Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome
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ClinicalTrials.gov Identifier: NCT02670525 |
Recruitment Status :
Active, not recruiting
First Posted : February 1, 2016
Results First Posted : July 18, 2023
Last Update Posted : May 8, 2024
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Tracking Information | ||||
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First Submitted Date ICMJE | January 28, 2016 | |||
First Posted Date ICMJE | February 1, 2016 | |||
Results First Submitted Date ICMJE | May 15, 2023 | |||
Results First Posted Date ICMJE | July 18, 2023 | |||
Last Update Posted Date | May 8, 2024 | |||
Actual Study Start Date ICMJE | August 17, 2016 | |||
Actual Primary Completion Date | May 16, 2022 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
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%.
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Original Primary Outcome Measures ICMJE |
Rate of patients with actionable alterations [ Time Frame: 2 Years ] | |||
Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Matched Targeted Therapy For High-Risk Leukemias and Myelodysplastic Syndrome | |||
Official Title ICMJE | Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome | |||
Brief Summary | 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. |
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Detailed Description | 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. |
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Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Not Applicable | |||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Diagnostic |
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Condition ICMJE |
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Intervention ICMJE | 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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Study Arms ICMJE |
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Active, not recruiting | |||
Actual Enrollment ICMJE |
338 | |||
Original Estimated Enrollment ICMJE |
200 | |||
Estimated Study Completion Date ICMJE | May 2027 | |||
Actual Primary Completion Date | May 16, 2022 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Cohort 1: Relapsed/refractory leukemia
Cohort 2: New diagnosis
Pathologic Criteria
Specimen Samples
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | up to 30 Years (Child, Adult) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT02670525 | |||
Other Study ID Numbers ICMJE | 15-384 | |||
Has Data Monitoring Committee | No | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE |
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Current Responsible Party | Yana Pikman, MD, Dana-Farber Cancer Institute | |||
Original Responsible Party | Same as current | |||
Current Study Sponsor ICMJE | Dana-Farber Cancer Institute | |||
Original Study Sponsor ICMJE | Same as current | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | Dana-Farber Cancer Institute | |||
Verification Date | May 2024 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |