Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

A Randomized, Double-blind, Vehicle-controlled, Multicenter Trial of Topically Administered LDE225 Cream (0.75% Bid) to Evaluate Clearance of Basal Cell Carcinoma in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome

Status

Withdrawn

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03070691

Enrollment

Registered

2017-03-03

Start date

Unknown

Completion date

Unknown

Last updated

2017-05-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Basal Cell Carcinoma

Keywords

Basal cell carcinoma, Nevoid Basal Cell Carcinoma Syndrome, NBCCS, Gorlin syndrome, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, BCNS, Hedgehog pathway inhibitor, topical, Smoothened

Brief summary

This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks. Treatment success is defined as complete clinical clearance and complete histological clearance in BCCs.

Interventions

DRUGLDE225B

Cream, 2x daily

DRUGVehicle

cream, 2x daily

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Adult greater than 18 years old, male or female. 2. Patient must be able to understand and communicate with the investigator and comply with the requirements of the study. 3. Typical presentation of NBCCS in the opinion of the investigator 4. At least one of the major clinical criteria of NBCCS. 5. Multiple BCCs during the screening period

Exclusion criteria

1. Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator. 2. Prior exposure to LDE225. 3. Use of systemic treatment for BCC in the 4 weeks prior to Baseline. 4. Use of topical treatment or photodynamic therapy (PDT) in the 12 weeks prior to Baseline. 5. Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer. 6. Clinically significant medical condition, as per judgment of the investigator. 7. History of hypersensitivity to any of the ingredient of the study drug. 8. Pregnant or nursing (lactating) women 9. Women of child-bearing potential and fertile males, UNLESS they are using two birth control methods Other protocol-defined inclusion/

Design outcomes

Primary

Measure	Time frame
To demonstrate superiority of topical LDE225 versus vehicle in terms of treatment success in patients with NBCCs. Measure: Complete clinical clearance and complete histological clearance of BCCs	4 weeks after LDE225 treatment

Secondary

Measure	Time frame
To assess the safety of topical treatment with LDE225, compared to vehicle. Measure: Reported systemic adverse events in patients with NBCCS	Treatment phase and up to 4 weeks after LDE225 treatment
To evaluate the local tolerability of topical treatment with LDE225, compared to vehicle. Measure:Reported local adverse events in patients with NBCCS	Treatment phase and up to 4 weeks after LDE225 treatment
To assess the rate of complete clinical clearance of BCCs in patients with NBCCS. Measure: No clinical residual signs of carcinoma	4 weeks after LDE225 treatment
To assess the rate of complete histological clearance of BCCs in patients with NBCCS. Measure: No residual tumor tissue	4 weeks after LDE225 treatment

Countries

Belgium, Canada, Czechia, Finland, Germany, Greece, Italy, Spain, Switzerland, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026