iMATRIXcobi: Safety and Pharmacokinetics of Cobimetinib in Pediatric and Young Adult Patients With Previously Treated Solid Tumors

Status: Recruiting
DFCI Protocol ID: 15-524

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.

Conducting Institutions:

Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:

Steven Dubois MD, Dana Farber Cancer Institute

Site-responsible Investigators:


Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,

Eligibility Criteria

Inclusion Criteria:

  -  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

  -  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

Exclusion Criteria:

  -  Pregnant or lactating women

  -  Close proximity in time to treatment with high dose chemotherapy, differentiation
     therapy, immunotherapy, autologous stem cell transplant, radiotherapy, hormonal
     therapy, hematopoietic growth factor, or investigational therapy according to
     protocol-defined criteria prior to initiation of study drug

  -  Inability to swallow tablets

  -  Impaired gastrointestinal absorption

  -  History or evidence of retinal pathology according to protocol-defined criteria,
     including serous retinopathy

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