TriangleNCT00833417 · CITED IN SOURCE DOCUMENTS
A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE2
- Status at capture
- COMPLETED
- Registry last update
- 2026-05-11
This is 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 receive vismodegib until evidence of progression, intolerable toxicities most probably attributable to vismodegib, or withdrawal from the study.
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What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
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 From Baseline in Short Form 36 (SF-36) Health Survey Scores
Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- Inclusion Criteria: * Men and women ≥ 18 years of age. * For patients with metastatic basal cell carcinoma (BCC), histological confirmation of distant BCC metastasis (eg, lung, liver, lymph nodes, or bone), with metastatic disease that is Response Evaluation Criteria in Solid Tumors (RECIST) measurable using computed tomography (CT) or magnetic resonance imaging (MRI). * For patients with locally advanced BCC, histologically confirmed disease that is considered to be inoperable. * For patients with locally advanced BCC, radiotherapy must have been previously administered for their locally advanced BCC, unless radiotherapy is contraindicated or inappropriate. For patients whose locally advanced BCC has been irradiated, disease must have progressed after radiation. * For women of childbearing potential, agreement to the use of two acceptable methods of contraception, including one barrier method, during the study and for 12 months after discontinuation of vismodegib (GDC-0449). * For men with female partners of childbearing potential, agreement to use a latex condom, and to advise their female partner to use an additional method of contraception during the study and for 3 months after discontinuation of vismodegib. Exclusion Criteria: * Prior treatment with vismodegib or other Hedgehog pathway inhibitors. * Pregnancy or lactation. * Life expectancy of \< 12 weeks. * Patients with superficial multifocal BCC who may be considered unresectable due to breadth of involvement. * Concurrent non-protocol-specified anti-tumor therapy (eg, chemotherapy, other targeted therapy, radiation therapy, or photodynamic therapy). * Recent, current, or planned participation in an experimental drug study. * History of other malignancies within 3 years of the first day of treatment with vismodegib in this study (Day 1), except for tumors with a negligible risk for metastasis or death, such as adequately treated squamous-cell carcinoma of the skin, ductal carcinoma in situ of the breast, or carcinoma in situ of the cervix. * Uncontrolled medical illnesses such as infection requiring treatment with intravenous antibiotics.
- healthy Volunteers
- false
- minimum Age
- 18 Years
- sex
- ALL
- std Ages
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm 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.
- intervention Names
- Drug: Vismodegib 150 mg
- label
- Vismodegib 150 mg
- type
- EXPERIMENTAL
- interventions
- arm Group Labels
- Vismodegib 150 mg
- description
- Vismodegib 150 mg was provided in hard gelatin capsules.
- name
- Vismodegib 150 mg
- other Names
- GDC-0449
- type
- DRUG
Study design
- allocation
- NA
- intervention Model
- SINGLE_GROUP
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 104
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- 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.
- measure
- Objective Response (OR) Determined by the Independent Review Facility
- 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)
- secondary Outcomes
- description
- 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.
- measure
- Duration of Objective Response (OR) Determined by the Independent Review Facility
- time Frame
- From initial OR until the earliest documented disease progression (PD) or death (until clinical cutoff date of 26 November 2010, up to 90 weeks)
- description
- 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.
- measure
- Progression-Free Survival (PFS) Determined by the Independent Review Facility
- time Frame
- 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)
- description
- Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.
- measure
- Overall Survival
- time Frame
- 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)
- description
- 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.
- measure
- Change From Baseline in Short Form 36 (SF-36) Health Survey Scores
- 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)
- description
- 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.
- measure
- Percentage of Participants With Absence of Residual Basal Cell Carcinoma (BCC) in the Efficacy-Evaluable Locally Advanced BCC Cohort
- time Frame
- 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)
Full study description
- brief Summary
- This is 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 receive vismodegib until evidence of progression, intolerable toxicities most probably attributable to vismodegib, or withdrawal from the study.
Source references
- references
- citation
- Sekulic A, Migden MR, Basset-Seguin N, Garbe C, Gesierich A, Lao CD, Miller C, Mortier L, Murrell DF, Hamid O, Quevedo JF, Hou J, McKenna E, Dimier N, Williams S, Schadendorf D, Hauschild A; ERIVANCE BCC Investigators. Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study. BMC Cancer. 2017 May 16;17(1):332. doi: 10.1186/s12885-017-3286-5.
- pmid
- 28511673
- type
- DERIVED
- citation
- Chang AL, Arron ST, Migden MR, Solomon JA, Yoo S, Day BM, McKenna EF, Sekulic A. Safety and efficacy of vismodegib in patients with basal cell carcinoma nevus syndrome: pooled analysis of two trials. Orphanet J Rare Dis. 2016 Sep 1;11(1):120. doi: 10.1186/s13023-016-0506-z.
- pmid
- 27581207
- type
- DERIVED
- citation
- Sekulic A, Migden MR, Oro AE, Dirix L, Lewis KD, Hainsworth JD, Solomon JA, Yoo S, Arron ST, Friedlander PA, Marmur E, Rudin CM, Chang AL, Low JA, Mackey HM, Yauch RL, Graham RA, Reddy JC, Hauschild A. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med. 2012 Jun 7;366(23):2171-9. doi: 10.1056/NEJMoa1113713.
- pmid
- 22670903
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- 14 CLINICAL STUDIES A single, international, single-arm, multi-center, open-label, 2-cohort trial [SHH4476g (NCT00833417)] was conducted in 104 patients with either metastatic basal cell carcinoma (mBCC) (n = 33) or locally advanced BCC (laBCC) (n = 71). Patients with laBCC were
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Preserved source evidence · Not medical advice