Matched Targeted Therapy For High-Risk Leukemias and MDS

  • End date
    Jun 12, 2022
  • participants needed
  • sponsor
    Dana-Farber Cancer Institute
Updated on 12 June 2021


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.


This study will determine whether it is possible to use profiling results and 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.

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 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.

Condition Recurrent, Refractory, or High Risk Leukemias, Matched Targeted Therapy, Recurrent, Refractory, or High Risk Leukemias, Recurrent, Refractory, or High Risk Leukemias, Recurrent, Refractory, or High Risk Leukemias, Recurrent, Refractory, or High Risk Leukemias, Recurrent, Refractory, or High Risk Leukemias, Recurrent, Refractory, or High Risk Leukemias
Treatment Leukemia Profiling
Clinical Study IdentifierNCT02670525
SponsorDana-Farber Cancer Institute
Last Modified on12 June 2021


Yes No Not Sure

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, first or greater relapse
Acute myeloid leukemia, 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, new diagnosis
New diagnosis infant MLL-rearranged ALL or low hypodiploid (<40 chromosomes) ALL
Rare leukemia- e.g., 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
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How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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