Phase II study GDC-0449 IN PATIENTS WITH ADVANCED BASAL CELL CARCINOMA

• Research type

  Research Study

• Full 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

• IRAS ID

  14636

• Sponsor organisation

  Genentech Inc

• Eudract number

  2008-004945-27

• ISRCTN Number

  Not submitted

• Research summary

  Basal cell carcinoma (BCC) is a type of skin cancer and is the most common one in the UK. In almost all patients they are small BCCs that can be effectively treated by dermatologists, usually by removing the tumour surgically. In a small subset of patients, the cancer grows deeper into the skin and damages nearby structures, this is known as locally advanced BCC. Locally advanced BCC is associated with significant morbidity as a result of conditions such as of chronic pain, bacterial infection, sepsis and bleeding/oozing. Function can also be lost if the cancer spreads to other structures or organs of the body which may result in death.Metastatic BCC (mBCC) is an extremely rare type of BCC and is where the cancer has spread to one or several parts of the body other than the skin. Once metastasis is detected, prognosis is poor as just like locally advanced BCC no standard therapy for mBCC exists.This study aims to estimate the clinical benefit of study drug GDC-0449 given as therapy for patients with locally advanced or metastatic BCC. Approximately 100 patients will be enrolled at approximately 40 study sites in the United States as well as sites outside the U.S.Patients will be enrolled into either a metastatic or locally advanced BCC cohort. A minimum of 20 patients will be enrolled in the mBCC cohort. The expected enrollment duration of this study is up to 22 months. All patients will be enrolled in a single stage and a control group will not be used, as there is no accepted standard of care and no data suggesting spontaneous responses in advanced BCC. All patients will receive 150 mg of GDC-0449 daily by mouth, beginning on Day 1, and continuously until one of the following occurs: Disease progression, Intolerable toxicity most probably attributable to GDC-0449 or Withdrawal from the study. Patients who discontinue study drug treatment will be followed for survival approximately every 3 months until death, loss to follow-up, or study termination by the sponsor Genentech.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  09/H0720/66

• Date of REC Opinion

  29 Apr 2009

• REC opinion

  Favourable Opinion