Phase I/II Study of MEK162 for Children With Ras/Raf Pathway Activated Tumors
Status:
Active, not recruiting
Trial end date:
2023-02-01
Target enrollment:
Participant gender:
Summary
The main purpose of phase I studies in general is to determine the best dose ("maximum
tolerated dose") of a drug, and to find out the most common side effects. The main purpose of
the phase I component of this study specifically is to determine the best dose of the
experimental drug MEK162 and to find out whether the drug is safe in children and adolescents
with tumors that have grown or come back despite standard therapy.
Another purpose of this study is to measure the concentration of drug in the blood to help
understand how much drug gets into the body and how quickly the drug is removed from the
body. Another purpose of this study is to determine whether MEK162 turns off the Ras/Raf/MAP
pathway as expected by measuring this pathway in blood cells. Finally, in this study, the
investigators hope to start finding out whether or not MEK162 causes different types of
tumors in children to shrink or stop growing.
The main purpose of the phase II component of the study is to determine whether MEK162 causes
specific types of tumors in children and adolescents to shrink or stop growing. These
specific types of tumors include low-grade gliomas, tumors in patients with a genetic
condition called neurofibromatosis type 1, and other tumors thought to be caused by abnormal
activation of the Ras/Raf/MEK molecular pathway.
Another purpose of this study is for researchers to learn whether specific abnormalities in
the DNA of tumors can help predict whether tumors will respond to MEK162.