Trial Outcomes & Findings for Vismodegib in Treating Patients With Basal Cell Carcinoma (BCC) (NCT NCT01631331)

Last Updated: 2017-12-08

Results Overview

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.

Recruitment status

COMPLETED

Study phase

EARLY_PHASE1

Target enrollment

15 participants

Primary outcome timeframe

average of 4 months

Results posted on

2017-12-08

Participant Flow

Patients had 1 to 2 target BCCs identified at baseline for surgical excision. n = 13 target lesions n = 30 non-target lesions

Unit of analysis: BCC (target and non-target)

Participant milestones

Participant milestones
Measure	Treatment (Vismodegib and Mohs Surgery) 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 Study STARTED	15 76
Overall Study COMPLETED	11 43
Overall Study NOT COMPLETED	4 33

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Vismodegib and Mohs Surgery) n=11 Participants 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
Age, Categorical <=18 years	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Age, Categorical >=65 years	2 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Sex: Female, Male Female	6 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Sex: Female, Male Male	5 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) Asian	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) White	11 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) More than one race	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.
Region of Enrollment United States	11 participants n=5 Participants • Only patients who were treated with vismodegib for an average of 4 months were included in our analysis.

PRIMARY outcome

Timeframe: average of 4 months

Population: 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.

Outcome measures

Outcome measures
Measure	Treatment (Vismodegib and Mohs Surgery) n=13 BCCs 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
Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated	-26.8 percentage size change from baseline Interval -86.0 to -5.0

SECONDARY outcome

Timeframe: Average of 4 months

Population: 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.

Determination of histologic cure (no residual BCC on the ﬁrst piece of excised tissue) post serial sectioning of parafﬁn embedded Mohs specimens

Outcome measures

Outcome measures
Measure	Treatment (Vismodegib and Mohs Surgery) n=13 BCCs 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
Number of Tumors Demonstrating Histologic Cure	6 BCCs

SECONDARY outcome

Timeframe: average of 22 months

Population: 11 patients completed the trial and 13 target BCCs were excised.

Recurrence rate of BCCs during a 22 month average (range 12 to 28 months) follow up period.

Outcome measures

Outcome measures
Measure	Treatment (Vismodegib and Mohs Surgery) n=13 BCCs 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
Tumor Recurrence Rate of Treated BCCs	1 BCCs

SECONDARY outcome

Timeframe: 4 months (average)

Population: 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.

We measured the length and width of all tumors (target and non-target) before and after vismodegib treatment.

Outcome measures

Outcome measures
Measure	Treatment (Vismodegib and Mohs Surgery) n=43 basal cell carcinomas 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
Tumor Size Measurements Before and After Short Term Vismodegib Treatment	-40 percentage change in tumor size Interval -50.0 to -30.5

Adverse Events

Treatment (Vismodegib and Mohs Surgery)

Serious events: 3 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Vismodegib and Mohs Surgery) n=15 participants at risk 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
Surgical and medical procedures ventriculoperitoneal shunt obstruction	6.7% 1/15 • Number of events 1 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Skin and subcutaneous tissue disorders development of squamous cell carcinoma	13.3% 2/15 • Number of events 2 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).

Other adverse events

Other adverse events
Measure	Treatment (Vismodegib and Mohs Surgery) n=15 participants at risk 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
Investigations aspartate/alanine aminotransferase elevation	13.3% 2/15 • Number of events 2 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
General disorders fatigue	40.0% 6/15 • Number of events 6 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Metabolism and nutrition disorders weight loss	40.0% 6/15 • Number of events 10 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Skin and subcutaneous tissue disorders alopecia	73.3% 11/15 • Number of events 15 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Musculoskeletal and connective tissue disorders myalgia	60.0% 9/15 • Number of events 9 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Gastrointestinal disorders dysgeusia	60.0% 9/15 • Number of events 9 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Gastrointestinal disorders diarrhea	20.0% 3/15 • Number of events 3 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Nervous system disorders depression	13.3% 2/15 • Number of events 2 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Reproductive system and breast disorders reversible amenorrhea	6.7% 1/15 • Number of events 1 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).
Investigations creatine phosphokinase elevation	6.7% 1/15 • Number of events 1 • An average of 24 months We evaluated patients monthly for skin examinations and adverse events (National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4).

Additional Information

Dr. Gina Kwon

Stanford University

Phone: 650-724-3859

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place