A Phase II Study of BAY 43-9006 (NSC 724772) in Unresectable Stage III and IV Melanoma (IND 69,869)
I. Determine the efficacy of sorafenib, in terms of anti-tumor effects and proportion of
clinical responses, in patients with previously untreated unresectable stage III or stage IV
I. Correlate the efficacy of this drug with the presence of mutant or wild-type BRAF gene in
tumors of these patients.
II. Determine the toxicity profile of this drug in these patients. III. Correlate serum
cryptic collagen epitopes with the extent of tumor burden, invasion, and metastasis in
patients treated with this drug.
IV. Determine the potential of serum cryptic collagen epitopes to serve as a surrogate
marker for monitoring the course of disease in patients treated with this drug.
OUTLINE: This is a multicenter study. Patients are stratified according to presence of BRAF
gene mutation in tumor sample (yes vs no).
Patients receive oral sorafenib twice daily on days 1-28. Courses repeat every 28 days in
the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed annually.
PROJECTED ACCRUAL: A total of 26-74 patients (13-37 per stratum) will be accrued for this
study within 5.2-18.5 months.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Response rate (RR) defined as is either a complete or a partial response using RECIST criteria
The overall response rate along with subgroup-specific response rates will be estimated at the end of the trial along with 95% confidence interval.
Montefiore Medical Center
United States: Food and Drug Administration
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