TABLE 2.
Primary efficacy endpoint‐responder rate at EOS visit‐FAS
| Ig‐naïveN = 23 | Ig‐pre‐treatedN = 19 | OverallN = 42 | |
|---|---|---|---|
| Number of responders (%) a | 16 (69.6) | 16 (84.2) | 32 (76.2) |
| 95% Confidence interval d | 47.1; 86.8 | 60.4; 96.6 | 60.5; 87.9 |
| P‐value b | – | – | <0.0001 |
| Number of non‐responders (%) c | 7 (30.4) | 3 (15.8) | 10 (23.8) |
Abbreviations: EOS, end of study; FAS, full analysis set; INCAT, inflammatory neuropathy cause and treatment; N, number of patients.
a
Responders were defined as patients with a decrease of ≥1 point in the adjusted INCAT disability score between baseline and EOS visit (week 24) or the last study visit in case of premature discontinuation.
b
one‐sided Exact Clopper‐Pearson (exact binomial test) against the historical placebo response rate of 33.3%.
c
A patient was also considered as a non‐responder if the patient was early withdrawn due to lack of efficacy of the treatment (ie, insufficient response to IqYmune).
d
Two‐sided Exact Clopper‐Pearson 95% confidence interval.