TriangleNCT00833417 · STUDY IN CONTEXT
A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
Draft scope assessment: unclear. Attached because the study's own record describes locally advanced disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
Read the full study record and all posted results →
Linked outcomes
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 (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.
Reported safety
Quality of life and patient-reported measures
SF-36 / SF-12 · Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
Score direction: Not stated by the study. Direction is never inferred from an instrument name.
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- qol key
- qol-0004
- source section id
- NCT00833417-om-0004
- source outcome index
- 4
- instrument
- SF-36 / SF-12
- instrument basis
- matched 'sf-36' in the study's own wording
- scale title
- Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
- measure definition
- 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.
- time frame
- 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)
- analysis role
- secondary
- reporting status
- POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- trial label
- A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
- applicability
- unclear
- applicability note
- Attached because the study's own record describes locally advanced disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- provenance class
- regulator-cited
- discovery note
- Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
- outcome Count
- 6
- harms Count
- 1
- qol Count
- 1
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0000
- source section id
- NCT00833417-om-0000
- source outcome index
- 0
- endpoint
- Objective Response (OR) Determined by the Independent Review Facility
- endpoint definition
- 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.
- time origin
- 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)
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0001
- source section id
- NCT00833417-om-0001
- source outcome index
- 1
- endpoint
- Duration of Objective Response (OR) Determined by the Independent Review Facility
- endpoint definition
- 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.
- time origin
- From initial OR until the earliest documented disease progression (PD) or death (until clinical cutoff date of 26 November 2010, up to 90 weeks)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0002
- source section id
- NCT00833417-om-0002
- source outcome index
- 2
- endpoint
- Progression-Free Survival (PFS) Determined by the Independent Review Facility
- endpoint definition
- 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.
- time origin
- 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)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0003
- source section id
- NCT00833417-om-0003
- source outcome index
- 3
- endpoint
- Overall Survival
- endpoint definition
- Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.
- time origin
- 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)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0004
- source section id
- NCT00833417-om-0004
- source outcome index
- 4
- endpoint
- Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
- endpoint definition
- 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.
- time origin
- 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)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0005
- source section id
- NCT00833417-om-0005
- source outcome index
- 5
- endpoint
- Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
- endpoint definition
- 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.
- time origin
- 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)
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- arms
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0000
- arm key
- OG000
- arm label
- Metastatic Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 33
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0000
- arm key
- OG001
- arm label
- Locally Advanced Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 63
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0000#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0001
- arm key
- OG000
- arm label
- Metastatic Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 10
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0001#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0001
- arm key
- OG001
- arm label
- Locally Advanced Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 27
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0001#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0002
- arm key
- OG000
- arm label
- Metastatic Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 33
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0002#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0002
- arm key
- OG001
- arm label
- Locally Advanced Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 63
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0002#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0003
- arm key
- OG000
- arm label
- Metastatic Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 33
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0003#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0003
- arm key
- OG001
- arm label
- Locally Advanced Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 63
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0003#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0004
- arm key
- OG000
- arm label
- Metastatic and Locally Advanced Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 82
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0004#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- outcome key
- om-0005
- arm key
- OG000
- arm label
- Locally Advanced Basal-Cell Carcinoma
- regimen
- Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
- analyzed count
- 63
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT00833417-om-0005#groups/OG000
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- harm key
- EG000
- source section id
- NCT00833417-ae
- event group key
- EG000
- event group label
- Metastatic and Locally Advanced Basal-Cell Carcinoma
- serious affected
- 36
- serious at risk
- 104
- other affected
- 103
- other at risk
- 104
- frequency threshold
- 5
- collection period
- 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).
- event definition
- Adverse events were reported for the safety analysis population, which was defined as all enrolled patients who received any amount of study drug.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- quality of life
- release id
- clinical-indications-draft-1
- disease state id
- ds-basal-cell-carcinoma-odomzo-0783c8f4
- nct id
- NCT00833417
- qol key
- qol-0004
- source section id
- NCT00833417-om-0004
- source outcome index
- 4
- instrument
- SF-36 / SF-12
- instrument basis
- matched 'sf-36' in the study's own wording
- scale title
- Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
- measure definition
- 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.
- time frame
- 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)
- analysis role
- secondary
- reporting status
- POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- setting source links
Preserved source evidence · Not medical advice