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Trial record 1 of 1 for:    MTT 15-384
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Matched Targeted Therapy For High-Risk Leukemias and MDS

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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

Tracking Information
First Submitted Date  ICMJE January 28, 2016
First Posted Date  ICMJE February 1, 2016
Last Update Posted Date April 10, 2020
Study Start Date  ICMJE February 2016
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 29, 2016)
Rate of patients with actionable alterations [ Time Frame: 2 Years ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 29, 2016)
  • Rate of Somatic genomic alterations [ Time Frame: 2 Years ]
  • Rate of Results Reporting [ Time Frame: 2 Years ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Matched Targeted Therapy For High-Risk Leukemias and MDS
Official Title  ICMJE Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
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.

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  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Condition  ICMJE
  • Recurrent, Refractory, or High Risk Leukemias
  • Matched Targeted Therapy
Intervention  ICMJE 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.
Study Arms  ICMJE
  • 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.
    Intervention: Genetic: Leukemia Profiling
  • 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.
    Intervention: Genetic: Leukemia Profiling
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Suspended
Estimated Enrollment  ICMJE
 (submitted: January 29, 2016)
200
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2021
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 30 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02670525
Other Study ID Numbers  ICMJE 15-384
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Yana Pikman, MD, Dana-Farber Cancer Institute
Study Sponsor  ICMJE Dana-Farber Cancer Institute
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Yana Pikman, MD Dana-Farber Cancer Institute
PRS Account Dana-Farber Cancer Institute
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP