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Trial record 1 of 32 for:    "Nevoid basal cell carcinoma syndrome"
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Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01350115
Recruitment Status : Completed
First Posted : May 9, 2011
Results First Posted : October 19, 2015
Last Update Posted : October 19, 2015
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Basal Cell Carcinoma
Gorlin Syndrome
Nevoid Basal Cell Carcinoma Syndrome
Interventions Drug: LDE225
Drug: Placebo
Enrollment 10
Recruitment Details  
Pre-assignment Details Participants were assigned to one of the following 2 treatment arms in a ratio of 6:1,LDE225 400 mg QD and Placebo QD.
Arm/Group Title LDE225 Placebo
Hide Arm/Group Description Participants received 400 mg once daily. Participants received matching placebo.
Period Title: Core Study (All Patients)
Started 8 2
Safety Anlysis Set 8 2
Pharmacodynamic Set 7 2
Completed 8 2
Not Completed 0 0
Period Title: Long Term Follow-Up (All Patients)
Started 8 2
Completed 7 0
Not Completed 1 2
Reason Not Completed
Lost to Follow-up             0             1
Adverse Event             0             1
Withdrawal by Subject             1             0
Arm/Group Title LDE225 Placebo Total
Hide Arm/Group Description Participants received 400 mg once daily. Participants received matching placebo. Total of all reporting groups
Overall Number of Baseline Participants 8 2 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 2 participants 10 participants
50.5  (9.90) 66  (2.00) 53.6  (10.95)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 2 participants 10 participants
Female
4
  50.0%
0
   0.0%
4
  40.0%
Male
4
  50.0%
2
 100.0%
6
  60.0%
1.Primary Outcome
Title Clinical Clearance Assessment of Main Target Basal Cell Carcinomas (BCCs)
Hide Description 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.
Time Frame Day 113
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LDE225 Placebo
Hide Arm/Group Description:
Participants received 400 mg once daily.
Participants received matching placebo.
Overall Number of Participants Analyzed 7 2
Measure Type: Number
Unit of Measure: Participants
Complete Clearance (100% Improvement) 3 0
Marked Clearance (76-99% Improvement) 3 0
Moderate Clearance (26-75% improvement) 1 0
Slight Clearance (1-25%) 0 1
Worsening 0 1
2.Secondary Outcome
Title Histological Clearance Assessment of Main Target BCCs
Hide Description The main (and secondary, if appropriate) target BCC tumor area(s) was/were excised surgically and sent to a central laboratory for histological examination.
Time Frame day 113
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LDE225 Placebo
Hide Arm/Group Description:
Participants received 400 mg once daily.
Participants received matching placebo.
Overall Number of Participants Analyzed 7 2
Measure Type: Number
Unit of Measure: Percentage of participants
Histological clearance 57 0
Complete clinical (up to day 85) & histological 14 0
Complete clinical (up to day 113) & histological 29 0
3.Secondary Outcome
Title Measure: Disease Burden by BCC Tumor Counts
Hide Description 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.
Time Frame Baseline, day 85, and day 113
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title LDE225 Placebo
Hide Arm/Group Description:
Participants received 400 mg once daily.
Participants received matching placebo.
Overall Number of Participants Analyzed 7 2
Measure Type: Number
Unit of Measure: Number of BCC tumors
Baseline 566 510
Day 85 341 571
Day 113 309 619
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title LDE225 - Core Placebo - Core LDE225 - Long Term Follow-up Placebo - Long Term Follow-up
Hide Arm/Group Description Participants received 400 mg once daily. Participants received matching placebo. [Not Specified] [Not Specified]
All-Cause Mortality
LDE225 - Core Placebo - Core LDE225 - Long Term Follow-up Placebo - Long Term Follow-up
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
LDE225 - Core Placebo - Core LDE225 - Long Term Follow-up Placebo - Long Term Follow-up
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/8 (0.00%)   1/2 (50.00%)   1/8 (12.50%)   0/2 (0.00%) 
Infections and infestations         
Cellulitis  1  0/8 (0.00%)  0/2 (0.00%)  1/8 (12.50%)  0/2 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Basal cell carcinoma  1  0/8 (0.00%)  1/2 (50.00%)  0/8 (0.00%)  0/2 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
LDE225 - Core Placebo - Core LDE225 - Long Term Follow-up Placebo - Long Term Follow-up
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   7/8 (87.50%)   1/2 (50.00%)   2/8 (25.00%)   0/2 (0.00%) 
Cardiac disorders         
Cardiovascular disorder  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Ear and labyrinth disorders         
Vertigo positional  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Eye disorders         
Eyelid irritation  1  0/8 (0.00%)  1/2 (50.00%)  0/8 (0.00%)  0/2 (0.00%) 
Vision blurred  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Gastrointestinal disorders         
Constipation  1  0/8 (0.00%)  1/2 (50.00%)  0/8 (0.00%)  0/2 (0.00%) 
Diarrhoea  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Food poisoning  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Gingival pain  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Nausea  1  2/8 (25.00%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Vomiting  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
General disorders         
Fatigue  1  2/8 (25.00%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Infections and infestations         
Folliculitis  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Laryngitis  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Nasopharyngitis  1  2/8 (25.00%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Postoperative wound infection  1  0/8 (0.00%)  1/2 (50.00%)  0/8 (0.00%)  0/2 (0.00%) 
Sinusitis  1  0/8 (0.00%)  0/2 (0.00%)  1/8 (12.50%)  0/2 (0.00%) 
Wound infection  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Injury, poisoning and procedural complications         
Electric shock  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Humerus fracture  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Investigations         
Alanine aminotransferase increased  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Aspartate aminotransferase increased  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Blood creatine phosphokinase increased  1  2/8 (25.00%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Gamma-glutamyltransferase increased  1  1/8 (12.50%)  0/2 (0.00%)  1/8 (12.50%)  0/2 (0.00%) 
Musculoskeletal and connective tissue disorders         
Back pain  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Muscle spasms  1  3/8 (37.50%)  1/2 (50.00%)  0/8 (0.00%)  0/2 (0.00%) 
Musculoskeletal pain  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Myalgia  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Osteoarthritis  1  0/8 (0.00%)  1/2 (50.00%)  0/8 (0.00%)  0/2 (0.00%) 
Pain in extremity  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Nervous system disorders         
Dizziness  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Dysgeusia  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Headache  1  2/8 (25.00%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Rhinitis allergic  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Skin and subcutaneous tissue disorders         
Alopecia  1  2/8 (25.00%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Neurodermatitis  1  0/8 (0.00%)  0/2 (0.00%)  1/8 (12.50%)  0/2 (0.00%) 
Night sweats  1  1/8 (12.50%)  0/2 (0.00%)  0/8 (0.00%)  0/2 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01350115     History of Changes
Other Study ID Numbers: CLDE225B2209
2010-023819-34 ( EudraCT Number )
First Submitted: May 6, 2011
First Posted: May 9, 2011
Results First Submitted: August 18, 2015
Results First Posted: October 19, 2015
Last Update Posted: October 19, 2015