Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
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ClinicalTrials.gov Identifier: NCT01350115 |
Recruitment Status :
Completed
First Posted : May 9, 2011
Results First Posted : October 19, 2015
Last Update Posted : October 19, 2015
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Condition or disease | Intervention/treatment | Phase |
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Basal Cell Carcinoma Gorlin Syndrome Nevoid Basal Cell Carcinoma Syndrome | Drug: LDE225 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 10 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Study Start Date : | April 2011 |
Actual Primary Completion Date : | October 2012 |
Actual Study Completion Date : | October 2012 |

Arm | Intervention/treatment |
---|---|
Active Comparator: LDE225
Participants received 400 mg once daily.
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Drug: LDE225
supplied as 100 mg capsules |
Placebo Comparator: Placebo
Participants received matching placebo.
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Drug: Placebo
supplied in capsules |
- Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs) [ Time Frame: 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.
- Histological Clearance Assessment of Main Target BCCs [ Time Frame: 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.
- Measure: Disease Burden by BCC Tumor Counts [ Time Frame: 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.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01350115
Austria | |
Novartis Investigative Site | |
Wien, Austria, A-1090 | |
Belgium | |
Novartis Investigative Site | |
Leuven, Belgium, 3000 | |
Canada, Ontario | |
Novartis Investigative Site | |
Markham, Ontario, Canada, L3P 1A8 | |
Novartis Investigative Site | |
Waterloo, Ontario, Canada, N2J 1C4 | |
Canada, Quebec | |
Novartis Investigative Site | |
Montreal, Quebec, Canada, H2K 4L5 | |
Germany | |
Novartis Investigative Site | |
Kiel, Germany, 24105 |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01350115 |
Other Study ID Numbers: |
CLDE225B2209 2010-023819-34 ( EudraCT Number ) |
First Posted: | May 9, 2011 Key Record Dates |
Results First Posted: | October 19, 2015 |
Last Update Posted: | October 19, 2015 |
Last Verified: | September 2015 |
Basal Cell Carcinoma Gorlin Syndrome, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, Nevoid Basal Cell Carcinoma Syndrome, |
Basal Cell Carcinoma Nevus Syndrome Smo inhibitor, Hedgehog pathway inhibitor BCCs |
Carcinoma Carcinoma, Basal Cell Basal Cell Nevus Syndrome Syndrome Disease Pathologic Processes Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms Neoplasms, Basal Cell Odontogenic Cysts Jaw Cysts |
Bone Cysts Cysts Neoplastic Syndromes, Hereditary Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Jaw Diseases Stomatognathic Diseases Abnormalities, Multiple Congenital Abnormalities Genetic Diseases, Inborn |