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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01350115
First received: May 6, 2011
Last updated: June 26, 2013
Last verified: June 2013
  Purpose

This study is a double-blind, randomized, placebo-controlled Proof of Concept (PoC) study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral LDE225 in treatment of skin basal cell carcinomas in patients with Nevoid Basal Cell Carcinoma Syndrome (NBCCS).

The core study starts after a screening period, and patients will be exposed to oral LDE225 once a day for 12 weeks in a double-blind manner. A long-term follow-up period for safety and disease burden assessment is planned after the end of the core study.

The study will be conducted in up to three treatment group cohorts at different doses, progressing to a subsequent cohort once data generated from a previous cohort provides rationale, based on safety and efficacy.


Condition Intervention Phase
Basal Cell Carcinoma
Gorlin Syndrome
Nevoid Basal Cell Carcinoma Syndrome
Drug: LDE225
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Measure: Clinical evaluation of BCC tumors (complete response) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Measure: Histological signs of tumor regression; apoptosis; tumor proliferation [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Measure: Disease burden by BCC tumor counts [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Enrollment: 10
Study Start Date: April 2011
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: LDE225 Drug: LDE225
Placebo Comparator: Placebo Drug: Placebo

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01350115

Locations
Austria
Novartis Investigative Site
Wien, Austria, A-1090
Belgium
Novartis Investigative Site
Leuven, Belgium, 3000
Canada, Ontario
Novartis Investigative Site
Markham, Ontario, Canada, L3P 1A8
Novartis Investigative Site
Waterloo, Ontario, Canada, N2J 1C4
Canada, Quebec
Novartis Investigative Site
Montreal, Quebec, Canada, H2K 4L5
Germany
Novartis Investigative Site
Kiel, Germany, 24105
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01350115     History of Changes
Other Study ID Numbers: CLDE225B2209, 2010-023819-34
Study First Received: May 6, 2011
Last Updated: June 26, 2013
Health Authority: Canada: Health Canada
Austria: Agency for Health and Food Safety
Germany: Federal Institute for Drugs and Medical Devices
Belgium: Federal Agency for Medicinal Products and Health Products

Keywords provided by Novartis:
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

Additional relevant MeSH terms:
Basal Cell Nevus Syndrome
Eye Abnormalities
Tooth Abnormalities
Carcinoma
Carcinoma, Basal Cell
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Basal Cell
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Eye Diseases
Stomatognathic System Abnormalities
Tooth Diseases

ClinicalTrials.gov processed this record on April 15, 2014