Matched Targeted Therapy For High-Risk Leukemias

Status: Recruiting
Phase: N/A
DFCI Protocol ID: 15-384

This research study is seeking to gain new knowledge about recurrent, refractory, or high risk leukemias and myelodysplastic syndrome in children and young adults. It is evaluating the use of specialized testing which analyzes the DNA and RNA of leukemia cells, called leukemia profiling. Once profiling is performed, the results are evaluated by an expert panel of physicians and scientists to determine if there is a targeted drug available that could attack the identified leukemia-associated alteration. This matched targeted therapy (MTT) recommendation is then communicated to the participant's primary oncologist.

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Yana Pikman, MD, Dana Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,

Eligibility Criteria

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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