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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Genentech
ClinicalTrials.gov Identifier:
NCT00833417
First received: January 30, 2009
Last updated: March 12, 2014
Last verified: March 2014
  Purpose

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 will receive vismodegib until evidence of progression, intolerable toxicities most probably attributable to vismodegib, or withdrawal from the study.


Condition Intervention Phase
Basal Cell Carcinoma
Drug: Vismodegib 150 mg
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II, Multicenter, Single-Arm, Two-Cohort Trial Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Patients With Advanced Basal Cell Carcinoma

Resource links provided by NLM:


Further study details as provided by Genentech:

Primary Outcome Measures:
  • Objective Response (OR) Determined by the Independent Review Facility [ Time Frame: From study initiation (enrollment of first patient) through 9 months following the first treatment of the last enrolled patient (clinical cutoff date of 26 November 2010), up to 90 weeks ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Duration of Objective Response (OR) Determined by the Independent Review Facility [ Time Frame: From study initiation (enrollment of first patient) through 9 months following the first treatment of the last enrolled patient (clinical cutoff date of 26 November 2010), up to 90 weeks ] [ Designated as safety issue: No ]
    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.

  • Progression-free Survival (PFS) Determined by the Independent Review Facility [ Time Frame: From study initiation (enrollment of first patient) through 9 months following the first treatment of the last enrolled patient (clinical cutoff date of 26 November 2010), up to 90 weeks ] [ Designated as safety issue: No ]
    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 [ Time Frame: From study initiation (enrollment of first patient) through 9 months following the first treatment of the last enrolled patient (clinical cutoff date of 26 November 2010), up to 90 weeks ] [ Designated as safety issue: No ]
    Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.

  • 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 90 weeks ] [ Designated as safety issue: No ]
    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.

  • Number of Patients With Absence of Residual Basal Cell Carcinoma BCC in Patients With Locally Advanced BCC [ Time Frame: From baseline through end of the study, up to 90 weeks ] [ Designated as safety issue: No ]
    In patients with locally advanced BCC, the histopathological effect of vismodegib was determined in tissue biopsies obtained at baseline and following vismodegib treatment. A histopathological response was defined as the absence of residual BCC post-baseline assessed by the independent pathological review.


Enrollment: 104
Study Start Date: February 2009
Estimated Study Completion Date: April 2014
Primary Completion Date: November 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Vismodegib 150 mg
Patients received vismodegib 150 mg orally once daily until disease progression; intolerable toxicity, most probably attributable to vismodegib; or withdrawal from the study.
Drug: Vismodegib 150 mg
Vismodegib 150 mg was provided in hard gelatin capsules.
Other Name: GDC-0449

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00833417

  Hide Study Locations
Locations
United States, Arizona
Scottsdale, Arizona, United States, 85259
United States, California
Los Angeles, California, United States, 90025
San Francisco, California, United States, 94115
Stanford, California, United States, 94305
United States, Colorado
Aurora, Colorado, United States, 80045
United States, Florida
Ormond Beach, Florida, United States, 32174
United States, Illinois
Chicago, Illinois, United States, 60611
United States, Iowa
Sioux City, Iowa, United States, 51101
United States, Maryland
Baltimore, Maryland, United States, 21231-1000
United States, Massachusetts
Boston, Massachusetts, United States, 02215
Boston, Massachusetts, United States, 02114
United States, Minnesota
Rochester, Minnesota, United States, 55905
United States, Nevada
Las Vegas, Nevada, United States, 89103
United States, New Hampshire
Lebanon, New Hampshire, United States, 03756
United States, New York
New York, New York, United States, 10029
United States, North Carolina
Chapel Hill, North Carolina, United States, 27599-7305
United States, Ohio
Columbus, Ohio, United States, 43210
United States, Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Nashville, Tennessee, United States, 37203
United States, Texas
Houston, Texas, United States, 77030-4095
Australia
Kogarah, New South Wales, Australia, 2217
Melbourne, Australia, 3002
Woolloongabba, Australia, 4102
Belgium
Bruxelles, Belgium, 1000
Wilrijk, Belgium, 2610
France
Lille, France, 59037
Nantes Cedex 1, France, 44093
Paris, France, 75010
Pierre Benite, France, 69495
Germany
Essen, Germany, 45122
Kiel, Germany, 24105
Tübingen, Germany, 72076
Wurzburg, Germany, 97080
United Kingdom
London, United Kingdom, SW3 6JJ
Poole, United Kingdom, BH15 2JB
Sponsors and Collaborators
Genentech
Investigators
Study Director: Jeannie Hou, M.D. Genentech
  More Information

No publications provided by Genentech

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Genentech
ClinicalTrials.gov Identifier: NCT00833417     History of Changes
Other Study ID Numbers: SHH4476g, GO01541
Study First Received: January 30, 2009
Results First Received: February 23, 2012
Last Updated: March 12, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Genentech:
BCC
Hedgehog Pathway Inhibitor
Hedgehog
Basal Cell Cancer

Additional relevant MeSH terms:
Carcinoma
Carcinoma, Basal Cell
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Basal Cell

ClinicalTrials.gov processed this record on September 18, 2014