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Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

A Phase 2 Study of the MEK Inhibitor Trametinib (NSC# 763093) in Children With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

  • Clinical Trial Information

    Trial Contact: Cevallos, Claudia; El-Shami, Jessica; Leffin, Melissa; Spinelli, Jennifer

  • IRB No: ADVL1521

    Protocol Abbrev: ADVL1521

    Principal Investigator: Amy A Smith, MD

    Phase: Drug: Phase II

    Age Group: Adult;Pediatric

    Secondary Protocol No: ADVL1521

    Treatment: Other: Laboratory Biomarker Analysis Other: Pharmacological Study Drug: Trametinib

    Therapies Involved: Chemotherapy

    ClinicalTrials.gov ID: NCT03190915

  • Objective

    To determine the objective response rate to trametinib in children with recurrent or refractory juvenile myelomonocytic leukemia (JMML).

  • Key Eligibility

    •  Patients must have had histologic verification of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease

    •  Patients with refractory or relapsed JMML must have had at least one cycle of intensive frontline therapy or at least 2 cycles of a deoxyribonucleic acid (DNA) demethylating agent with persistence of disease, defined by clinical symptoms or the presence of a clonal abnormality; frontline therapy is defined as one cycle of intravenous chemotherapy that includes of any of the following agents: fludarabine, cytarabine, or any anthracycline but specifically excludes oral 6-mercaptopurine; frontline therapy will also include any conditioning regimen as part of a stem cell transplant; patients who transform to AML at any point with more than 20% blasts are not eligible for this trial

    •  Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score

    •  Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study enrollment

    •  Patients must not be known to be refractory to red blood cell or platelet transfusions