Matched Targeted Therapy For High-Risk Leukemias and MDS



Status:Recruiting
Conditions:Blood Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:Any - 30
Updated:2/28/2019
Start Date:February 2016
End Date:November 2020
Contact:Yana Pikman, MD
Email:YPIKMAN@PARTNERS.ORG
Phone:617-632-4754

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

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.

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.

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.
We found this trial at
15
sites
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Sarah Tasian, MD
Phone: 267-425-0118
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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5801 South Ellis Avenue
Chicago, Illinois 60637
 773.702.1234
Principal Investigator: Jennifer McNeer, MD
Phone: 773-702-1762
University of Chicago One of the world's premier academic and research institutions, the University of...
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Chicago, IL
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1600 Divisadero Street
San Francisco, California 94115
888.689.8273
Principal Investigator: Mignon Loh, MD
Phone: 415-514-0853
UCSF Helen Diller Family Comprehensive Cancer Center UCSF’s long tradition of excellence in cancer research...
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San Francisco, CA
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1405 Clifton Road NE
Atlanta, Georgia 30322
404-785-6000
Principal Investigator: Melinda Pauly, MD
Children's Healthcare of Atlanta Whether treating a toddler in an emergency or supporting a teen...
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Kelly Maloney, MD
Phone: 720-777-6740
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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1800 Orleans St.
Baltimore, Maryland 21287
410-955-5000
Principal Investigator: Patrick Brown, MD
Phone: 410-955-8817
Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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Baltimore, MD
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450 Brookline Ave
Boston, Massachusetts 2215
617-632-3000
Principal Investigator: Yana Pikman, MD
Phone: 617-632-4754
Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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Boston, MA
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Bronx, New York 10467
Principal Investigator: Lisa Gennarini, MD
Phone: 718-741-2356
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665 Elm Street
Buffalo, New York 14263
Principal Investigator: Matthew Barth, MD
Phone: 716-845-2333
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Buffalo, NY
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9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Principal Investigator: Michael Burke, MD
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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Milwaukee, WI
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Minneapolis, Minnesota 55404
Principal Investigator: Nathan Gossai, MD
Phone: 612-813-5940
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630 W 168th St
New York, New York
212-305-2862
Principal Investigator: Justine Kahn, MD
Phone: 212-305-5808
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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1275 York Ave
New York, New York 10021
(212) 639-2000
Principal Investigator: Neerav Shukla, MD
Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering Cancer Center — the world's oldest and...
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Saint Louis, Missouri 63110
Principal Investigator: Jeffrey Magee, MD, PhD
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Todd Cooper, MD
Phone: 206-987-1533
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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Seattle, WA
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