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Matched Targeted Therapy For High-Risk Leukemias

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ClinicalTrials.gov Identifier: NCT02670525
Recruitment Status : Recruiting
First Posted : February 1, 2016
Last Update Posted : August 29, 2018
Sponsor:
Information provided by (Responsible Party):
Yana Pikman, MD, Dana-Farber Cancer Institute

Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Recurrent, Refractory, or High Risk Leukemias Matched Targeted Therapy Genetic: Leukemia Profiling Not Applicable

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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias
Study Start Date : February 2016
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : November 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Leukemia

Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. Rate of patients with actionable alterations [ Time Frame: 2 Years ]

Secondary Outcome Measures :
  1. Rate of Somatic genomic alterations [ Time Frame: 2 Years ]
  2. Rate of Results Reporting [ Time Frame: 2 Years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02670525


Contacts
Contact: Yana Pikman, MD 617-632-4754 YPIKMAN@PARTNERS.ORG
Contact: Andrew Place, MD, PhD 617-632-2313 andrew_place@dfci.harvard.edu

Locations
United States, California
UCSF Helen Diller Family Comprehensive Cancer Center Recruiting
San Francisco, California, United States, 94158
Contact: Mignon Loh, MD    415-514-0853    Lohm@peds.ucsf.edu   
Principal Investigator: Mignon Loh, MD         
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Kelly Maloney, MD    720-777-6740    Kelly.Maloney@ucdenver.edu   
Principal Investigator: Kelly Maloney, MD         
United States, Georgia
Children's Healthcare of Atlanta Active, not recruiting
Atlanta, Georgia, United States, 30322
United States, Illinois
The University of Chicago Recruiting
Chicago, Illinois, United States, 60637
Contact: Jennifer McNeer, MD    773-702-1762    jmcneer@peds.bsd.uchicago.edu   
Principal Investigator: Jennifer McNeer, MD         
United States, Maryland
Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287
Contact: Patrick Brown, MD    410-955-8817    Pbrown2@jhmi.edu   
Principal Investigator: Patrick Brown, MD         
United States, Massachusetts
Dana Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
Contact: Yana Pikman, MD    617-632-4754    YPIKMAN@PARTNERS.ORG   
Principal Investigator: Yana Pikman, MD         
Sub-Investigator: Lewis B Silverman, MD         
Sub-Investigator: Andrew Place, MD, PhD         
United States, Minnesota
Children's Hospital's and Clinics of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Nathan Gossai, MD    612-813-5940    Nathan.gossai@childrensmn.org   
Principal Investigator: Nathan Gossai, MD         
United States, New York
The Children's Hospital at Montefiore Recruiting
Bronx, New York, United States, 10467
Contact: Peter Cole, MD    718-741-2356    peter.cole@einstein.yu.edu   
Principal Investigator: Peter Cole, MD         
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Jing Chen, MD       jc3722@cumc.columbia.edu   
Principal Investigator: Jing Chen, MD         
Memorial Sloan Kettering Cancer Center Active, not recruiting
New York, New York, United States, 10065
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19404
Contact: Sarah Tasian, MD    267-425-0118    TasianS@email.chop.edu   
Principal Investigator: Sarah Tasian, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Todd Cooper, MD    206-987-1533    Todd.Cooper@seattlechildrens.org   
Principal Investigator: Todd Cooper, MD         
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Michael Burke, MD       mmburke@mcw.edu   
Principal Investigator: Michael Burke, MD         
Sponsors and Collaborators
Dana-Farber Cancer Institute
Investigators
Principal Investigator: Yana Pikman, MD Dana-Farber Cancer Institute

Responsible Party: Yana Pikman, MD, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT02670525     History of Changes
Other Study ID Numbers: 15-384
First Posted: February 1, 2016    Key Record Dates
Last Update Posted: August 29, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Yana Pikman, MD, Dana-Farber Cancer Institute:
Recurrent, Refractory, or High Risk Leukemias
Matched targeted therapy

Additional relevant MeSH terms:
Leukemia
Neoplasms by Histologic Type
Neoplasms