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

Phase 2

Withdrawn

• Conditions: Basal Cell Carcinoma
• Interventions: Drug: LDE225B; Drug: Vehicle

• Registration Number: NCT03070691
• Lead Sponsor: Novartis Pharmaceuticals

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 Description

Not available

Study Timeline

• Study First Submitted: 2010-07-06
• Status Verified: 2017-02
• Study First Submitted QC: 2017-02-28
• Study First Posted: 2017-03-03
• Last Update Submitted: 2017-05-03
• Last Update Posted: 2017-05-05

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
LDE225 0.75% cream	LDE225B	-
Vehicle	Vehicle	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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
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
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
To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue	4 weeks after LDE225 treatment

Trial Locations

Locations (2)

Novartis Investigative Site; 🇬🇷 Athens, Greece

Novartis Investigative site; 🇬🇧 Manchester, United Kingdom