To Evaluate the Safety, Local Tolerability, PK and PD of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas (sBCC)

Study Overview
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Brief Summary
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This was a double-blinded, randomized, vehicle-controlled study in sporadic superficial BCC (sBCC) and nodular BCC (nBCC) patients which consisted of a 21-day screening period, a treatment period of 6 weeks (topical 0.75% LDE225 cream application b.i.d) ending with post treatment biopsies, as safety visit one week after final study drug administration (Day 50), a visit on Day 83 for excision of the treated BCC, and an end of study evaluation (Day 90).

Official Title
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Double-blind, Randomized, Vehicle-controlled Proof of Concept Study on the Efficacy, Safety, Local Tolerability, PK and Pharmacodynamics of Multiple Topical Administrations of LDE225 on Sporadic Superficial and Nodular Skin Basal Cell Carcinomas

Conditions
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Sporadic Superficial and Nodular Skin Basal Cell Carcinomas

Intervention / Treatment
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* Drug: LDE225 0.75%
* Drug: Vehicle


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with one histologically confirmed superficial and nodular basal cell carcinoma (8-20 mm) eligible for surgical excision on selected body areas (scalp, arm, frontal trunk, posterior trunk, upper legs) Exclusion Criteria: Previous treatment of the sBCC that are selected for treatment. Any systemic treatment which is known to affect BCCs esp. cytostatic treatments, retinoids and photodynamic treatments. Dark-skinned persons whose skin color prevents readily assessment of skin reactions Other protocol defined Incl./Excl. criteria may apply.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LDE225 0.75%<br>Participants topically applied 0.75% LDE225 cream twice daily for 6 weeks. | Drug: LDE225 0.75%<br>* 0.75% cream<br>|
| Placebo Comparator: Vehicle<br>Participants topically applied matching placebo cream twice daily for 6 weeks. | Drug: Vehicle<br>* matching placebo cream to 0.75% LDE225 cream<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Evaluation of sBCCs Tumors | The clinical response parameters were defined as (i) complete response (i.e., there is no longer any visible evidence of a lesion consistent with BCC at this site), (ii) partial response (i.e., although a BCC still remains at this site, it has demonstrated a visible decrease in size compared with baseline), and (iii) no response / worsening (i.e., the BCC has not demonstrated any visible decrease in size compared with baseline). | Day 43 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Basal cell carcinomas,, sporadic,, superficial and nodular,, skin