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Completed

NCT Number: NCT00833417

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

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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Key information

Conditions

Basal Cell Carcinoma Carcinoma Carcinoma, Basal Cell Neoplasms Neoplasms by Histologic Type Neoplasms, Basal Cell Neoplasms, Glandular and Epithelial

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

St George Hospital, Kogarah, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Vismodegib 150 mg

Drug

Vismodegib 150 mg was provided in hard gelatin capsules.

Other names: GDC-0449

Primary outcomes

  1. 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)

    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.

Secondary outcomes

  1. 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)

    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.

  2. 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)

    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.

  3. 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)

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

  4. 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)

    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.

  5. 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)

    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.

Sponsors and collaborators

Lead sponsor

Genentech, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2009
Primary completion
2010
Study completion
2014
First posted
Feb 2, 2009
Registry last updated
May 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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