Neoplasms

All Conditions

Carcinoma

Carcinoma, Basal Cell

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Neoplasms, Basal Cell

Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.

Vismodegib 150 mg was provided in hard gelatin capsules.

Vismodegib 150 mg

Jeannie Hou, M.D.

This was a Phase II, single-arm, two-cohort multicenter clinical trial evaluating the efficacy and safety of vismodegib (GDC-0449) in patients with advanced basal cell carcinoma. All patients received vismodegib until evidence of progression, intolerable toxicities most probably attributable to vismodegib, or withdrawal from the study.

A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma

A Pivotal Phase II, Multicenter, Single-arm, Two-cohort Trial Evaluating the Efficacy and Safety of GDC-0449 in Patients With Advanced 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.

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.

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.

Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.

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.

In patients with locally advanced BCC, the histopathological effect of vismodegib was determined in tissue biopsies obtained at baseline and following vismodegib treatment. Reported are the percentage of patients with pathology confirmed BCC in baseline biopsy who had an absence of residual BCC post-baseline as assessed by an independent pathological review.

Genentech, Inc.

Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.

Metastatic and Locally Advanced BCC Cohorts

Metastatic BCC Cohort

Locally Advanced BCC Cohort

Total

Age, Continuous

Sex: Female, Male

Medical Communications

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.

Objective Response (OR) Determined by the Independent Review Facility

Duration of Objective Response (OR) Determined by the Independent Review Facility

Progression-free Survival (PFS) Determined by the Independent Review Facility

Overall Survival

Change in MCS score at Week 12, n=82

Change in MCS score at Week 24, n=75

Change in MCS score at end of study, n=20

Change in PCS score at Week 12, n=82

Change in PCS score at Week 24, n=75

Change in PCS score at end of study, n=20

Change From Baseline in Short Form 36 (SF-36) Health Survey Scores

Prior to Week 24

At Week 24

After Week 24

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. Only locally advanced BCC patients with available post-baseline biopsy assessed by an independent pathologist were included in the analysis.

Spots Global Cancer Trial Database for A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma

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