Phase 2
N=10
Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
Basal Cell Carcinoma · Gorlin Syndrome · Nevoid Basal Cell Carcinoma Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01350115 ↗Enrolled (actual)
10
Serious AEs
10.0%
Results posted
Oct 2015
Primary outcome: Primary: Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs) — 3; 0; 3; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- LDE225 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Oct 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs) |
3; 0; 3; 0; 1; 0 | — |
| SECONDARY Histological Clearance Assessment of Main Target BCCs |
57; 0; 14; 0; 29; 0 | — |
| SECONDARY Measure: Disease Burden by BCC Tumor Counts |
566; 510; 341; 571; 309; 619 | — |
Summary
This was a phase II, double-blind, randomized, proof-of-concept, dose-ranging trial evaluating the efficacy, safety and pharmacokinetics of oral LDE225 in treatment of adult patients with NBCCS. This was an exploratory study designed to demonstrate preliminary efficacy of LDE225 in this indication. This study included a Screening period of approximately 4 weeks, treatment period duration of 12 weeks with initial follow-up of approximately 6-8 weeks followed by a long-term follow-up period.
Eligibility Criteria
Inclusion Criteria
- Patients with multiple basal cell carcinomas (at least two) and typical presentation of NBCCS.
- Female patients must be women of non-childbearing potential (WONCBP).
Exclusion Criteria
- Use of any topical treatment to treat BCCs, including prescription and over the counter in the 4 weeks prior to first dose of study drug.
- Use of photodynamic therapy (PDT), radiation or systemic treatment known to affect BCCs or neoplasm in the 12 weeks prior to first dose of study drug.
- Patients receiving medications that are recognized to cause rhabdomyolysis or patients with a prior history of rhabdomyolysis.
- Patients with a histologically confirmed diagnosis of locally advanced or metastatic BCC.
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01350115). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.