Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS BCC)
This study is currently recruiting participants.
Verified October 2012 by Novartis
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01350115
First received: May 6, 2011
Last updated: October 2, 2012
Last verified: October 2012
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Purpose
This study is a double-blind, randomized, placebo-controlled Proof of Concept (PoC) study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral LDE225 in treatment of skin basal cell carcinomas in patients with Nevoid Basal Cell Carcinoma Syndrome (NBCCS).
The core study starts after a screening period, and patients will be exposed to oral LDE225 once a day for 12 weeks in a double-blind manner. A long-term follow-up period for safety and disease burden assessment is planned after the end of the core study.
The study will be conducted in up to three treatment group cohorts at different doses, progressing to a subsequent cohort once data generated from a previous cohort provides rationale, based on safety and efficacy.
| Condition | Intervention | Phase |
|---|---|---|
|
Basal Cell Carcinoma Gorlin Syndrome Nevoid Basal Cell Carcinoma Syndrome |
Drug: LDE225 Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double Blind (Subject, 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
focal dermal hypoplasia
Gorlin syndrome
oculodentodigital dysplasia
U.S. FDA Resources
Further study details as provided by Novartis:
Primary Outcome Measures:
- Measure: Clinical evaluation of BCC tumors (complete response) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Measure: Histological signs of tumor regression; apoptosis; tumor proliferation [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Measure: Disease burden by BCC tumor counts [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 42 |
| Study Start Date: | April 2011 |
| Estimated Study Completion Date: | October 2012 |
| Estimated Primary Completion Date: | October 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Active Comparator: LDE225 | Drug: LDE225 |
| Placebo Comparator: Placebo | Drug: Placebo |
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01350115
Contacts
| Contact: Novartis Pharmaceuticals | +41-61-324-1111 |
Locations
| Austria | |
| Novartis Investigative Site | Withdrawn |
| Graz, Austria, A-8036 | |
| Novartis Investigative Site | Recruiting |
| Wien, Austria, A-1090 | |
| Belgium | |
| Novartis Investigative Site | Withdrawn |
| Bruxelles, Belgium, 1070 | |
| Novartis Investigative Site | Active, not recruiting |
| Leuven, Belgium, 3000 | |
| Canada, Ontario | |
| Novartis Investigative Site | Terminated |
| Markham, Ontario, Canada, L3P 7N8 | |
| Novartis Investigative Site | Active, not recruiting |
| Waterloo, Ontario, Canada, N2J 1C4 | |
| Canada, Quebec | |
| Novartis Investigative Site | Active, not recruiting |
| Montreal, Quebec, Canada, H2K 4L5 | |
| Germany | |
| Novartis Investigative Site | Active, not recruiting |
| Kiel, Germany, 24105 | |
| Italy | |
| Novartis Investigative Site | Withdrawn |
| L'Aquila, AQ, Italy, 67100 | |
| Novartis Investigative Site | Withdrawn |
| Catania, CT, Italy, 95123 | |
| Novartis Investigative Site | Withdrawn |
| Napoli, Italy, 80131 | |
| Novartis Investigative Site | Withdrawn |
| Padova, Italy, 35128 | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
Additional Information:
No publications provided
| Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT01350115 History of Changes |
| Other Study ID Numbers: | CLDE225B2209, 2010-023819-34 |
| Study First Received: | May 6, 2011 |
| Last Updated: | October 2, 2012 |
| Health Authority: | Canada: Health Canada Austria: Agency for Health and Food Safety Germany: Federal Institute for Drugs and Medical Devices Belgium: Federal Agency for Medicinal Products and Health Products |
Keywords provided by Novartis:
|
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 |
Additional relevant MeSH terms:
|
Tooth Diseases Basal Cell Nevus Syndrome Eye Abnormalities Tooth Abnormalities Carcinoma Carcinoma, Basal Cell Odontogenic Cysts Jaw Cysts Bone Cysts Cysts Neoplasms Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type |
Neoplasms, Basal Cell Neoplastic Syndromes, Hereditary Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Jaw Diseases Stomatognathic Diseases Abnormalities, Multiple Congenital Abnormalities Genetic Diseases, Inborn Eye Diseases Stomatognathic System Abnormalities |
ClinicalTrials.gov processed this record on May 22, 2013