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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