Skip to main content

Table 2 Comparison of primary endpoints and key secondary endpoints (ITT population)

From: Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study)

  SD-101 6%
n = 82
Vehicle
n = 87
Nominal
P value
Primary endpoints
 Time to complete target wound closure within 3 months, mean (SD), day (n = 39) 41.6 (25.5) (n = 45) 53.6 (28.6) 0.985
 Complete closure of target wound within 3 months, response rate (n = 79) 49.4% (n = 84) 53.6% 0.390
Secondary endpoints
 Complete closure of target wound within 2 months, response rate (n = 79) 43.0% (n = 84) 42.9% 0.802
 Complete closure of target wound within 1 month, response rate (n = 79) 31.6% (n = 84) 22.6% 0.212
 Change in BSAi of lesional skin at Month 3, % (n = 75) (n = 78)  
  Mean (SEM) −4.4 (1.05) −5.0 (1.53) 0.706
  Median (range) −2.7 (− 37.4, 18.0) −3.0 (− 69.2, 26.5)  
 Change in BSAi of total body wound burden at Month 3, % (n = 75) (n = 79)  
  Mean (SEM) −3.5 (0.94) −2.3 (0.70) 0.900
  Median (range) −1.5 (−42.6, 12.5) −1.4 (−24.1, 17.0)  
 Change in itching score at Day 7 (n = 77) (n = 79)  
  Mean (SEM) −0.5 (0.15) −0.3 (0.14) 0.262
  Median (range) 0.0 (−4, 2) 0.0 (− 4, 3)  
 Change in pain score at Day 7 (n = 77) (n = 80)  
 Mean (SEM) −0.3 (0.29) −0.6 (0.34) 0.098
 Median (range) 0.0 (−6, 10) 0.0 (−8, 10)  
  1. BSAi body surface area index; ITT intent-to-treat; SEM standard error of the mean