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Vismodegib in Treating Patients With Basal Cell Carcinoma (BCC)

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ClinicalTrials.gov Identifier: NCT01631331
Recruitment Status : Completed
First Posted : June 29, 2012
Results First Posted : October 2, 2017
Last Update Posted : December 8, 2017
Sponsor:
Collaborators:
National Cancer Institute (NCI)
University Hospitals Cleveland Medical Center
Information provided by (Responsible Party):
Jean Yuh Tang, Stanford University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Basal Cell Carcinoma of the Skin
Recurrent Skin Cancer
Interventions Drug: vismodegib
Procedure: Mohs surgery
Enrollment 15
Recruitment Details  
Pre-assignment Details Patients had 1 to 2 target BCCs identified at baseline for surgical excision. n = 13 target lesions n = 30 non-target lesions
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

Period Title: Overall Study
Number of participants Number of units (BCC (target and non-target))
Started 15 76
Completed 11 43
Not Completed 4 33
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
We enrolled patients with at least 1 biopsy-confirmed BCC of any histologic subtype, more than 5 mm in diameter, eligible for surgical removal. Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
<=18 years
0
   0.0%
Between 18 and 65 years
9
  81.8%
>=65 years
2
  18.2%
[1]
Measure Analysis Population Description: Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Female
6
  54.5%
Male
5
  45.5%
[1]
Measure Analysis Population Description: Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
11
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
[1]
Measure Analysis Population Description: Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Region of Enrollment   [1] 
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 11 participants
11
[1]
Measure Analysis Population Description: Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
1.Primary Outcome
Title Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated
Hide Description At baseline, we selected 1 to 2 tumors per patient for surgery (13 target tumors selected). At baseline,1 Mohs surgeon measured the estimated surgical defect area around the target tumor. For tumors to be excised by Mohs we defined estimated surgical defect as the tumor size plus a 2-mm circumferential margin, presuming tumor clearance after a Mohs stage-1 excision. For the tumor undergoing standard (non-Mohs) excision, we used tumor size plus a standard 4-mm margin11 for the estimated surgical defect. On the day of the surgery, we measured the surgical defect area as the final tumor-free defect after the Mohs procedure or non-Mohs excision immediately before closure. We used the Image J software program (National Institutes of Health, Bethesda, MD) to calculate tumor area (cm2). Only target tumors are included in this analysis.
Time Frame average of 4 months
Hide Outcome Measure Data
Hide Analysis Population Description
Only patients who were treated with vismodegib for an average of 4 months were included in our analysis. Only target tumors are included in this analysis.
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description:

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

Overall Number of Participants Analyzed 11
Overall Number of Units Analyzed
Type of Units Analyzed: BCCs
13
Mean (95% Confidence Interval)
Unit of Measure: percentage size change from baseline
-26.8
(-86 to -5)
2.Secondary Outcome
Title Number of Tumors Demonstrating Histologic Cure
Hide Description Determination of histologic cure (no residual BCC on the first piece of excised tissue) post serial sectioning of paraffin embedded Mohs specimens
Time Frame Average of 4 months
Hide Outcome Measure Data
Hide Analysis Population Description
Patients each had 1 to 2 target BCCs identified at baseline for surgical excision and were treated with vismodegib for an average of 4 months. Only target lesions are included in this analysis.
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description:

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

Overall Number of Participants Analyzed 11
Overall Number of Units Analyzed
Type of Units Analyzed: BCCs
13
Count of Units
Unit of Measure: BCCs
6
  46.2%
3.Secondary Outcome
Title Tumor Recurrence Rate of Treated BCCs
Hide Description Recurrence rate of BCCs during a 22 month average (range 12 to 28 months) follow up period.
Time Frame average of 22 months
Hide Outcome Measure Data
Hide Analysis Population Description
11 patients completed the trial and 13 target BCCs were excised.
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description:

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

Overall Number of Participants Analyzed 11
Overall Number of Units Analyzed
Type of Units Analyzed: BCCs
13
Count of Units
Unit of Measure: BCCs
1
   7.7%
4.Secondary Outcome
Title Tumor Size Measurements Before and After Short Term Vismodegib Treatment
Hide Description We measured the length and width of all tumors (target and non-target) before and after vismodegib treatment.
Time Frame 4 months (average)
Hide Outcome Measure Data
Hide Analysis Population Description
6 of the 11 patients who completed the study had multiple BCCs. We followed 13 target BCCs and 30 non-target BCCs for potential tumor size change from these patients.
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description:

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

Overall Number of Participants Analyzed 11
Overall Number of Units Analyzed
Type of Units Analyzed: Basal cell carcinomas
43
Mean (95% Confidence Interval)
Unit of Measure: percentage change in tumor size
-40
(-50 to -30.5)
Time Frame An average of 24 months
Adverse Event Reporting Description We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
 
Arm/Group Title Treatment (Vismodegib and Mohs Surgery)
Hide Arm/Group Description

Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.

vismodegib: Given PO

Mohs surgery: Undergo Mohs surgery

All-Cause Mortality
Treatment (Vismodegib and Mohs Surgery)
Affected / at Risk (%)
Total   0/15 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Treatment (Vismodegib and Mohs Surgery)
Affected / at Risk (%) # Events
Total   3/15 (20.00%)    
Skin and subcutaneous tissue disorders   
development of squamous cell carcinoma *  2/15 (13.33%)  2
Surgical and medical procedures   
ventriculoperitoneal shunt obstruction *  1/15 (6.67%)  1
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treatment (Vismodegib and Mohs Surgery)
Affected / at Risk (%) # Events
Total   14/15 (93.33%)    
Gastrointestinal disorders   
dysgeusia * [1]  9/15 (60.00%)  9
diarrhea *  3/15 (20.00%)  3
General disorders   
fatigue *  6/15 (40.00%)  6
Investigations   
aspartate/alanine aminotransferase elevation * [2]  2/15 (13.33%)  2
creatine phosphokinase elevation *  1/15 (6.67%)  1
Metabolism and nutrition disorders   
weight loss *  6/15 (40.00%)  10
Musculoskeletal and connective tissue disorders   
myalgia * [3]  9/15 (60.00%)  9
Nervous system disorders   
depression *  2/15 (13.33%)  2
Reproductive system and breast disorders   
reversible amenorrhea *  1/15 (6.67%)  1
Skin and subcutaneous tissue disorders   
alopecia * [4]  11/15 (73.33%)  15
*
Indicates events were collected by non-systematic assessment
[1]
changes in taste
[2]
Changes in lab test results for liver function.
[3]
muscle cramps
[4]
hair loss
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Gina Kwon
Organization: Stanford University
Phone: 650-724-3859
EMail: gkwon@stanford.edu
Layout table for additonal information
Responsible Party: Jean Yuh Tang, Stanford University
ClinicalTrials.gov Identifier: NCT01631331     History of Changes
Other Study ID Numbers: IRB-24313
NCI-2012-01055 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
SKIN0012 ( Other Identifier: OnCore )
First Submitted: June 25, 2012
First Posted: June 29, 2012
Results First Submitted: February 2, 2017
Results First Posted: October 2, 2017
Last Update Posted: December 8, 2017