Clinical trial NCT01160250: A Study of Vismodegib (GDC-0449) in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Official title: A Single-Arm, Open-Label, Expanded Access Study of Vismodegib (GDC-0449) in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Basal Cell Carcinoma
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2010-07-12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no

Brief summary: This is an open-label, non-comparative, multicenter, expanded access study of Vismodegib (GDC-0449) in patients with locally advanced basal cell carcinoma (BCC) or metastatic BCC (mBCC) who are otherwise without satisfactory treatment options.


Eligibility criteria: Inclusion Criteria:

* For patients with mBCC, histologic confirmation of distant BCC metastasis
* For patients with locally advanced BCC, at least one histologically confirmed lesion 10 mm or more in diameter and written confirmation from a surgical specialist that the tumor is considered inoperable or that surgery is contraindicated
* For patients with locally advanced BCC, radiotherapy must have been previously administered for locally advanced BCC, unless radiotherapy is contraindicated or inappropriate. For patients whose locally advanced BCC has been irradiated, disease must have progressed after radiation.
* Patients with Gorlin syndrome may enroll in this study but must meet the criteria for locally advanced or metastatic disease listed above
* Patients with measurable and/or non-measurable disease (as defined by RECIST) are allowed
* Adequate organ function
* 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 (GDC-0449)

Exclusion Criteria:

* Pregnancy or lactation
* Life expectancy \< 12 weeks
* Concurrent non-protocol-specified anti-tumor therapy
* Completion of most recent anti-tumor therapy at least 21 days prior to initiation of treatment
* Uncontrolled medical illnesses such as infection requiring treatment with intravenous antibiotics
* History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or renders the patient at high risk from treatment complications
* Unwillingness to practice effective birth control

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
state: Arizona
city: Scottsdale
country: United States
state: Arizona
city: Los Angeles
country: United States
state: California
city: Stanford
country: United States
state: California
city: Ormond Beach
country: United States
state: Florida
city: Detroit
country: United States
state: Michigan
city: New York
country: United States
state: New York
city: Columbus
country: United States
state: Ohio
city: Philadelphia
country: United States
state: Pennsylvania
city: Nashville
country: United States
state: Tennessee
city: Houston
country: United States
state: Texas
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References:
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.