Table 6

Clinical trials: Designs for treatment of skin IEN

Organ transplant patientsPatients with multiple AK
Study designRandomized, placebo-controlled, double-blind Phase III trial of a new treatment agent given for 3 yearsRandomized, placebo-controlled, double-blind Phase III trial of a new treatment agent
EndpointsPrimary, new, pathologically confirmed SCCPrimary, regression of existing AK to clinically normal-appearing skin and extent of new AK in 5 × 5-cm surface area
Secondary, new AKs
Secondary, new pathologically confirmed SCCs
Monitoring schedule12-week intervals (217)Monthly intervals (240)
Biopsy scheduleBaselineBaseline
Suspicious lesions as they occurSuspicious lesions as they occur
Sample size with assumptionsn = 103 per group (206 total)n = 328/group (656 total)
• α = 0.05, power = 80%• α = 0.05, power = 80%
• 50% of controls will have progressive AK to biopsy-proven SCC• 14% of controls will develop SCCs, and 50% of controls will develop new AK
• Ability to detect 40% reduction in AK progression to SCC• Ability to detect complete clearance of AK in the 5 × 5-cm area of skin in at least 33% more treated patients compared with placebo.
• Ability to detect 40% reduction in SCC incidence and/or a 40% reduction in the incidence of new AK