An Open-label, Non-comparative, Treatment Protocol for the Use of BAY 43-9006 (Sorafenib) in Patients With Advanced Renal Cell Carcinoma
Renal cell carcinoma (RCC) accounts for approximately 3 % of all cancers. RCC can be cured
if diagnosed and treated when still localized to the kidney or immediate surrounding
tissues. Since most of the RCC tumours are diagnosed when still localized, approximately
40 % of all patients survive 5 years. The median survival of Stage IV RCC after diagnosis is
8 to 12 months and the 5-year survival is less than 10 %. Surgery has been the primary
therapy for RCC for more than a century. Until recently, metastatic disease has been
refractory to any systemic therapy. Despite recent advances in immunotherapy (e.g.
Interferon and Interleukin-2), the response rates remain low (15 %) and few patients
experience durable remission. Surgical and radiation therapies for Stage IV disease are
generally limited to palliation of symptoms. For the majority of patients, metastatic RCC is
incurable and patients should be considered candidates for clinical trials when appropriate.
In summary, the available therapies for advanced unresectable and/or metastatic RCC have
limited clinical values, with response rates ranging from 6-20 % and little impact on the
natural history of the disease. Furthermore, the toxicities associated with these agents can
be severe, requiring careful patient selection, and this dramatically decreases the number
of patients who may benefit from therapy. Advanced RCC remains incurable and the need for
more effective therapies is high.
This is a non-randomized, open-label treatment protocol for patients with advanced RCC.
Patients will be treated with 400 mg oral sorafenib twice a day on a continuous. Patients in
this protocol may continue to be treated with sorafenib as a single agent until any of the
following criteria for drug or protocol discontinuation is reached:
1. Progression of disease.
2. The patient is unlikely to benefit from further treatment with sorafenib as judged by
3. Intolerable toxicity of the drug.
4. Withdrawal of consent for any reason.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To evaluate the efficacy (time to progression and progression free survival) of sorafenib in patients with advanced RCC
Vichien Srimuninnimit, Ass.Prof.
Siriraj Hospital, Bangkok, Thailand
Thailand: Food and Drug Administration