Trial Outcomes & Findings for A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma (NCT NCT00833417)
NCT ID: NCT00833417
Last Updated: 2026-05-11
Results Overview
OR=complete (CR) or partial response (PR). Metastatic-CR:Disappearance of all targets. PR:≥30% decreased sum of longest diameter (SLD) of targets compared to baseline (B). Locally advanced-Response=No progressive disease (PD) and ≥30% decreased SLD from baseline (radiography \[R\]) or ≥30% decreased SLD from B (externally visible dimension \[EVD\]) or completely resolved ulceration. CR:Response with no residual BCC on tumor biopsy (otherwise response was PR). PD:Any of ≥20% increased SLD from nadir (R or EVD), new ulceration, new lesions (R or physical exam) or non-target lesion progression by R.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
104 participants
Primary outcome timeframe
At Baseline and once every 8 weeks thereafter (responses confirmed within ≥4 weeks) until the end of study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)
Results posted on
2026-05-11
Participant Flow
The study population consisted of patients ≥ 18 years old with a histologically confirmed diagnosis of advanced basal cell carcinoma (BCC), either metastatic or locally advanced BCC. Enrollment of patients with locally advanced BCC was limited to 80 of a planned total of 100 patients. Both cohorts received the same vismodegib 150 mg treatment.
Participant milestones
| Measure |
Metastatic Basal-Cell Carcinoma
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Overall Study
STARTED
|
33
|
71
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
33
|
71
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
33
|
71
|
Reasons for withdrawal
| Measure |
Metastatic Basal-Cell Carcinoma
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Overall Study
Adverse Event
|
5
|
16
|
|
Overall Study
Death
|
1
|
3
|
|
Overall Study
Lost to Follow-up
|
1
|
2
|
|
Overall Study
Physician Decision
|
3
|
7
|
|
Overall Study
Withdrawal by Subject
|
4
|
23
|
|
Overall Study
Sponsor Decision to End the Study
|
1
|
2
|
|
Overall Study
Disease Progression
|
18
|
17
|
|
Overall Study
Reason Not Specified
|
0
|
1
|
Baseline Characteristics
A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
Baseline characteristics by cohort
| Measure |
Metastatic Basal-Cell Carcinoma
n=33 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
n=71 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Total
n=104 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
61.6 years
STANDARD_DEVIATION 11.4 • n=44 Participants
|
61.2 years
STANDARD_DEVIATION 16.8 • n=10 Participants
|
61.4 years
STANDARD_DEVIATION 15.2 • n=30 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=44 Participants
|
32 Participants
n=10 Participants
|
41 Participants
n=30 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=44 Participants
|
39 Participants
n=10 Participants
|
63 Participants
n=30 Participants
|
PRIMARY outcome
Timeframe: At Baseline and once every 8 weeks thereafter (responses confirmed within ≥4 weeks) until the end of study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)Population: Efficacy-evaluable population: All treated patients for whom the independent pathologist's interpretation of archival tissue or baseline biopsies was consistent with basal cell carcinoma.
OR=complete (CR) or partial response (PR). Metastatic-CR:Disappearance of all targets. PR:≥30% decreased sum of longest diameter (SLD) of targets compared to baseline (B). Locally advanced-Response=No progressive disease (PD) and ≥30% decreased SLD from baseline (radiography \[R\]) or ≥30% decreased SLD from B (externally visible dimension \[EVD\]) or completely resolved ulceration. CR:Response with no residual BCC on tumor biopsy (otherwise response was PR). PD:Any of ≥20% increased SLD from nadir (R or EVD), new ulceration, new lesions (R or physical exam) or non-target lesion progression by R.
Outcome measures
| Measure |
Metastatic Basal-Cell Carcinoma
n=33 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
n=63 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Objective Response (OR) Determined by the Independent Review Facility
|
30.3 Percentage of participants
Interval 15.6 to 48.2
|
42.9 Percentage of participants
Interval 30.5 to 56.0
|
SECONDARY outcome
Timeframe: From initial OR until the earliest documented disease progression (PD) or death (until clinical cutoff date of 26 November 2010, up to 90 weeks)Population: Efficacy-evaluable population: All treated patients for whom the independent pathologist's interpretation of archival tissue or baseline biopsies was consistent with basal cell carcinoma.
Duration of OR was defined as the time from the initial CR or PR to the earliest documented disease progression (PD) or death. Metastatic BCC - PD: ≥ 20% increased sum of the longest diameter (SLD) of targets from nadir, or 1 or more new lesions. Locally advanced BCC - PD: any of: (1) ≥ 20% increased SLD from nadir (radiography or externally visible dimension); (2) new ulceration; (3) new lesions (radiography or physical exam); (4) progression of non-target lesions by radiography.
