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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00833417




Registration number
NCT00833417
Ethics application status
Date submitted
30/01/2009
Date registered
30/01/2009
Date last updated
1/05/2015

Titles & IDs
Public title
A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449, Hedgehog Pathway Inhibitor) in Patients With Advanced Basal Cell Carcinoma
Scientific 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
Secondary ID [1] 0 0
GO01541
Secondary ID [2] 0 0
SHH4476g
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Basal Cell Carcinoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Non melanoma skin cancer
Cancer 0 0 0 0
Kidney

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vismodegib 150 mg

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.


Treatment: Drugs: Vismodegib 150 mg
Vismodegib 150 mg was provided in hard gelatin capsules.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Objective Response (OR) Determined by the Independent Review Facility - 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.
Timepoint [1] 0 0
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
Secondary outcome [1] 0 0
Duration of Objective Response (OR) Determined by the Independent Review Facility - 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.
Timepoint [1] 0 0
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
Secondary outcome [2] 0 0
Progression-free Survival (PFS) Determined by the Independent Review Facility - 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.
Timepoint [2] 0 0
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
Secondary outcome [3] 0 0
Overall Survival - Overall survival was defined as the time from the initial dose of vismodegib until death from any cause.
Timepoint [3] 0 0
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
Secondary outcome [4] 0 0
Change From Baseline in Short Form 36 (SF-36) Health Survey Scores - 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.
Timepoint [4] 0 0
Baseline, Week 12, Week 24, and at the end of the study or early termination visit, up to 90 weeks
Secondary outcome [5] 0 0
Percentage of Patients With Absence of Residual Basal Cell Carcinoma (BCC) in Patients With Locally Advanced BCC - In patients with locally advanced BCC, the histopathological effect of vismodegib was determined in tissue biopsies obtained at baseline and following vismodegib treatment. Reported are the percentage of patients with pathology confirmed BCC in baseline biopsy who had an absence of residual BCC post-baseline as assessed by an independent pathological review.
Timepoint [5] 0 0
From baseline through end of the study, up to 90 weeks

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Kogarah, New South Wales
Recruitment hospital [2] 0 0
- Melbourne
Recruitment hospital [3] 0 0
- Woolloongabba
Recruitment postcode(s) [1] 0 0
2217 - Kogarah, New South Wales
Recruitment postcode(s) [2] 0 0
3002 - Melbourne
Recruitment postcode(s) [3] 0 0
4102 - Woolloongabba
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Nevada
Country [11] 0 0
United States of America
State/province [11] 0 0
New Hampshire
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
North Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Ohio
Country [15] 0 0
United States of America
State/province [15] 0 0
Pennsylvania
Country [16] 0 0
United States of America
State/province [16] 0 0
Tennessee
Country [17] 0 0
United States of America
State/province [17] 0 0
Texas
Country [18] 0 0
Belgium
State/province [18] 0 0
Bruxelles
Country [19] 0 0
Belgium
State/province [19] 0 0
Wilrijk
Country [20] 0 0
France
State/province [20] 0 0
Lille
Country [21] 0 0
France
State/province [21] 0 0
Nantes Cedex 1
Country [22] 0 0
France
State/province [22] 0 0
Paris
Country [23] 0 0
France
State/province [23] 0 0
Pierre Benite
Country [24] 0 0
Germany
State/province [24] 0 0
Essen
Country [25] 0 0
Germany
State/province [25] 0 0
Kiel
Country [26] 0 0
Germany
State/province [26] 0 0
Tübingen
Country [27] 0 0
Germany
State/province [27] 0 0
Wurzburg
Country [28] 0 0
United Kingdom
State/province [28] 0 0
London
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Poole

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Genentech, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This was 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 received vismodegib until evidence of progression, intolerable toxicities most
probably attributable to vismodegib, or withdrawal from the study.
Trial website
https://clinicaltrials.gov/show/NCT00833417
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jeannie Hou, M.D.
Address 0 0
Genentech, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications