Clinical Trial: Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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 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.
Original Primary Outcome: Measure: Clinical evaluation of BCC tumors (complete response) [ Time Frame: 12 weeks ]
Current Secondary Outcome:
- Histological Clearance Assessment of Main Target BCCs [ Time Frame: day 113 ]The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination.
- Measure: Disease Burden by BCC Tumor Counts [ Time Frame: Baseline, day 85, and day 113 ]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.
Original Secondary Outcome:
- Measure: Histological signs of tumor regression; apoptosis; tumor proliferation [ Time Frame: 12 weeks ]
- Measure: Disease burden by BCC tumor counts [ Time Frame: 12 weeks ]
Information By: Novartis
Dates:
Date Received: May 6, 2011
Date Started: April 2011
Date Completion:
Last Updated: September 23, 2015
Last Verified: September 2015
