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

• Status: Withdrawn
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

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