Trial · NCT01350115

Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

NCT01350115, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2015-10-19

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS). Trials, record NCT01350115. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01350115

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
NCT number: NCT01350115
Status: Completed
Phases: Phase 2
Enrollment: 10 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Basal Cell Carcinoma, Gorlin Syndrome, Nevoid Basal Cell Carcinoma Syndrome
Interventions: LDE225, Placebo
First posted: 2011-05-09
Last updated: 2015-10-19
Other registries' IDs: eudract:2010-023819-34
Results first posted: 2015-10-19

Source ClinicalTrials.gov

Registered outcomes3

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Day 113	The clinical response of the main target (and secondary target, as appropriate) BCC(s) to treatment was evaluated using the following 6-point scale comparing the assessment at the visit to the clinical presentation at Baseline: 0 = Worsening, 1 = No change, 2 = Slight clearance (1-25% improvement), 3 = Moderate clearance (26-75% improvement), 4 = Marked clearance (76-99% improvement),5 = Complete clearance (100% improvement) Complete clearance was defined as no clinical residual signs of carcinoma, as evaluated by the Investigator at a post-Baseline visit, with the exception of post-inflammatory changes such as minimal residual erythema or residual hyper-pigmentation or hypo-pigmentation or residual scarring.
Secondary	Histological Clearance Assessment of Main Target BCCs	day 113	The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination.
Secondary	Measure: Disease Burden by BCC Tumor Counts	Baseline, day 85, and day 113	BCC tumor counts were performed separately for five body regions: head and neck, trunk back, trunk front (including axillae and groin), upper extremities and lower extremities (including buttocks). During the counting, the BCC tumors, were categorized upon inspection by their longest diameter measurement (\<10 mm, 10-19 mm, 20-29 mm, and \>+30mm), and also by the type of BCC (superficial, nodular, other). The counts for all of the BCC type and size categories were determined (or estimated if many small lesions) for each body region. The body region counts were summated to provide the overall BCC tumor count.

Source ClinicalTrials.gov

Outcomes3

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Day 113	All participants, who had evaluable (or complete) pharmacodynamic (PD) or biomarker parameter data and were without protocol deviations with significant impact on the PD data, were included in the PD analysis set.	Participants	NUMBER
SECONDARY	Histological Clearance Assessment of Main Target BCCs	day 113	All participants, who had evaluable (or complete) pharmacodynamic (PD) or biomarker parameter data and were without protocol deviations with significant impact on the PD data, were included in the PD analysis set.	Percentage of participants	NUMBER
SECONDARY	Measure: Disease Burden by BCC Tumor Counts	Baseline, day 85, and day 113	All participants, who had evaluable (or complete) pharmacodynamic (PD) or biomarker parameter data and were without protocol deviations with significant impact on the PD data, were included in the PD analysis set.	Number of BCC tumors	NUMBER

Source ClinicalTrials.gov

Results by group22

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	LDE225	Complete Clearance (100% Improvement)	3	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Placebo	Complete Clearance (100% Improvement)	0	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	LDE225	Marked Clearance (76-99% Improvement)	3	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Placebo	Marked Clearance (76-99% Improvement)	0	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	LDE225	Moderate Clearance (26-75% improvement)	1	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Placebo	Moderate Clearance (26-75% improvement)	0	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	LDE225	Slight Clearance (1-25%)	0	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Placebo	Slight Clearance (1-25%)	1	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	LDE225	Worsening	0	Participants
Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)	Placebo	Worsening	1	Participants

1 to 10 of 22Source ClinicalTrials.gov

See all 22 results by group

Participant flow16

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Core Study (All Patients)	STARTED	LDE225	8
Core Study (All Patients)	STARTED	Placebo	2
Core Study (All Patients)	Safety Anlysis Set	LDE225	8
Core Study (All Patients)	Safety Anlysis Set	Placebo	2
Core Study (All Patients)	Pharmacodynamic Set	LDE225	7
Core Study (All Patients)	Pharmacodynamic Set	Placebo	2
Core Study (All Patients)	COMPLETED	LDE225	8
Core Study (All Patients)	COMPLETED	Placebo	2
Core Study (All Patients)	NOT COMPLETED	LDE225	0
Core Study (All Patients)	NOT COMPLETED	Placebo	0

1 to 10 of 16Source ClinicalTrials.gov

See all 16 participant flow

Adverse events156

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Cardiovascular disorder	Cardiac disorders	LDE225 - Core	1	8
other	Cardiovascular disorder	Cardiac disorders	Placebo - Core	0	2
other	Cardiovascular disorder	Cardiac disorders	LDE225 - Long Term Follow-up	0	8
other	Cardiovascular disorder	Cardiac disorders	Placebo - Long Term Follow-up	0	2
other	Vertigo positional	Ear and labyrinth disorders	LDE225 - Core	1	8
other	Vertigo positional	Ear and labyrinth disorders	Placebo - Core	0	2
other	Vertigo positional	Ear and labyrinth disorders	LDE225 - Long Term Follow-up	0	8
other	Vertigo positional	Ear and labyrinth disorders	Placebo - Long Term Follow-up	0	2
other	Eyelid irritation	Eye disorders	LDE225 - Core	0	8
other	Eyelid irritation	Eye disorders	Placebo - Core	1	2

1 to 10 of 156Source ClinicalTrials.gov

See all 156 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	LDE225 - Core
Total, all-cause mortality	Placebo - Core
Total, all-cause mortality	LDE225 - Long Term Follow-up
Total, all-cause mortality	Placebo - Long Term Follow-up
Total, other adverse events	LDE225 - Core	7	8
Total, other adverse events	Placebo - Core	1	2
Total, other adverse events	LDE225 - Long Term Follow-up	2	8
Total, other adverse events	Placebo - Long Term Follow-up	0	2
Total, serious adverse events	LDE225 - Core	0	8
Total, serious adverse events	Placebo - Core	1	2

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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