Try the modernized beta website. Learn more about the modernization effort.
Working… Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01350115
Recruitment Status : Completed
First Posted : May 9, 2011
Results First Posted : October 19, 2015
Last Update Posted : October 19, 2015
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief 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.

Condition or disease Intervention/treatment Phase
Basal Cell Carcinoma Gorlin Syndrome Nevoid Basal Cell Carcinoma Syndrome Drug: LDE225 Drug: Placebo Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, 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
Study Start Date : April 2011
Actual Primary Completion Date : October 2012
Actual Study Completion Date : October 2012

Arm Intervention/treatment
Active Comparator: LDE225
Participants received 400 mg once daily.
Drug: LDE225
supplied as 100 mg capsules

Placebo Comparator: Placebo
Participants received matching placebo.
Drug: Placebo
supplied in capsules

Primary Outcome Measures :
  1. Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs) [ Time Frame: Day 113 ]
    The clinical response of the main target (and secondary target, as appropriate) BCC(s) to treatment was evaluated using the following 6-point scale comparing the assessment at the visit to the clinical presentation at Baseline: 0 = Worsening, 1 = No change, 2 = Slight clearance (1-25% improvement), 3 = Moderate clearance (26-75% improvement), 4 = Marked clearance (76-99% improvement),5 = Complete clearance (100% improvement) Complete clearance was defined as no clinical residual signs of carcinoma, as evaluated by the Investigator at a post-Baseline visit, with the exception of post-inflammatory changes such as minimal residual erythema or residual hyper-pigmentation or hypo-pigmentation or residual scarring.

Secondary Outcome Measures :
  1. Histological Clearance Assessment of Main Target BCCs [ Time Frame: day 113 ]
    The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination.

  2. Measure: Disease Burden by BCC Tumor Counts [ Time Frame: Baseline, day 85, and day 113 ]
    BCC tumor counts were performed separately for five body regions: head and neck, trunk back, trunk front (including axillae and groin), upper extremities and lower extremities (including buttocks). During the counting, the BCC tumors, were categorized upon inspection by their longest diameter measurement (<10 mm, 10-19 mm, 20-29 mm, and >+30mm), and also by the type of BCC (superficial, nodular, other). The counts for all of the BCC type and size categories were determined (or estimated if many small lesions) for each body region. The body region counts were summated to provide the overall BCC tumor count.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01350115

Layout table for location information
Novartis Investigative Site
Wien, Austria, A-1090
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
Novartis Investigative Site
Kiel, Germany, 24105
Sponsors and Collaborators
Novartis Pharmaceuticals
Layout table for investigator information
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Layout table for additonal information
Responsible Party: Novartis Pharmaceuticals Identifier: NCT01350115    
Other Study ID Numbers: CLDE225B2209
2010-023819-34 ( EudraCT Number )
First Posted: May 9, 2011    Key Record Dates
Results First Posted: October 19, 2015
Last Update Posted: October 19, 2015
Last Verified: September 2015
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
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
Additional relevant MeSH terms:
Layout table for MeSH terms
Carcinoma, Basal Cell
Basal Cell Nevus Syndrome
Pathologic Processes
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms, Basal Cell
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn