Trial Outcomes & Findings for A Study of Vismodegib With Surgery in Participants With Previously Untreated Basal Cell Carcinoma (NCT NCT01898598)
NCT ID: NCT01898598
Last Updated: 2017-05-08
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
18 participants
Primary outcome timeframe
Baseline, MMS visit (Week 12-14)
Results posted on
2017-05-08
Participant Flow
Participant milestones
| Measure |
Vismodegib
Participants received vismodegib 150 milligrams (mg) capsule orally once daily for 12 weeks.
|
Placebo
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
6
|
|
Overall Study
Treated
|
11
|
5
|
|
Overall Study
COMPLETED
|
8
|
4
|
|
Overall Study
NOT COMPLETED
|
4
|
2
|
Reasons for withdrawal
| Measure |
Vismodegib
Participants received vismodegib 150 milligrams (mg) capsule orally once daily for 12 weeks.
|
Placebo
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
3
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Other
|
0
|
1
|
Baseline Characteristics
A Study of Vismodegib With Surgery in Participants With Previously Untreated Basal Cell Carcinoma
Baseline characteristics by cohort
| Measure |
Vismodegib
n=11 Participants
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=5 Participants
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
71.5 years
STANDARD_DEVIATION 10.87 • n=5 Participants
|
73.6 years
STANDARD_DEVIATION 7.27 • n=7 Participants
|
72.2 years
STANDARD_DEVIATION 9.68 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, MMS visit (Week 12-14)Population: ITT population. Here 'Number of participants analyzed' represents participants evaluable for this outcome measure.
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.
Outcome measures
| Measure |
Vismodegib
n=7 Participants
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=4 Participants
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Percent Change in Target Basal Cell Carcinoma (BCC) Expected Surgical Defect Area at Mohs Micrographic Surgery (MMS) Visit
|
31.52 Percent change in surgical defect area
Standard Deviation 35.580
|
46.30 Percent change in surgical defect area
Standard Deviation 20.232
|
SECONDARY outcome
Timeframe: Baseline, MSS Visit (Week 12-14)Population: ITT Population. Here 'Number of participants analyzed' represents participants evaluable for this outcome measure.
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).
Outcome measures
| Measure |
Vismodegib
n=7 Participants
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=4 Participants
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Actual Change in Target BCC Expected Surgical Defect Area at MMS Visit
|
70.16 Square millimeter (mm^2)
Standard Deviation 82.483
|
93.33 Square millimeter (mm^2)
Standard Deviation 35.768
|
SECONDARY outcome
Timeframe: Baseline, MMS visit (Week 12-14)Population: ITT. Here 'Number of participants analyzed' represents participants evaluable for this outcome measure.
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.
Outcome measures
| Measure |
Vismodegib
n=6 Participants
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=4 Participants
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Percentage Change in Target BCC Actual Tumor-Free Margin Excision Area at MMS Visit
|
-12.24 Percent change in margin excision area
Standard Deviation 78.775
|
25.29 Percent change in margin excision area
Standard Deviation 65.763
|
SECONDARY outcome
Timeframe: MMS visit (Week 12-14)Population: ITT population.
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.
Outcome measures
| Measure |
Vismodegib
n=11 Participants
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=5 Participants
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Percentage of Participants With Clinical Response
|
18.2 Percentage of participants
Interval 2.3 to 51.8
|
40.0 Percentage of participants
Interval 5.3 to 85.3
|
SECONDARY outcome
Timeframe: MMS visit (Week 12-14)Population: ITT population.
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.
Outcome measures
| Measure |
Vismodegib
n=11 Participants
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=5 Participants
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Percentage of Participants With Skip Area
|
0.0 Percentage of participants
Interval 0.0 to 28.5
|
40.0 Percentage of participants
Interval 5.3 to 85.3
|
SECONDARY outcome
Timeframe: Baseline, 12, 24, and 52 weeks post MMS Visit (MMS Visit = Week 12-14)Population: 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.
Outcome measures
Outcome data not reported
Adverse Events
Vismodegib
Serious events: 3 serious events
Other events: 9 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Vismodegib
n=11 participants at risk
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=5 participants at risk
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Gastric ulcer
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Oesophagitis
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bladder neoplasm
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant melanoma
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
Other adverse events
| Measure |
Vismodegib
n=11 participants at risk
Participants received vismodegib 150 mg capsule orally once daily for 12 weeks.
|
Placebo
n=5 participants at risk
Participants received matching placebo to vismodegib capsule orally once daily for 12 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
27.3%
3/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
General disorders
Fatigue
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
General disorders
Influenza like illness
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Infections and infestations
Bronchitis
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Injury, poisoning and procedural complications
Laceration
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
63.6%
7/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Dysgeusia
|
45.5%
5/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Ageusia
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Nervous system disorders
Nerve compression
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
27.3%
3/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
0.00%
0/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
20.0%
1/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
|
Vascular disorders
Hypertension
|
9.1%
1/11 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
0.00%
0/5 • Baseline up to 52 weeks after last study treatment (Last Study Treatment = Week 12)
Safety population, included all participants who received at least one dose of study treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER