UAB 9405 
March 16, 1994 
PAGE - 19 
11.0 STATISTICAL CONSIDERATIONS 
11.1 This is a phase la, non-randomized open label study to examine the toxicity of 
single and repetitive doses of a CEA polynucleotide vaccine. Patients will receive 
three single vaccinations with 0.1, 0.3 and 1.0 mg of vaccine. If no significant 
toxicity occurs (^rade II) due to single vaccine administration, subsequent 
groups will receive 0.3 mg every 3 weeks times 3 and if no toxicity 1 .0 mg every 
3 weeks times 3. Patient groups will comprise 3 patients. Since no significant 
side effects should occur with this treatment strategy, the presence of grade II 
toxicity due to vaccine administration will stop accrual with subsequent 
consultation with FDA regarding proceeding with the study. 
11.2 The immune response to these vaccinations will be assayed by both humoral and 
cellular immune assays. These results will be used as a guide for future phase I/II 
trials that vdll define optimal biologic dose/schedule using larger patient numbers 
for statistical significance. 
11.3 The proposed accrual of 15 patients should be accomplished in a 1-year time 
frame. 
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Recombinant DNA Research, Volume 19 
