling | Not Applicable |
Our tissues and organs are made up of cells. Cancer occurs when the molecules that normally control cell growth are damaged. The damage results in unchecked cell growth which causes a tumor, a collection of cancer cells. The damage is referred to as an alteration. There are different types of cancer-causing alterations. Genes are the part of cells that contain the instructions which tell our cells how to make the right proteins to grow and work. Genes are composed of Deoxyribonucleic Acid (DNA) letters that spell out these instructions.
By participating in this study, the participant's leukemia cells will be tested for cancer causing alterations. This testing is called leukemia profiling. The leukemia profiling will be performed using bone marrow or blood that has already been obtained during a clinical test. Alternately, the profiling may be done on leukemia cells that are planned to be obtained as part of routine clinical care.
This study will determine whether it is possible to use profiling results to determine a matched targeted therapy for patients with leukemia. It will describe the range of mutations found in patients with leukemia with this type of profiling, and describe the clinical outcomes of patients who receive a matched targeted therapy.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 338 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Diagnostic |
| Official Title: | Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome |
| Actual Study Start Date : | August 17, 2016 |
| Actual Primary Completion Date : | May 16, 2022 |
| Estimated Study Completion Date : | May 2027 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Relapsed/Refractory Leukemia
Cohort 1: Relapsed/Refractory Leukemia
After the screening procedures confirms patient eligibility:
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Genetic: Leukemia Profiling
Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. |
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Experimental: New Diagnosis
Cohort 2: New Diagnosis
After the screening procedures confirms eligibility:
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Genetic: Leukemia Profiling
Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist. |
- Rate of Patients With Actionable Alterations [ Time Frame: Actionable alteration was identified based on a combination of fluorescence in situ hybridization (FISH)/cytogenetics and sequencing. Average time to full results was 2 weeks. ]An actionable alteration was defined as a cancer-associated genomic event for which there was a targeted drug available. Actionable genetic alterations were identified by a combination of standard-of-care cytogenetics/fluorescence in situ hybridization (FISH) and sequencing performed as part of the study. This study is considered feasible if at least 13% of participants analyzed have profile data and identifiable actionable alterations. A 90% exact binomial confidence interval is presented for the rate of patients with actionable alterations to compare the observed rate against 13%.
- Rate of Somatic Genomic Alterations [ Time Frame: 2 Years ]This Secondary Outcome will be addressed by describing the somatic genomic alterations in Cohort 1 (Relapsed/Refractory Leukemia arm) and Cohort 2 (New Diagnosis arm) that are discovered by genomic analyses.
- Rate of Results Reporting [ Time Frame: 2 Years ]This Secondary Outcome will be addressed by a description of the time of results reporting. Documentation of the time of sample receipt and processing, resources and personnel utilized at each step of the process, time to results reporting, interpretation and review by Expert panel and communication of results to the primary oncologist will be captured.
- Parent's Feelings and Understanding of Genomic Testing [ Time Frame: 2 Years ]This Secondary Outcome will be addressed by describing the hopes and concerns of parents of children with recurrent/refractory/high-risk de novo leukemia regarding genomic testing of their child's leukemia as well as their understanding of the testing and evaluate whether the hopes and concerns were realized following the return of results.
- Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models [ Time Frame: 2 Years ]This Secondary Outcome will be addressed by completing analysis of the primary leukemia sensitivity testing to a panel of drugs or shRNA and establishing xenograft models in dedicated participating research laboratories and conducting co-clinical trials with the recommended matched therapy once the animal model is established.
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 |
Inclusion Criteria:
- Birth to ≤ 30 years at study entry
- Diagnosis: Patients will be enrolled in one of the two cohorts based on diagnosis:
Cohort 1: Relapsed/refractory leukemia
- Acute lymphoblastic leukemia (ALL), first or greater relapse
- Acute myeloid leukemia (AML), first or greater relapse
- Leukemia refractory to induction chemotherapy
- Other recurrent leukemia
- Myelodysplastic syndrome (MDS), first or greater relapse, or refractory to initial therapy
Cohort 2: New diagnosis
- Acute myeloid leukemia (AML), new diagnosis
- New diagnosis infant mixed-lineage leukemia (MLL)-rearranged ALL or low hypodiploid (<40 chromosomes) ALL
- Rare leukemia- e.g., juvenile myelomonocytic leukemia (JMML), leukemia of ambiguous lineage
- Secondary leukemia
- Myelodysplastic syndrome (MDS) not eligible for stem cell transplant
Pathologic Criteria
- Histologic confirmation of leukemia at the time of diagnosis or recurrence
Specimen Samples
- Sufficient leukemia specimen available for profiling from diagnosis or recurrence OR bone marrow aspirate/blood draw/pheresis/other fresh sample of patient leukemia cells planned for clinical care anticipated to allow collection of minimum specimen for testing.
Exclusion Criteria:
- Insufficient leukemia specimen available for profiling from diagnosis or recurrence; or bone marrow evaluation/blood draw/other leukemia cell sample NOT planned to be obtained for clinical care; or peripheral blast percentage <20% AND 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): NCT02670525
| United States, California | |
| UCSF Helen Diller Family Comprehensive Cancer Center | |
| San Francisco, California, United States, 94158 | |
| United States, Colorado | |
| Children's Hospital Colorado | |
| Aurora, Colorado, United States, 80045 | |
| United States, Georgia | |
| Children's Healthcare of Atlanta | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| The University of Chicago | |
| Chicago, Illinois, United States, 60637 | |
| United States, Maryland | |
| Johns Hopkins Hospital | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Massachusetts | |
| Dana Farber Cancer Institute | |
| Boston, Massachusetts, United States, 02215 | |
| United States, Minnesota | |
| Children's Hospital's and Clinics of Minnesota | |
| Minneapolis, Minnesota, United States, 55404 | |
| United States, Missouri | |
| Washington University at St. Louis School of Medicine | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, New York | |
| The Children's Hospital at Montefiore | |
| Bronx, New York, United States, 10467 | |
| Roswell Park Comprehensive Cancer Center | |
| Buffalo, New York, United States, 14263 | |
| Columbia University Medical Center | |
| New York, New York, United States, 10032 | |
| Memorial Sloan Kettering Cancer Center | |
| New York, New York, United States, 10065 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19404 | |
| United States, Washington | |
| Seattle Children's Hospital | |
| Seattle, Washington, United States, 98105 | |
| United States, Wisconsin | |
| Children's Hospital of Wisconsin | |
| Milwaukee, Wisconsin, United States, 53226 | |
| Principal Investigator: | Yana Pikman, MD | Dana-Farber Cancer Institute |
Documents provided by Yana Pikman, MD, Dana-Farber Cancer Institute:
| Responsible Party: | Yana Pikman, MD, Principal Investigator, Dana-Farber Cancer Institute |
| ClinicalTrials.gov Identifier: | NCT02670525 |
| Other Study ID Numbers: |
15-384 |
| First Posted: | February 1, 2016 Key Record Dates |
| Results First Posted: | July 18, 2023 |
| Last Update Posted: | November 1, 2023 |
| Last Verified: | October 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
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Recurrent, Refractory, or High Risk Leukemias Matched targeted therapy |
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Leukemia Preleukemia Myelodysplastic Syndromes Recurrence Neoplasms by Histologic Type Neoplasms |
Hematologic Diseases Disease Attributes Pathologic Processes Bone Marrow Diseases Precancerous Conditions |