CompletedPhase 2

Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

Austria, Belgium, Canada10 participantsStarted 2011-04

Plain-language 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.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Patients with multiple basal cell carcinomas (at least two) and typical presentation of NBCCS. * Female patients must be women of non-childbearing potential (WONCBP). Exclusion Criteria: * Use of any topical treatment to treat BCCs, including prescription and over the counter in the 4 weeks prior to first dose of study drug. * Use of photodynamic therapy (PDT), radiation or systemic treatment known to affect BCCs or neoplasm in the 12 weeks prior to first dose of study drug. * Patients receiving medications that are recognized to cause rhabdomyolysis or patients with a prior history of rhabdomyolysis. * Patients with a histologically confirmed diagnosis of locally advanced or metastatic BCC. Other protocol-defined inclusion/exclusion criteria may apply

What they're measuring

Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)

Timeframe: Day 113

Trial details

NCT IDNCT01350115

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2012-10

Results submitted2015-08-18