A Pilot Open-Label Study to Examine the Safety and Efficacy of Oral LDE225 in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma Who Have Been Previously Treated With Non-LDE225 Smoothened Inhibitor(s)
This is a prospective single-center, open label, pilot study to investigate the safety and efficacy of LDE225 in patients with locally advanced or metastatic basal cell carcinoma. Primary Objectives: ? To explore the effects of oral LDE225 on the Progression Free Survival (PFS) of individuals with locally advanced or metastatic BCC who have been previously treated with a non-LDE225 Smo inhibitor. Secondary Objectives: - To evaluate the effect of oral LDE225 on tumor tissue biomarkers of BCC activation (Gii 1, 2, Patched 1,2 and Ki67) in individuals which are non-na"ive to Smo inhibitors other than LDE225, at baseline and at end-of-treatment - To describe adverse effects of oral LDE225 in individuals with a history of non-LDE225 Smo inhibitor usage - To assess the overall survival rates of individuals with locally advanced BCC or metastatic BCC who have previously taken a non-LDE225 Smo inhibitor after treatment with LDE225
Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.
- drug : LDE225
Ages Eligible For Study:
1. Age 18 years or older. 2. Histologically documented diagnosis of basal cell carcinoma deemed to be locally advanced or metastatic who have previously received a non-LDE225 Smo inhibitor. 3. World Health Organization (WHO) performance status <= 2 4. At least one measurable site of disease (as defined by Response Evaluation Criteria in Solid Tumors), or other disease specific response assessment criteria, as appropriate. State age restriction and/or gender/race-ethnic restrictions. 5. Patients with adequate bone marrow, liver and renal function, as specified below: - Absolute Neutrophil Count (ANC) >= 1.5 x 10^9/L - Hemoglobin (Hgb) >= 9 g/dL - Platelets >= 80 x 10^9/L - Serum total bilirubin <= 1.5 x upper limit of normal (ULN) - Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <= 2.5 x ULN or <= 5 x ULN if liver metastases are present - Plasma creatine phosphokinase (CK) < 1.5 x ULN - Serum creatinine <= 1.5 x ULN or 24-hour clearance >= 50ml/min 6. Written informed consent obtained prior to any screening procedures