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Matched Targeted Therapy For High-Risk Leukemias and MDS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02670525
Recruitment Status : Suspended (Temporarily closed for interim data analysis.)
First Posted : February 1, 2016
Last Update Posted : April 10, 2020
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.

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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 and Myelodysplastic Syndrome
Study Start Date : February 2016
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : November 2021

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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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


Locations
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United States, California
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, United States, 94158
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
United States, Illinois
The University of Chicago
Chicago, Illinois, United States, 60637
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
United States, Minnesota
Children's Hospital's and Clinics of Minnesota
Minneapolis, Minnesota, United States, 55404
United States, Missouri
Washington University at St. Louis School of Medicine
Saint Louis, Missouri, United States, 63110
United States, New York
The Children's Hospital at Montefiore
Bronx, New York, United States, 10467
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States, 14263
Columbia University Medical Center
New York, New York, United States, 10032
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19404
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Dana-Farber Cancer Institute
Investigators
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Principal Investigator: Yana Pikman, MD Dana-Farber Cancer Institute
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Responsible Party: Yana Pikman, MD, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT02670525    
Other Study ID Numbers: 15-384
First Posted: February 1, 2016    Key Record Dates
Last Update Posted: April 10, 2020
Last Verified: April 2020
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:
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Leukemia
Neoplasms by Histologic Type
Neoplasms