Clinical trial · Interventional
Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
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 Nevoid Basal Cell Carcinoma Syndrome
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Basal Cell Carcinoma | Basal Cell Carcinoma | ONTOLOGY_EXACT | 0.90 |
| Gorlin Syndrome | — | UNRESOLVED | — |
| Nevoid Basal Cell Carcinoma Syndrome | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| LDE225 | Drug | Sonidegib | ALIAS |
| Placebo | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- ACTIVE_COMPARATOR
- label
- LDE225
- description
- Participants received 400 mg once daily.
- interventionNames
- Drug: LDE225
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Participants received matching placebo.
- interventionNames
- Drug: Placebo
Primary outcomes (1)
- measure
- Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)
- timeFrame
- Day 113
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available