A Trial to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of LDE225 on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients
Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients
About this trial
This is an interventional treatment trial for Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients focused on measuring Basal Cell Carcinoma, Gorlin Syndrome,, Gorlin-Goltz Syndrome,, Basal Cell Nevus Syndrome,, Nevoid Basal Cell Carcinoma Syndrome,, Basal Cell Carcinoma Nevus Syndrome
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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.
Sites / Locations
- Novartis Investigative Site
- Novartis Investigator Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Active Comparator
Placebo Comparator
Active Comparator
Active Comparator
LDE225 (applied in parallel with vehicle) [Part I]
Vehicle cream (applied in parallel with LDE225 [Part I]
LDE225 0.25% [Part II]
LDE225 0.75% [Part II]
Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant.
Participants were exposed to both topically applied 0.75% LDE225 cream and LDE225 vehicle cream twice daily for 28 days where each treatment was randomized to two different test areas on each participant.
Participants were exposed to topically applied 0.25% LDE225 cream twice daily for 6 weeks.
Participants were exposed to topically applied 0.75% LDE225 cream twice daily where some basal cell carcinomas (BCCs) were teated for 6 weeks and some BCCs were treated for 9 weeks.