A 2-part, Randomized Study to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Topical Administrations of LDE225 (a Specific Smoothened Inhibitor) on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients
- Patients with multiple basal cell carcinomas and Gorlin syndrome, or patients with
multiple basal cell carcinomas and a mutation in the PTCH1 gene at chromosome 9q22.3
- Previous treatment of the BCC's that are selected for treatment.
- Any systemic treatment which is known to affect BCCs esp. cytostatic treatments,
retinoids and photodynamic treatments.
Other protocol defined Incl./Excl. criteria may apply.
Type of Study:
Allocation: Randomized, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Clinical evaluation of BCC tumors (complete response)
Outcome Time Frame:
4 weeks/6 weeks/9 weeks
Austria: Agency for Health and Food Safety
- Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients
- Basal Cell Carcinoma
- Gorlin Syndrome,
- Gorlin-Goltz Syndrome,
- Basal Cell Nevus Syndrome,
- Nevoid Basal Cell Carcinoma Syndrome,
- Basal Cell Carcinoma Nevus Syndrome
- Basal Cell Nevus Syndrome
- Eye Abnormalities
- Tooth Abnormalities
- Carcinoma, Basal Cell