Clinical trial NCT03070691: Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
PHASE3
Conditions: Basal Cell Carcinoma
Keywords: Basal cell carcinoma
Nevoid Basal Cell Carcinoma Syndrome
NBCCS, Gorlin syndrome
Gorlin-Goltz Syndrome
Basal Cell Nevus Syndrome
BCNS, Hedgehog pathway inhibitor
topical
Smoothened
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2017-03-03
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: 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
timeFrame: 4 weeks after LDE225 treatment
Secondary outcomes:
measure: To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS
timeFrame: Treatment phase and up to 4 weeks after LDE225 treatment
measure: To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS
timeFrame: Treatment phase and up to 4 weeks after LDE225 treatment
measure: To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma
timeFrame: 4 weeks after LDE225 treatment
measure: To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue
timeFrame: 4 weeks after LDE225 treatment
Other outcomes:
Locations:
city: Bruxelles
country: Belgium
facility: Novartis Investigative site
city: Leuven
country: Belgium
facility: Novartis Investigative site
city: Waterloo
country: Canada
facility: Novartis Investigative site
state: Ontario
city: Sainte-Foy
country: Canada
facility: Novartis Investigative site
state: Quebec
city: Praha
country: Czechia
facility: Novartis Investigative site
city: Helsinki
country: Finland
facility: Novartis Investigative site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Bonn
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Jena
country: Germany
facility: Novartis Investigative Site
city: Kiel
country: Germany
facility: Novartis Investigative Site
city: Luebeck
country: Germany
facility: Novartis Investigative Site
city: Mainz
country: Germany
facility: Novartis Investigative Site
city: Muenster
country: Germany
facility: Novartis Investigative Site
city: Munich
country: Germany
facility: Novartis Investigative Site
city: Recklinghausen
country: Germany
facility: Novartis Investigative Site
city: Tuebingen
country: Germany
facility: Novartis Investigative Site
city: Wuerzburg
country: Germany
facility: Novartis Investigative Site
city: Athens
country: Greece
facility: Novartis Investigative Site
city: Thessaloniki
country: Greece
facility: Novartis Investigative site
city: Catania
country: Italy
facility: Novartis Investigative site
city: L'Aquila
country: Italy
facility: Novartis Investigative site
city: Napoli
country: Italy
facility: Novartis Investigative site
city: Padova
country: Italy
facility: Novartis Investigative site
city: Siena
country: Italy
facility: Novartis Investigative site
city: Barcelona
country: Spain
facility: Novartis Investigative site
city: Madrid
country: Spain
facility: Novartis Investigative site
city: Lausanne
country: Switzerland
facility: Novartis Investigative site
city: Zuerich
country: Switzerland
facility: Novartis Investigative site
city: Glasgow
country: United Kingdom
facility: Novartis Investigative site
city: London
country: United Kingdom
facility: Novartis Investigative site
city: Manchester
country: United Kingdom
facility: Novartis Investigative site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: