Conditions: Basal Cell Carcinoma Gorlin Syndrome Nevoid Basal Cell Carcinoma Syndrome
Keywords: Basal Cell Carcinoma Gorlin Syndrome, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, Nevoid Basal Cell Carcinoma Syndrome, Basal Cell Carcinoma Nevus Syndrome Smo inhibitor, Hedgehog pathway inhibitor BCCs
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 10
Healthy volunteers: no

Brief summary:
This was a phase II, double-blind, randomized, proof-of-concept, dose-ranging trial evaluating the efficacy, safety and pharmacokinetics of oral LDE225 in treatment of adult patients with NBCCS. This was an exploratory study designed to demonstrate preliminary efficacy of LDE225 in this indication. This study included a Screening period of approximately 4 weeks, treatment period duration of 12 weeks with initial follow-up of approximately 6-8 weeks followed by a long-term follow-up period.

Detailed description:

Arms:
arm 1: Participants received 400 mg once daily. arm 2: Participants received matching placebo.

Interventions: intervention 1: LDE225 intervention 2: Placebo
Intervention descriptions: intervention 1: supplied as 100 mg capsules intervention 2: supplied in capsules

Protocol document: