for people ages up to 30 years (full criteria)
study started
completion around
Principal Investigator
by Elliot Stieglitz (ucsf)
Headshot of Elliot Stieglitz
Elliot Stieglitz



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.

Official Title

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


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 Deoxyribonucleic Acid (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.

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


Recurrent, Refractory, or High Risk Leukemias, Matched Targeted Therapy, Leukemia, Preleukemia, Myelodysplastic Syndromes, Recurrence, Leukemia Profiling, Relapsed/Refractory Leukemia, New Diagnosis


You can join if…

Open to people ages up to 30 years

  • 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 therapy

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 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.

You CAN'T join if...

  • 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.


  • UCSF Helen Diller Family Comprehensive Cancer Center
    San Francisco California 94158 United States
  • Seattle Children's Hospital
    Seattle Washington 98105 United States

Lead Scientist at University of California Health

  • Elliot Stieglitz (ucsf)
    Juvenile myelomonocytic leukemia (JMML) is a blood cancer that affects young children and is difficult to diagnose. Currently available therapies cure only half of patients, with some children experiencing an aggressive disease course while some children get better with very little treatment. We have now shown that the presence of secondary mutations at diagnosis predicts a poor outcome.


in progress, not accepting new patients
Start Date
Completion Date
Dana-Farber Cancer Institute
Study Type
About 338 people participating
Last Updated