A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Operable Basal Cell Carcinoma

• ClinicalTrials.gov Identifier: NCT01201915
• Recruitment Status: Completed
• First Posted: September 15, 2010
• Results First Posted: June 24, 2014
• Last Update Posted: June 24, 2014
• Sponsor: Genentech, Inc.
• Information provided by (Responsible Party): Genentech, Inc.

Study Description

Brief Summary:

This was a 3-cohort, open-label study of vismodegib (GDC-0449) in new (non-recurrent) operable basal cell carcinoma of the nodular subtype.

Condition or disease	Intervention/treatment	Phase
Basal Cell Carcinoma	Drug: Vismodegib	Phase 2

Detailed Description:

For Cohort 1, the response to treatment was determined at the end of the 12 weeks of treatment. For Cohort 2, the response to treatment was determined after 12 weeks of treatment and 24 weeks of observation. For Cohort 3, the response to treatment was determined after intermittent dosing over a 20-week period, consisting of an initial 8-week treatment period, followed by a 4-week drug holiday period, followed by a second 8-week treatment period.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 74 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase II, Multicenter, Open-label, Three-cohort Trial Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Operable Basal Cell Carcinoma (BCC)
• Study Start Date: October 2010
• Actual Primary Completion Date: May 2013
• Actual Study Completion Date: May 2013

Arms and Interventions

Arm	Intervention/treatment
Experimental: Cohort 1: Vismodegib 150 mg; Participants received vismodegib 150 mg orally daily for 12 weeks.	Drug: Vismodegib; Vismodegib was supplied in gelatin capsules.; Other Name: GDC-0449
Experimental: Cohort 2: Vismodegib 150 mg; Participants received vismodegib 150 mg orally daily for 12 weeks.	Drug: Vismodegib; Vismodegib was supplied in gelatin capsules.; Other Name: GDC-0449
Experimental: Cohort 3: Vismodegib 150 mg; Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.	Drug: Vismodegib; Vismodegib was supplied in gelatin capsules.; Other Name: GDC-0449

Outcome Measures

Primary Outcome Measures :

1. Percentage of Participants With Complete Histologic Clearance [ Time Frame: Baseline to Week 12 (Cohort 1), Baseline to Week 36 (Cohort 2), Baseline to Week 20 (Cohort 3) ]
   Complete histologic clearance was defined as the absence of histological evidence of basal cell carcinoma at the target tumor site. Histological examination was performed by an independent pathologist on specimens collected within 2 weeks of the end of treatment period, ie, at 12 weeks after Baseline in Cohort 1, at 36 weeks after Baseline in Cohort 2, and at 20 weeks after Baseline in Cohort 3.

Secondary Outcome Measures :

1. Time to Complete Clinical Clearance [ Time Frame: Baseline to the end of the study (up to 12 weeks for Cohort 1; up to 36 weeks for Cohort 2, up to 20 weeks for Cohort 3) ]
   Time to complete clinical clearance was defined as the time from the first treatment with vismodegib until complete clinical clearance as determined by the investigator.

Eligibility Criteria

• Ages Eligible for Study: 21 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Confirmed new (not recurrent or previously treated) nodular basal cell carcinoma (BCC) at 1 of the listed anatomical sites, which must be biopsy confirmed at the study site.
• Willingness to consent to biopsy of the lesion.
• Willingness to delay excision of the target tumor site until the time mandated in the protocol, unless evidence of disease progression or lack of drug tolerability.
• Adequate hematopoietic capacity.
• Adequate hepatic function.
• For women of childbearing potential, agreement to use 2 acceptable forms of birth control (including one barrier method) during the study and for 7 months after discontinuation of study drug.
• For men with female partners of childbearing potential, agreement to use a male condom (with spermicide) and to advise their female partners to use an additional acceptable method of birth control during the study and for 2 months after discontinuation of study drug.
• Agreement not to donate blood/blood products during the study and for 7 months after discontinuing study drug.
• Agreement not to donate sperm or semen during treatment and for 2 months after the last dose of vismodegib.

Exclusion Criteria:

• Prior treatment with vismodegib or any Hedgehog pathway inhibitor.
• Inability or unwillingness to swallow capsules.
• Pregnancy or lactation.
• BCC with any clinical and histological pattern other than nodular BCC.
• Patients with known Gorlin's syndrome (basal cell nevus syndrome) or clinical suspicion of Gorlin's syndrome.
• Recent (ie, within the past 28 days), current, or planned participation in another experimental drug study.
• Use of any excluded medication or therapy within 21 days of study entry.
• History of other malignancies within 3 years of the first day of treatment (Day 1), except for tumors with a negligible risk of metastasis, such as other non-melanoma skin cancer (BCC, squamous cell cancer), ductal carcinoma in situ of the breast, or carcinoma in situ of the cervix.
• Uncontrolled medical illness.
• History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that might affect interpretation of the results of the study or renders the patient at high risk for treatment complications.
• Any medical or psychological illness or condition preventing adequate consent.

Contacts and Locations

Locations

United States, Arizona

Scottsdale, Arizona, United States, 85259

United States, California

Los Angeles, California, United States, 90045

Redwood City, California, United States, 94063

San Diego, California, United States, 92117

United States, Florida

St. Petersburg, Florida, United States, 33716

United States, Georgia

Alpharetta, Georgia, United States, 30005

United States, Illinois

Chicago, Illinois, United States, 60611

United States, Kentucky

Louisville, Kentucky, United States, 40217

United States, Pennsylvania

Philadelphia, Pennsylvania, United States, 19111

United States, Tennessee

Bartlett, Tennessee, United States, 38134

United States, Texas

Houston, Texas, United States, 77030

United States, Wisconsin

Madison, Wisconsin, United States, 53705

Sponsors and Collaborators

Genentech, Inc.

Investigators

• Study Director: Ivor Caro, M.D.; Genentech, Inc.

More Information

• Responsible Party: Genentech, Inc.
• ClinicalTrials.gov Identifier: NCT01201915
• Other Study ID Numbers: SHH4812g; GS01354 ( Other Identifier: Hoffmann-La Roche )
• First Posted: September 15, 2010
• Results First Posted: June 24, 2014
• Last Update Posted: June 24, 2014
• Last Verified: May 2014

Additional relevant MeSH terms:

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