An Open Label, Multicenter, Phase II Study Evaluating the Safety and Efficacy of Temozolomide Treatment in Patients With Invasive Pituitary Tumors
- To assess the effect of temozolomide on pituitary tumor growth in patients with
invasive pituitary tumors.
- To assess the effect of temozolomide on pituitary tumor response and the duration of
tumor response in these patients.
- To assess the effect of temozolomide on pituitary tumor hormone secretion in these
- To assess the effect of temozolomide on other aspects of pituitary function in these
- To assess the overall safety and tolerability of temozolomide in these patients.
- To assess the overall quality of life of patients treated with temozolomide.
OUTLINE: This is a multicenter study.
Patients receive oral temozolomide once daily on days 1-5. Treatment repeats every 28 days
for up to 12 courses in the absence of disease progression or unacceptable toxicity. After
completion of 12 courses, patients achieving a complete or partial tumor response may
continue to receive temozolomide at the investigator's discretion in the absence of disease
progression or unacceptable toxicity.
Tumor tissue samples are collected periodically to assess methylation status of the
methyl-guanine methyl-transferase promoter (MGMT) gene and to quantitate immunocytochemical
expression of the tumor suppressor proteins p53, p16, and p27. Tissue samples are also
analyzed by microarray and proteomics to determine a genetic "signature" of invasive vs
non-invasive pituitary tumors and to determine if this signature correlates with response to
temozolomide. Blood samples are also periodically for biomarker laboratory studies.
Patients complete a quality of life questionnaire periodically.
After completion of study therapy, patients are followed for 28 days.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Change from baseline of pituitary tumor control as assessed by MRI at 3, 6, 9, and 12 months
Anthony Heaney, MD
Jonsson Comprehensive Cancer Center
United States: Food and Drug Administration