Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
A Randomized, Double-blind, Vehicle-controlled, Multicenter Trial of Topically Administered LDE225 Cream (0.75% Bid) to Evaluate Clearance of Basal Cell Carcinoma in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome
1 other identifier
interventional
N/A
10 countries
35
Brief Summary
This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks. Treatment success is defined as complete clinical clearance and complete histological clearance in BCCs.
Trial Health
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 6, 2010
CompletedFirst Posted
Study publicly available on registry
March 3, 2017
CompletedMay 5, 2017
February 1, 2017
July 6, 2010
May 3, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To demonstrate superiority of topical LDE225 versus vehicle in terms of treatment success in patients with NBCCs. Measure: Complete clinical clearance and complete histological clearance of BCCs
4 weeks after LDE225 treatment
Secondary Outcomes (4)
To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS
Treatment phase and up to 4 weeks after LDE225 treatment
To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS
Treatment phase and up to 4 weeks after LDE225 treatment
To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma
4 weeks after LDE225 treatment
To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue
4 weeks after LDE225 treatment
Study Arms (2)
LDE225 0.75% cream
ACTIVE COMPARATORVehicle
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- Adult greater than 18 years old, male or female.
- Patient must be able to understand and communicate with the investigator and comply with the requirements of the study.
- Typical presentation of NBCCS in the opinion of the investigator
- At least one of the major clinical criteria of NBCCS.
- Multiple BCCs during the screening period
You may not qualify if:
- Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator.
- Prior exposure to LDE225.
- Use of systemic treatment for BCC in the 4 weeks prior to Baseline.
- Use of topical treatment or photodynamic therapy (PDT) in the 12 weeks prior to Baseline.
- Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer.
- Clinically significant medical condition, as per judgment of the investigator.
- History of hypersensitivity to any of the ingredient of the study drug.
- Pregnant or nursing (lactating) women
- Women of child-bearing potential and fertile males, UNLESS they are using two birth control methods
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (35)
Novartis Investigative site
Brussels, Belgium
Novartis Investigative site
Leuven, Belgium
Novartis Investigative site
Waterloo, Ontario, Canada
Novartis Investigative site
Sainte-Foy, Quebec, Canada
Novartis Investigative site
Prague, Czechia
Novartis Investigative site
Helsinki, Finland
Novartis Investigative Site
Berlin, Germany
Novartis Investigative Site
Bonn, Germany
Novartis Investigative Site
Dresden, Germany
Novartis Investigative Site
Essen, Germany
Novartis Investigative Site
Frankfurt, Germany
Novartis Investigative Site
Heidelberg, Germany
Novartis Investigative Site
Jena, Germany
Novartis Investigative Site
Kiel, Germany
Novartis Investigative Site
Lübeck, Germany
Novartis Investigative Site
Mainz, Germany
Novartis Investigative Site
Munich, Germany
Novartis Investigative Site
Münster, Germany
Novartis Investigative Site
Recklinghausen, Germany
Novartis Investigative Site
Tübingen, Germany
Novartis Investigative Site
Würzburg, Germany
Novartis Investigative Site
Athens, Greece
Novartis Investigative site
Thessaloniki, Greece
Novartis Investigative site
Catania, Italy
Novartis Investigative site
L’Aquila, Italy
Novartis Investigative site
Napoli, Italy
Novartis Investigative site
Padua, Italy
Novartis Investigative site
Siena, Italy
Novartis Investigative site
Barcelona, Spain
Novartis Investigative site
Madrid, Spain
Novartis Investigative site
Lausanne, Switzerland
Novartis Investigative site
Zurich, Switzerland
Novartis Investigative site
Glasgow, United Kingdom
Novartis Investigative site
London, United Kingdom
Novartis Investigative site
Manchester, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 6, 2010
First Posted
March 3, 2017
Last Updated
May 5, 2017
Record last verified: 2017-02