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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-04-30

Study Completion Date

2012-10-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Basal Cell Carcinoma Gorlin Syndrome Nevoid Basal Cell Carcinoma Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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LDE225

Participants received 400 mg once daily.

Group Type ACTIVE_COMPARATOR

LDE225

Intervention Type DRUG

supplied as 100 mg capsules

Placebo

Participants received matching placebo.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

supplied in capsules

Interventions

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LDE225

supplied as 100 mg capsules

Intervention Type DRUG

Placebo

supplied in capsules

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No