NCT00833417 · OUTCOME

Objective Response (OR) Determined by the Independent Review Facility

A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma · Source last updated 2026-05-11

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure: NUMBER
Unit: Percentage of participants
Interval / dispersion: 95% Confidence Interval
Time frame: 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)

What was measured

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.

Analysis 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.

Groups in this outcome

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.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

Group	Source count
Metastatic Basal-Cell Carcinoma	33
Locally Advanced Basal-Cell Carcinoma	63

Reported measurements

Source class 1

Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Metastatic Basal-Cell Carcinoma	30.3	Not reported	15.6	48.2	Not reported
Locally Advanced Basal-Cell Carcinoma	42.9	Not reported	30.5	56.0	Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

group Description
In the metastatic BCC cohort, a one-sided exact binomial test was conducted for the following hypotheses: Null hypothesis (Ho): objective response rate (ORR) ≤10%; Alternative hypothesis (Ha): ORR \>10%.
group Ids
OG000
non Inferiority Type
SUPERIORITY
p Value
0.0011
p Value Comment
The hypothesis was tested at a one-sided α = 0.025 level.
statistical Method
Exact binomial test
group Description
In the locally advanced BCC cohort, a one-sided exact binomial test was conducted for the following hypotheses: Null hypothesis (Ho): objective response rate (ORR) ≤20%; Alternative hypothesis (Ha): ORR \>20%.
group Ids
OG001
non Inferiority Type
SUPERIORITY
p Value
<0.0001
p Value Comment
The hypothesis was tested at a one-sided α = 0.025 level.
statistical Method
Exact binomial test

Complete source fields

analyses

group Description
In the metastatic BCC cohort, a one-sided exact binomial test was conducted for the following hypotheses: Null hypothesis (Ho): objective response rate (ORR) ≤10%; Alternative hypothesis (Ha): ORR \>10%.
group Ids
OG000
non Inferiority Type
SUPERIORITY
p Value
0.0011
p Value Comment
The hypothesis was tested at a one-sided α = 0.025 level.
statistical Method
Exact binomial test
group Description
In the locally advanced BCC cohort, a one-sided exact binomial test was conducted for the following hypotheses: Null hypothesis (Ho): objective response rate (ORR) ≤20%; Alternative hypothesis (Ha): ORR \>20%.
group Ids
OG001
non Inferiority Type
SUPERIORITY
p Value
<0.0001
p Value Comment
The hypothesis was tested at a one-sided α = 0.025 level.
statistical Method
Exact binomial test

classes

categories
measurements
group Id
OG000
lower Limit
15.6
upper Limit
48.2
value
30.3
group Id
OG001
lower Limit
30.5
upper Limit
56.0
value
42.9

denoms

counts
group Id
OG000
value
33
group Id
OG001
value
63
units
Participants

description

dispersion Type

95% Confidence Interval

groups

description
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
id
OG000
title
Metastatic Basal-Cell Carcinoma
description
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
id
OG001
title
Locally Advanced Basal-Cell Carcinoma

param Type

NUMBER

population Description

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.

reporting Status

POSTED

time Frame

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)

title

Objective Response (OR) Determined by the Independent Review Facility

type

PRIMARY

unit Of Measure

Percentage of participants

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Preserved source evidence · Not medical advice