Study of Safety & PK of Cobimetinib Pts w/Solid Tumors

A Phase I/II, Multicenter, Open-Label, Dose-Escalation Study Of The Safety And Pharmacokinetics Of Cobimetinib In Pediatric And Young Adult Patients With Previously Treated Solid Tumors

November 22, 2017

  • Clinical Trial Information

    Trial Contact: El-Shami, Jessica; Francois, Laetitia Claire; Leffin, Melissa; Spinelli, Jennifer

  • IRB No: 16.032.02

    Protocol Abbrev: GO29665

    Principal Investigator: Amy A Smith, MD

    Sub Investigators: AguilarBonilla, Ana MD; Eslin, Don MD; Giusti, Vincent MD; Levy, Alejandro MD; Sutphin, Robert MD; Pope, Michele ARNP; Story, Allison ARNP; Sullivan, Kelly ARNP

    Phase: Drug: Phase I

    Age Group: Pediatric

    Secondary Protocol No: GO29665

    Treatment: Medication

    Applicable Disease Sites: Solid Tumor

    Therapies Involved: Cobimetinib ID: NCT02639546

  • Objective

    This open-label, dose-escalation study is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of cobimetinib in pediatric and young adult participants with solid tumors with known or potential kinase pathway activation for which standard therapy has proven to be ineffective or intolerable and for which no curative standard-of-care treatment options exist. The study will be conducted in two stages: a dose-escalation stage and an expansion stage at the recommended dose.

  • Key Eligibility

    For dose-escalation stage: age at study entry to be greater than (>) 6 years to less than (<) 18 years and weighing >= 20 kilograms
    •  For expansion stage: age at study entry to be >6 years to <30 years
    •  Histologically or cytologically confirmed tumors for which prior treatment has proven to be ineffective or intolerable or for which no standard therapy exists
    •  Tumor with known or expected RAS/RAF/MEK/ERK pathway involvement. Diagnosis MUST be one of the following tumor types:

    Central nervous system gliomas, including high- and low-grade gliomas, and DIPG Embryonal rhabdomyosarcoma and other non-rhabdomyosarcoma soft tissue sarcomas Neuroblastoma Melanoma Malignant peripheral nerve sheath tumor Rhabdoid tumors, including ATRT NF1-associated tumor (including plexiform neurofibroma), schwannoma, or RASopathy-associated tumor that in the judgment of the investigator is life threatening, results in severe symptoms (including severe pain), or is in close proximity to vital structures;
    •  Measurable disease as defined by International Neuroblastoma Response Criteria, Response Assessment in Neuro-Oncology Criteria, Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, or evaluable by nuclear medicine techniques, immunocytochemistry, tumor markers or other reliable measures
    •  Availability of tumor tissue at study enrollment
    •  Lansky performance status or Karnofsky performance status of at least 50 percent
    •  Life expectancy of at least 3 months
    •  Adequate hematologic, cardiac, and end-organ function
    •  Body weight must be greater than or equal to 20 kg