Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients Clinical Trial
Official title:
A Double-blind, Randomized, Vehicle-controlled Proof of Concept (PoC) Study to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Topical Administrations of LDE225 (a Specific Smoothened Inhibitor) on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients Followed by an Open Label, Randomized Expansion Group to Test Two Different Strengths of an Improved LDE225 Formulation for Extended Treatment Durations
Part I was a double-blind, randomized, vehicle-controlled Proof of Concept (PoC) study to
evaluate the safety, local tolerability, pharmacokinetics and pharmacodynamics of multiple
topical administrations of LDE225 (a specific Smoothened inhibitor) on skin basal cell
carcinomas in Gorlin's syndrome patients.
Following a 21-day screening period, patients were exposed to multiple doses of topically
applied LDE225 twice daily for 4 weeks in a double-blind manner. The patients returned
weekly for visits where each BCC was clinically evaluated and digital photographs taken.
Local safety and tolerability was also assessed. After the last application of treatment,
biopsies were taken from treated (both vehicle and LDE225) BCCs (three per patient) for
histology, biomarker evaluation and for pharmacokinetics (skin exposure). In addition, a
biopsy from LDE225-treated uninvolved perilesional skin was taken for pharmacokinetic
evaluation. In total, 4 biopsies were taken: 2 for histology and biomarker and 2 for PK.
Part II of this study consisted of a 21-day screening period, a baseline period (directly
before commencing the treatment period) and a treatment period of 6 or 9 weeks, depending on
randomization. A clinical assessment was performed on site on the last treatment day and if
a full clinical response had been observed, approximately 3 weeks after the last treatment
an excision of the BCC(s) would have been performed. The study completion visit occurred
either 1 week after the excision (when this visit was planned) or 1 week after the last
treatment. For a subset of patients, skin biopsies were collected on the last treatment day
and an excision of a BCC was also performed at that same visit.
n/a
Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment