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

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

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 C

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.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: 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 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: July 6, 2010
Date Started:
Date Completion:
Last Updated: May 3, 2017
Last Verified: February 2017