A Phase I/II Study of Trametinib in Children and Adolescents with Solid Tumors


Full Title

An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects with Cancer or Plexiform Neurofibromas and Trametinib in Combination with Dabrafenib in Children and Adolescents with Cancers Harboring V600 mutation


The purpose of this study is to find the highest dose of the anticancer drug trametinib that can be given safely in children and adolescents with solid tumors that have come back after or have stopped responding to standard treatment. Examples of these tumors include Ewing sarcoma, rhabdomyosarcoma, neuroblastoma, osteosarcoma, Wilms’ tumor, liver tumors, germ cell tumors, and brain tumors.

Trametinib is approved by the U.S. Food and Drug Administration for treating melanoma in adults, but its use in children and adolescents is considered investigational. It is taken orally (by mouth) either as a liquid or tablet.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • This study is for children and adolescents ages 1 month to 17 years (inclusive).
  • Patients must have an advanced solid tumor that continues to grow despite standard treatment.
  • Patients should recover from the serious side effects of prior therapies before entering the study.

For more information about this study and to inquire about eligibility, please contact Dr. Stephen Roberts at 212-639-4034.