Outcome measures
| Measure |
Metastatic Basal-Cell Carcinoma
n=10 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
n=27 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Duration of Objective Response (OR) Determined by the Independent Review Facility
|
7.6 Months
Interval 5.62 to
The upper limit of the 95% confidence interval could not be estimated due to the small number of patients who had an objective response.
|
7.6 Months
Interval 5.65 to 9.66
|
SECONDARY outcome
Timeframe: From the initial dose of study drug until the earliest documented disease progression (PD) or death (up to the clinical cutoff date of 28 November 2011, up to 2 years, 5.5 months)Population: Efficacy-evaluable population: All treated patients for whom the independent pathologist's interpretation of archival tissue or baseline biopsies was consistent with basal cell carcinoma.
PFS was defined as the time from start of treatment to the earliest documented disease progression (PD) or death. Metastatic BCC - PD: ≥ 20% increased sum of the longest diameter (SLD) of targets from nadir, or 1 or more new lesions. Locally advanced BCC - PD: any of: (1) ≥ 20% increased SLD from nadir (radiography or externally visible dimension); (2) new ulceration; (3) new lesions (radiography or physical exam); (4) progression of non-target lesions by radiography.
Outcome measures
| Measure |
Metastatic Basal-Cell Carcinoma
n=33 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
n=63 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Progression-Free Survival (PFS) Determined by the Independent Review Facility
|
9.5 Months
Interval 7.36 to 11.07
|
9.5 Months
Interval 7.43 to 14.75
|
SECONDARY outcome
Timeframe: From the initial dose of study drug until death from any cause (up to the clinical cutoff date of 30 May 2013, up to 4 years)Population: Efficacy-evaluable population: All treated patients for whom the independent pathologist's interpretation of archival tissue or baseline biopsies was consistent with basal cell carcinoma.
Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.
Outcome measures
| Measure |
Metastatic Basal-Cell Carcinoma
n=33 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
n=63 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Overall Survival
|
33.4 Months
Interval 18.1 to
The upper limit of the 95% confidence interval could not be estimated because of the small number of patients who had an event (17 patients had died by the cutoff date).
|
NA Months
The median and 95% confidence interval could not be estimated because of the small number of patients who had an event (13 patients had died by the cutoff date).
|
SECONDARY outcome
Timeframe: Baseline, Week 12, Week 24, and at the end of the study or early termination visit (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)Population: Efficacy-evaluable population: All treated patients for whom the independent pathologist's interpretation of archival tissue or baseline biopsies was consistent with basal cell carcinoma. Number analyzed (n) reflects the number of participants with both baseline and visit data available for a given timepoint.
The SF-36 Health Survey (Version 2) uses patient-reported symptoms on 8 subscales to assess health-related quality of life (HRQoL). The Physical Component Summary (PCS) score summarizes the subscales Physical Functioning, Role-Physical, Bodily Pain, and General Health. The Mental Component Summary (MCS) score summarizes the subscales Vitality, Social Functioning, Role-Emotional, and Mental Health. Each score was scaled from 0 to 100. A positive change score indicates better HRQoL.
Outcome measures
| Measure |
Metastatic Basal-Cell Carcinoma
n=82 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Change in PCS score at end of study, n=20
|
-2.86 Units on a scale
Interval -7.39 to 1.66
|
—
|
|
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Change in MCS score at Week 12, n=82
|
2.20 Units on a scale
Interval -0.22 to 4.62
|
—
|
|
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Change in MCS score at Week 24, n=75
|
2.29 Units on a scale
Interval 0.05 to 4.53
|
—
|
|
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Change in MCS score at end of study, n=20
|
-3.80 Units on a scale
Interval -10.55 to 2.96
|
—
|
|
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Change in PCS score at Week 12, n=82
|
-1.25 Units on a scale
Interval -2.86 to 0.36
|
—
|
|
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Change in PCS score at Week 24, n=75
|
-1.90 Units on a scale
Interval -3.75 to -0.05
|
—
|
SECONDARY outcome
Timeframe: At Baseline and 24 weeks, and at any optional point post-baseline through end of the study (up to the clinical cutoff date of 26 November 2010, up to 90 weeks)Population: Efficacy-evaluable population: All treated patients for whom the independent pathologist's interpretation of archival tissue or baseline biopsies was consistent with basal cell carcinoma.
In patients with locally advanced BCC, the histopathological effect of vismodegib was determined in tissue biopsies obtained at baseline (prior to study drug dosing) and at 24 weeks after the start of vismodegib treatment, if the patient was still on study without evidence of progression, or at the investigator's assessment of best clinical response (or best clinical/RECIST response), if occurring prior to 24 weeks. At any time during a patient's participation in the study, an optional tumor biopsy might have been requested to clarify the response status of the patient. Reported is the percentage of efficacy-evaluable patients with locally advanced BCC pathology that was confirmed in a baseline biopsy who had an absence of residual BCC post-baseline as assessed by an independent pathological review.
