Trial Information
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 Nevoid Basal Cell Carcinoma Syndrome
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
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Outcome Measure:
Measure: Clinical evaluation of BCC tumors (complete response)
Outcome Time Frame:
12 weeks
Safety Issue:
No
Principal Investigator
Novartis Pharmaceuticals
Investigator Role:
Study Director
Investigator Affiliation:
Novartis Pharmaceuticals
Authority:
Canada: Health Canada
Study ID:
CLDE225B2209
NCT ID:
NCT01350115
Start Date:
April 2011
Completion Date:
October 2012
Related Keywords:
- Basal Cell Carcinoma
- Gorlin Syndrome
- Nevoid Basal Cell Carcinoma Syndrome
- 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
- Basal Cell Nevus Syndrome
- Eye Abnormalities
- Tooth Abnormalities
- Carcinoma
- Carcinoma, Basal Cell