Conditions: Basal Cell Carcinoma
Keywords: basal cell carcinoma, sonidegib, smoothened inhibitor, hedgehog
Allocation: NON_RANDOMIZED | Intervention model: PARALLEL
Enrollment: 11
Healthy volunteers: no

Brief summary:
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

Detailed description:
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 (e.g. Gli and Ki67) in individuals which are non-naive 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

Arms:
arm 1: Patients previously treated with non-LDE225 Smo inhibitor who were refractory. arm 2: Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.

Interventions: intervention 1: LDE225
Intervention descriptions: intervention 1: 800-mg (4 200-mg capsules/day) capsule

Protocol document: