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 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.

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:

  -  Age ≤ 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

Cohort 2: New diagnosis

  -  Acute myeloid leukemia, new diagnosis (excluding acute promyelocytic leukemia (APL))

  -  New diagnosis infant MLL-rearranged ALL or low hypodiploid (<40 chromosomes) ALL

  -  Rare leukemia- e.g., JMML, leukemia of ambiguous lineage

  -  Secondary leukemia

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

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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