Study Evaluating the Efficacy of Oral Vismodegib in Various Histologic Subtypes
Condition: Basal Cell Carcinoma
MeSH terms: Carcinoma; Carcinoma, Basal Cell
Intervention: vismodegib (150 mg PO daily)
Intervention description: Biopsies will be performed on all participants at baseline, week 12 and week 24.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 28.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: advanced BCC; high risk; locally advanced

Summary:

      The purpose of this study is to investigate the safety and effectiveness of oral vismodegib
      therapy in the treatment of different 'histologic subtypes' of basal cell skin cancer (BCC).
      The term 'histologic subtype' refers to how the cells and tumor tissue looks under the
      microscope. Three different 'histologic subtypes' of basal cell skin cancer
      (infiltrative/morpheaform, nodular and superficial) will be examined in this study.
    

Eligibility criteria:

        Inclusion Criteria:

          1. A signed and data informed consent

          2. Willing to comply with scheduled visits, treatment plan, laboratory tests, and other
             study procedures

          3. 18 years of age or older at time of informed consent

          4. Have one or more clinically suspicious lesions for BCC at Pre-Study screening Visit
             that has:

               1. a diameter ≥ 6 mm if located on the "mask areas" of face (central face, eyelids,
                  eyebrows, periorbital,nose,lips,chin,mandible,preauricular and postauricular
                  skin/sulci,temple,ear),genitalia,hands,or feet

               2. a diameter ≥ 10 mm if located on cheeks,forehead,scalp,or neck

               3. a diameter ≥ 20 mm if located on trunk and extremities

                  or has a lesion suspicious for locally advanced BCC defined as a lesion that:

               4. is ≥ 10 mm,

               5. has recurred following surgery or surgical resection would result in substantial
                  deformity, and

               6. has been deemed not appropriate for radiation.

          5. Have a histologically-confirmed BCC prior to first dose of study drug

          6. Have an Eastern Cooperative Oncology Group performance status of 2 or less at Baseline

          7. Female of reproductive potential must use 2 effective methods to avoid pregnancy
             during therapy and for 7 months after completing therapy

          8. Male patients must use effective measures to avoid pregnancy in their partner at all
             times during treatment and for 2 months after the last dose

          9. Agree not to donate blood or blood products during the study and for 7 months after
             the last dose

         10. Subjects with Basal Cell Nevus Syndrome are eligible for enrollment

        Exclusion Criteria:

          1. Women who are pregnant, lactating, or planning pregnancy while in the study

          2. History of prior treatment with vismodegib or any Hh Pathway Inhibitor

          3. Evidence of clinically significant and unstable diseases or conditions; Subjects with
             clinically stable chronic medical conditions will be allowed to enter the study

          4. Any dermatological disease at treatment site that the investigator thinks may be
             exacerbated by treatment with vismodegib or cause difficulty with examination

          5. The target lesion identified at Pre-study Screening visit has been determined to be
             mBCC by radiological assessment prior to first dose of study drug

          6. Inability or unwillingness to swallow capsules

          7. History of infection requiring hospitalization, IV antimicrobial therapy, or is
             otherwise judged to be clinically significant by the investigator within 4 wks prior
             to first dose of study drug

          8. History of infection requiring antimicrobial therapy within 2 wks prior to first dose
             of study drug

          9. History of alcohol or substance abuse, unless in full remission for greater than 6
             months prior to first dose of study drug

         10. Known to be infected with human immunodeficiency virus, hepatitis B or hepatitis C
             viruses

         11. Participation in other study using an investigational or experimental therapy or
             procedure within 4 weeks or 5 half-lives (whichever is longer) before the study begins
             and/or during study participation

         12. Other severe acute or chronic medical or psychiatric condition or laboratory
             abnormality that may increase the risk associated with study participation or
             investigational product administration or may interfere with the interpretation of
             study results in the judgment of the investigator

         13. Subjects who are study site staff members or who are Sponsor employees directly
             involved in the conduct of the trial

         14. A subject who, in the opinion of the investigator or sponsor, will be uncooperative or
             unable to comply with study procedures