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

Basal Cell Carcinoma Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01350115
• Other study ID #: CLDE225B2209
• Secondary ID: 2010-023819-34
• Status: Completed
• Phase: Phase 2
• First received: May 6, 2011
• Last updated: September 23, 2015
• Start date: April 2011
• Est. completion date: October 2012

Study information

• Verified date: September 2015
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaAustria: Agency for Health and Food SafetyGermany: Federal Institute for Drugs and Medical DevicesBelgium: Federal Agency for Medicinal Products and Health Products
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: October 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Basal Cell Carcinoma
• Basal Cell Nevus Syndrome
• Carcinoma
• Carcinoma, Basal Cell
• Gorlin Syndrome
• Nevoid Basal Cell Carcinoma Syndrome
• Syndrome

Intervention

Drug:
• LDE225
supplied as 100 mg capsules
• Placebo
supplied in capsules

Locations

Country	Name	City	State
Austria	Novartis Investigative Site	Wien
Belgium	Novartis Investigative Site	Leuven
Canada	Novartis Investigative Site	Markham	Ontario
Canada	Novartis Investigative Site	Montreal	Quebec
Canada	Novartis Investigative Site	Waterloo	Ontario
Germany	Novartis Investigative Site	Kiel

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

Austria,  Belgium,  Canada,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	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.	Day 113	No
Secondary	Histological Clearance Assessment of Main Target BCCs	The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination.	day 113	No
Secondary	Measure: Disease Burden by BCC Tumor Counts	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.	Baseline, day 85, and day 113	No

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