Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

Basal Cell Carcinoma Clinical Trial

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03070691
• Other study ID #: CLDE225B2307
• Status: Withdrawn
• Phase: Phase 2/Phase 3
• First received: July 6, 2010
• Last updated: May 3, 2017

Study information

• Verified date: February 2017
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Basal Cell Carcinoma
• Basal Cell Nevus Syndrome
• Carcinoma
• Carcinoma, Basal Cell
• Syndrome

Intervention

Drug:
• LDE225B
Cream, 2x daily
• Vehicle
cream, 2x daily

Locations

Country	Name	City	State
Belgium	Novartis Investigative site	Bruxelles
Belgium	Novartis Investigative site	Leuven
Canada	Novartis Investigative site	Sainte-Foy	Quebec
Canada	Novartis Investigative site	Waterloo	Ontario
Czechia	Novartis Investigative site	Praha
Finland	Novartis Investigative site	Helsinki
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
Greece	Novartis Investigative Site	Athens
Greece	Novartis Investigative site	Thessaloniki
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
Spain	Novartis Investigative site	Barcelona
Spain	Novartis Investigative site	Madrid
Switzerland	Novartis Investigative site	Lausanne
Switzerland	Novartis Investigative site	Zuerich
United Kingdom	Novartis Investigative site	Glasgow
United Kingdom	Novartis Investigative site	London
United Kingdom	Novartis Investigative site	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

Belgium,  Canada,  Czechia,  Finland,  Germany,  Greece,  Italy,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue		4 weeks after LDE225 treatment