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| ID | Type | Description | Link |
|---|---|---|---|
| 2010-023819-34 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LDE225 | Active Comparator | Participants received 400 mg once daily. |
|
| Placebo | Placebo Comparator | Participants received matching placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LDE225 | Drug | supplied as 100 mg capsules |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs) | 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. | Day 113 |
| Measure | Description | Time Frame |
|---|---|---|
| Histological Clearance Assessment of Main Target BCCs | The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination. | day 113 |
| Measure: Disease Burden by BCC Tumor Counts |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Vienna | A-1090 | Austria | |||
| Novartis Investigative Site |
Participants were assigned to one of the following 2 treatment arms in a ratio of 6:1,LDE225 400 mg QD and Placebo QD.
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| ID | Title | Description |
|---|---|---|
| FG000 | LDE225 | Participants received 400 mg once daily. |
| FG001 | Placebo | Participants received matching placebo. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Core Study (All Patients) |
|
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| Drug |
supplied in capsules |
|
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. |
| Baseline, day 85, and day 113 |
| Leuven |
| 3000 |
| Belgium |
| Novartis Investigative Site | Markham | Ontario | L3P 1A8 | Canada |
| Novartis Investigative Site | Waterloo | Ontario | N2J 1C4 | Canada |
| Novartis Investigative Site | Montreal | Quebec | H2K 4L5 | Canada |
| Novartis Investigative Site | Kiel | 24105 | Germany |
| Safety Anlysis Set |
|
| Pharmacodynamic Set |
|
| COMPLETED |
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| NOT COMPLETED |
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| Long Term Follow-Up (All Patients) |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | LDE225 | Participants received 400 mg once daily. |
| BG001 | Placebo | Participants received matching placebo. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs) | 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. | 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. | Posted | Number | Participants | Day 113 |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Histological Clearance Assessment of Main Target BCCs | The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination. | 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. | Posted | Number | Percentage of participants | day 113 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Measure: Disease Burden by BCC Tumor Counts | 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. | 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. | Posted | Number | Number of BCC tumors | Baseline, day 85, and day 113 |
|
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | LDE225 - Core | Participants received 400 mg once daily. | 0 | 8 | 7 | 8 | ||
| EG001 | Placebo - Core | Participants received matching placebo. | 1 | 2 | 1 | 2 | ||
| EG002 | LDE225 - Long Term Follow-up | 1 | 8 | 2 | 8 | |||
| EG003 | Placebo - Long Term Follow-up | 0 | 2 | 0 | 2 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cellulitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiovascular disorder | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| Vertigo positional | Ear and labyrinth disorders | MedDRA | Systematic Assessment |
| |
| Eyelid irritation | Eye disorders | MedDRA | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Food poisoning | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Gingival pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Laryngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Postoperative wound infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA | Systematic Assessment |
| |
| Electric shock | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Neurodermatitis | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 |
| ID | Term |
|---|---|
| D002280 | Carcinoma, Basal Cell |
| D001478 | Basal Cell Nevus Syndrome |
| ID | Term |
|---|---|
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D018295 | Neoplasms, Basal Cell |
| D009807 | Odontogenic Cysts |
| D007570 | Jaw Cysts |
| D001845 | Bone Cysts |
| D003560 | Cysts |
| D009386 | Neoplastic Syndromes, Hereditary |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D007571 | Jaw Diseases |
| D009057 | Stomatognathic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
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| ID | Term |
|---|---|
| C561435 | sonidegib |
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| Withdrawal by Subject |
|
| Male |
|
| Moderate Clearance (26-75% improvement) |
|
| Slight Clearance (1-25%) |
|
| Worsening |
|
|
|
|