Outcome measures
| Measure |
Metastatic Basal-Cell Carcinoma
n=63 Participants
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
Locally Advanced Basal-Cell Carcinoma
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|---|
|
Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
Total with Absence of Residual BCC at Any Time
|
54.0 Percentage of participants
|
—
|
|
Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
Absence of Residual BCC Prior to Week 24
|
9.5 Percentage of participants
|
—
|
|
Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
Absence of Residual BCC at Week 24
|
42.9 Percentage of participants
|
—
|
|
Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
Absence of Residual BCC After Week 24
|
1.6 Percentage of participants
|
—
|
Adverse Events
Metastatic and Locally Advanced Basal-Cell Carcinoma
Serious events: 36 serious events
Other events: 103 other events
Deaths: 35 deaths
Serious adverse events
| Measure |
Metastatic and Locally Advanced Basal-Cell Carcinoma
n=104 participants at risk
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|
|
Cardiac disorders
Cardiac failure
|
1.9%
2/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Acute myocardial infarction
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Angina pectoris
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Left ventricular dysfunction
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Myocardial infarction
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Restrictive cardiomyopathy
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Eye disorders
Eye haemorrhage
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Aphagia
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Hepatobiliary disorders
Cholestasis
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Pneumonia
|
3.8%
4/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Cellulitis
|
1.9%
2/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Meningitis viral
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Urinary tract infection
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Cervical vertebral fracture
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant melanoma
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastatic malignant melanoma
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastatic squamous cell carcinoma
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Oesophageal carcinoma
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Sarcoma
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
1.9%
2/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Convulsion
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Ischaemic stroke
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Syncope
|
3.8%
4/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Renal and urinary disorders
Renal failure
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.9%
2/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Vascular disorders
Deep vein thrombosis
|
1.9%
2/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Vascular disorders
Haemorrhage
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Vascular disorders
Hypovolaemic shock
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Vascular disorders
Orthostatic hypotension
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Atrial fibrillation
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Cardiac disorders
Cardiac flutter
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Eye disorders
Ulcerative keratitis
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Food poisoning
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Asthenia
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Chest discomfort
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Chest pain
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
General physical health deterioration
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Pyrexia
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Osteomyelitis
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Sepsis
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Hip fracture
|
2.9%
3/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Overdose
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Toxicity to various agents
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to meninges
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Cerebral haemorrhage
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Epilepsy
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Transient ischaemic attack
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Psychiatric disorders
Depression
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Psychiatric disorders
Suicidal ideation
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.9%
2/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.96%
1/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Death
|
2.9%
3/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
Other adverse events
| Measure |
Metastatic and Locally Advanced Basal-Cell Carcinoma
n=104 participants at risk
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.7%
8/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Nausea
|
32.7%
34/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
26.9%
28/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Constipation
|
19.2%
20/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Vomiting
|
18.3%
19/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Dyspepsia
|
10.6%
11/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Flatulence
|
5.8%
6/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Gastrointestinal disorders
Abdominal pain
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Nasopharyngitis
|
13.5%
14/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Upper respiratory tract infection
|
8.7%
9/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Urinary tract infection
|
9.6%
10/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Infections and infestations
Sinusitis
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Investigations
Weight decreased
|
51.9%
54/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
27.9%
29/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
72.1%
75/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
14.4%
15/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.8%
6/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
11.5%
12/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Dysgeusia
|
55.8%
58/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Headache
|
15.4%
16/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Ageusia
|
11.5%
12/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Hypogeusia
|
10.6%
11/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Paraesthesia
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Nervous system disorders
Dizziness
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Psychiatric disorders
Depression
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Psychiatric disorders
Anxiety
|
5.8%
6/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Psychiatric disorders
Insomnia
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.2%
21/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
7.7%
8/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
66.3%
69/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.6%
11/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Skin and subcutaneous tissue disorders
Hair growth abnormal
|
5.8%
6/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.7%
8/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Influenza like illness
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
6.7%
7/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
Skin and subcutaneous tissue disorders
Actinic keratosis
|
5.8%
6/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Fatigue
|
43.3%
45/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Asthenia
|
9.6%
10/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
|
General disorders
Pain
|
9.6%
10/104 • For all-cause mortality, deaths were recorded from enrollment through the end of treatment-free survival follow-up (up to 5 years, 2 months). Adverse events and serious adverse events were recorded from enrollment through 45 days after the last dose of study drug or study discontinuation/termination, whichever is later (up to 4 years, 7 months).
Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
|
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