Matched Targeted Therapy (MTT) Recommendation for Patients with Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome

Protocol # :
Recurrent, Refractory, or High Risk Leukemias
Matched Targeted Therapy
Not Applicable
Disease Sites
Lymphoid Leukemia
Leukemia, other
Principal Investigator
Pikman, Yana

Trial Description

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.

Eligibility Requirements

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

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

- 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

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.