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Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult 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.
 
ClinicalTrials.gov Identifier: NCT03070691
Recruitment Status : Withdrawn
First Posted : March 3, 2017
Last Update Posted : May 5, 2017
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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.

Condition or disease Intervention/treatment Phase
Basal Cell Carcinoma Drug: LDE225B Drug: Vehicle Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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


Arm Intervention/treatment
Active Comparator: LDE225 0.75% cream Drug: LDE225B
Cream, 2x daily

Placebo Comparator: Vehicle Drug: Vehicle
cream, 2x daily




Primary Outcome Measures :
  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 [ Time Frame: 4 weeks after LDE225 treatment ]

Secondary Outcome Measures :
  1. To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS [ Time Frame: Treatment phase and up to 4 weeks after LDE225 treatment ]
  2. To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS [ Time Frame: Treatment phase and up to 4 weeks after LDE225 treatment ]
  3. To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma [ Time Frame: 4 weeks after LDE225 treatment ]
  4. To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue [ Time Frame: 4 weeks after LDE225 treatment ]


Information from the National Library of Medicine

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

Inclusion Criteria:

  1. Adult greater than 18 years old, male or female.
  2. Patient must be able to understand and communicate with the investigator and comply with the requirements of the study.
  3. Typical presentation of NBCCS in the opinion of the investigator
  4. At least one of the major clinical criteria of NBCCS.
  5. Multiple BCCs during the screening period

Exclusion Criteria:

  1. Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator.
  2. Prior exposure to LDE225.
  3. Use of systemic treatment for BCC in the 4 weeks prior to Baseline.
  4. Use of topical treatment or photodynamic therapy (PDT) in the 12 weeks prior to Baseline.
  5. Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer.
  6. Clinically significant medical condition, as per judgment of the investigator.
  7. History of hypersensitivity to any of the ingredient of the study drug.
  8. Pregnant or nursing (lactating) women
  9. Women of child-bearing potential and fertile males, UNLESS they are using two birth control methods

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 ClinicalTrials.gov identifier (NCT number): NCT03070691


Locations
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Belgium
Novartis Investigative site
Bruxelles, Belgium
Novartis Investigative site
Leuven, Belgium
Canada, Ontario
Novartis Investigative site
Waterloo, Ontario, Canada
Canada, Quebec
Novartis Investigative site
Sainte-Foy, Quebec, Canada
Czechia
Novartis Investigative site
Praha, Czechia
Finland
Novartis Investigative site
Helsinki, Finland
Germany
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
Luebeck, Germany
Novartis Investigative Site
Mainz, Germany
Novartis Investigative Site
Muenster, Germany
Novartis Investigative Site
Munich, Germany
Novartis Investigative Site
Recklinghausen, Germany
Novartis Investigative Site
Tuebingen, Germany
Novartis Investigative Site
Wuerzburg, Germany
Greece
Novartis Investigative Site
Athens, Greece
Novartis Investigative site
Thessaloniki, Greece
Italy
Novartis Investigative site
Catania, Italy
Novartis Investigative site
L'Aquila, Italy
Novartis Investigative site
Napoli, Italy
Novartis Investigative site
Padova, Italy
Novartis Investigative site
Siena, Italy
Spain
Novartis Investigative site
Barcelona, Spain
Novartis Investigative site
Madrid, Spain
Switzerland
Novartis Investigative site
Lausanne, Switzerland
Novartis Investigative site
Zuerich, Switzerland
United Kingdom
Novartis Investigative site
Glasgow, United Kingdom
Novartis Investigative site
London, United Kingdom
Novartis Investigative site
Manchester, United Kingdom
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03070691    
Other Study ID Numbers: CLDE225B2307
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: May 5, 2017
Last Verified: February 2017

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Basal cell carcinoma
Nevoid Basal Cell Carcinoma Syndrome
NBCCS, Gorlin syndrome
Gorlin-Goltz Syndrome
Basal Cell Nevus Syndrome
BCNS, Hedgehog pathway inhibitor
topical
Smoothened
Additional relevant MeSH terms:
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Carcinoma
Carcinoma, Basal Cell
Basal Cell Nevus Syndrome
Syndrome
Disease
Pathologic Processes
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Basal Cell
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn