Purpose

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.

Conditions

Eligibility

Eligible Ages
Under 30 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
N/A
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Diagnostic
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Relapsed/Refractory leukemia
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 After the screening procedures confirms patient eligibility: Leukemia Profiling will be performed Identifying an actionable genomic alteration and making a matched targeted therapy treatment recommendation.
  • 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.
Experimental
New diagnosis
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 Myelodysplastic syndrome (MDS) not eligible for stem cell transplant After the screening procedures confirms eligibility: Leukemia Profiling will be performed Identifying an actionable genomic alteration and making a matched targeted therapy treatment recommendation.
  • 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.

Recruiting Locations

The Children's Hospital at Montefiore
Bronx, New York 10467
Contact:
Lisa Gennarini, MD
718-741-2356
lfigueir@montefiore.org

More Details

Status
Recruiting
Sponsor
Dana-Farber Cancer Institute

Study Contact

Yana Pikman, MD
617-632-4754
YPIKMAN@PARTNERS.ORG

Detailed Description

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.