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A Study of Vismodegib With Surgery in Participants With Previously Untreated Basal Cell Carcinoma

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ClinicalTrials.gov Identifier: NCT01898598
Recruitment Status : Terminated
First Posted : July 12, 2013
Results First Posted : May 8, 2017
Last Update Posted : May 8, 2017
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Basal Cell Carcinoma
Interventions Drug: Placebo
Drug: Vismodegib
Enrollment 18
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description Participants received vismodegib 150 milligrams (mg) capsule orally once daily for 12 weeks. Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Period Title: Overall Study
Started 12 6
Treated 11 5
Completed 8 4
Not Completed 4 2
Reason Not Completed
Adverse Event             1             0
Withdrawal by Subject             3             0
Lost to Follow-up             0             1
Other             0             1
Arm/Group Title Vismodegib Placebo Total
Hide Arm/Group Description Participants received vismodegib 150 mg capsule orally once daily for 12 weeks. Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks. Total of all reporting groups
Overall Number of Baseline Participants 11 5 16
Hide Baseline Analysis Population Description
The Intent-to-treat (ITT) population included all randomized participants who received at least one dose of study treatment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 5 participants 16 participants
71.5  (10.87) 73.6  (7.27) 72.2  (9.68)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 5 participants 16 participants
Female
1
   9.1%
1
  20.0%
2
  12.5%
Male
10
  90.9%
4
  80.0%
14
  87.5%
1.Primary Outcome
Title Percent Change in Target Basal Cell Carcinoma (BCC) Expected Surgical Defect Area at Mohs Micrographic Surgery (MMS) Visit
Hide Description The percent change in target BCC expected surgical defect area was defined as ([baseline expected surgical defect area − expected surgical defect area at MMS visit]/ baseline expected surgical defect area) × 100 percent (%) where expected surgical defect area was manually outlined on a digital photograph and measured by a computer (computer aided planimetry). MMS visit was defined as the visit that occurred within 2 weeks of the last study treatment.
Time Frame Baseline, MMS visit (Week 12-14)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population. Here 'Number of participants analyzed' represents participants evaluable for this outcome measure.
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description:
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: Percent change in surgical defect area
31.52  (35.580) 46.30  (20.232)
2.Secondary Outcome
Title Actual Change in Target BCC Expected Surgical Defect Area at MMS Visit
Hide Description Actual change was defined as (baseline expected surgical defect area - expected surgical defect area at MMS visit). MMS visit was defined as the visit that occurred within 2 weeks of the last study treatment. Expected surgical defect area was manually outlined on a digital photograph and measured by a computer (computer aided planimetry).
Time Frame Baseline, MSS Visit (Week 12-14)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT Population. Here 'Number of participants analyzed' represents participants evaluable for this outcome measure.
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description:
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Overall Number of Participants Analyzed 7 4
Mean (Standard Deviation)
Unit of Measure: Square millimeter (mm^2)
70.16  (82.483) 93.33  (35.768)
3.Secondary Outcome
Title Percentage Change in Target BCC Actual Tumor-Free Margin Excision Area at MMS Visit
Hide Description Percent change in target BCC actual tumor-free margin excision area was defined as = (expected surgical defect area pre-treatment - actual tumor-free margin excision area at MMS visit) / expected surgical defect area pre-treatment) * 100%. The actual tumor-free margin excision area (includes 2 millimeters [mm] margin) was measured during MMS. The area was photographed and traced on the digital photograph then calculated by computer-aided planimetry. MMS visit was defined as the visit that occurred within 2 weeks of the last study treatment.
Time Frame Baseline, MMS visit (Week 12-14)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT. Here 'Number of participants analyzed' represents participants evaluable for this outcome measure.
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description:
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Overall Number of Participants Analyzed 6 4
Mean (Standard Deviation)
Unit of Measure: Percent change in margin excision area
-12.24  (78.775) 25.29  (65.763)
4.Secondary Outcome
Title Percentage of Participants With Clinical Response
Hide Description Clinical response was defined as a complete response (CR) or partial response (PR) at the post-treatment MMS excision. CR was defined as no histological evidence of BCC. PR was defined as a reduction of at least 50 % in the expected surgical defect area with histologic evidence of residual BCC. MMS visit was defined the visit that occurred within 2 weeks of the last study treatment.
