Phase II Study of Single Agent RAD001 in Patients With Colon Cancer and Activating Mutations in the PI3KCA Gene
- Determine response rate, time to tumor progression, and survival of patients with
advanced or metastatic refractory colorectal cancer and mutations in the PI3K gene
treated with everolimus.
- Determine the toxicity profile of this drug in these patients.
- Measure the signaling pathways activated in these patients.
- Determine the pharmacodynamic effects of this drug in these patients.
OUTLINE: This is an open-label study.
Patients receive oral everolimus once daily on days 1-28. Treatment repeats every 28 days in
the absence of disease progression or unacceptable toxicity.
Patients undergo tumor biopsies and normal skin biopsies at baseline and after the first
course of study treatment. Tumor tissue is examined for biological markers (e.g., epidermal
growth factor receptor, ERK, Akt, p70s6k, p27, and Rb protein) by immunohistochemistry;
apoptosis quantification by TUNEL assay; Ki-67 quantification and Ki-index; gene expression;
and c-fos and p27 expression by reverse-transcriptase polymerase chain reaction.
PROJECTED ACCRUAL: A total of 37 patients will be accrued for this study.
Masking: Open Label, Primary Purpose: Treatment
Manuel Hidalgo, MD, PhD
Sidney Kimmel Comprehensive Cancer Center
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins||Baltimore, Maryland 21231-2410|