Time Frame MMS visit (Week 12-14)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description:
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Overall Number of Participants Analyzed 11 5
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
18.2
(2.3 to 51.8)
40.0
(5.3 to 85.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Vismodegib, Placebo
Comments The 95 % confidence interval (CI) for the difference in response rates was computed using the exact unconditional confidence limits method.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference in Clinical Response Rates
Estimated Value -21.8
Confidence Interval (2-Sided) 95%
-71.6 to 32.1
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Percentage of Participants With Skip Area
Hide Description Skip area was defined as the presence of non-contiguous residual tumor at the MMS visit, as determined by an independent dermatopathologist. MMS visit occurred within 2 weeks of the last study treatment.
Time Frame MMS visit (Week 12-14)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description:
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Overall Number of Participants Analyzed 11 5
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
0.0
(0.0 to 28.5)
40.0
(5.3 to 85.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Vismodegib, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Percentage Difference
Estimated Value -40.0
Confidence Interval (2-Sided) 95%
-85.3 to 14.2
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Percentage of Participants With BCC Recurrence
Hide Description [Not Specified]
Time Frame Baseline, 12, 24, and 52 weeks post MMS Visit (MMS Visit = Week 12-14)
Hide Outcome Measure Data
Hide Analysis Population Description
This outcome was based on the cumulative data post MMS Visit and due to early study termination with very few enrolled participants, complete data for this outcome measure could not be collected.
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description:
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Adverse Event Reporting Description Safety population, included all participants who received at least one dose of study treatment.
 
Arm/Group Title Vismodegib Placebo
Hide Arm/Group Description Participants received vismodegib 150 mg capsule orally once daily for 12 weeks. Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
All-Cause Mortality
Vismodegib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Vismodegib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   3/11 (27.27%)   0/5 (0.00%) 
Gastrointestinal disorders     
Gastric ulcer * 1  1/11 (9.09%)  0/5 (0.00%) 
Oesophagitis * 1  1/11 (9.09%)  0/5 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Bladder neoplasm * 1  1/11 (9.09%)  0/5 (0.00%) 
Malignant melanoma * 1  1/11 (9.09%)  0/5 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA v18.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Vismodegib Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   9/11 (81.82%)   2/5 (40.00%) 
Gastrointestinal disorders     
Diarrhoea * 1  3/11 (27.27%)  1/5 (20.00%) 
Vomiting * 1  0/11 (0.00%)  1/5 (20.00%) 
General disorders     
Fatigue * 1  1/11 (9.09%)  0/5 (0.00%) 
Influenza like illness * 1  0/11 (0.00%)  1/5 (20.00%) 
Infections and infestations     
Bronchitis * 1  1/11 (9.09%)  0/5 (0.00%) 
Injury, poisoning and procedural complications     
Laceration * 1  0/11 (0.00%)  1/5 (20.00%) 
Fall * 1  0/11 (0.00%)  1/5 (20.00%) 
Metabolism and nutrition disorders     
Decreased appetite * 1  1/11 (9.09%)  0/5 (0.00%) 
Musculoskeletal and connective tissue disorders     
Muscle spasms * 1  7/11 (63.64%)  1/5 (20.00%) 
Arthralgia * 1  0/11 (0.00%)  1/5 (20.00%) 
Muscular weakness * 1  1/11 (9.09%)  0/5 (0.00%) 
Pain in extremity * 1  0/11 (0.00%)  1/5 (20.00%) 
Nervous system disorders     
Dysgeusia * 1  5/11 (45.45%)  0/5 (0.00%) 
Ageusia * 1  1/11 (9.09%)  0/5 (0.00%) 
Nerve compression * 1  1/11 (9.09%)  0/5 (0.00%) 
Psychiatric disorders     
Anxiety * 1  0/11 (0.00%)  1/5 (20.00%) 
Confusional state * 1  0/11 (0.00%)  1/5 (20.00%) 
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain * 1  0/11 (0.00%)  1/5 (20.00%) 
Sinus congestion * 1  0/11 (0.00%)  1/5 (20.00%) 
Skin and subcutaneous tissue disorders     
Alopecia * 1  3/11 (27.27%)  1/5 (20.00%) 
Skin ulcer * 1  0/11 (0.00%)  1/5 (20.00%) 
Vascular disorders     
Hypertension * 1  1/11 (9.09%)  0/5 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA v18.1
The study was discontinued early by the Sponsor and due to a small sample size, no conclusions can be drawn.
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 Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor’s intellectual property rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann-La Roche
Phone: 800-821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01898598     History of Changes
Other Study ID Numbers: ML28726
First Submitted: July 3, 2013
First Posted: July 12, 2013
Results First Submitted: March 27, 2017
Results First Posted: May 8, 2017
Last Update Posted: May 8, 2017