A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD)
Status:
Not yet recruiting
Trial end date:
2024-04-01
Target enrollment:
Participant gender:
Summary
ARGX-113-2009 is an operationally seamless 2-part, phase 2/3, prospective, global,
multicenter, randomized, double-blinded, placebo-controlled study to investigate the
efficacy, safety, tolerability, immunogenicity, participant-reported outcome measures
(including those assessing participant QoL), PK, and PD of EFG PH20 SC administered via
subcutaneous (SC) injection in adult participants with moderate to severe BP. This study
intends to demonstrate that EFG is an effective and safe treatment for BP, providing
participants with control of disease activity (CDA) and eventually remission while reducing
their cumulative exposure to OCS.
study will consist of 2 parts:
- Part A of the study is a phase 2 evaluation which intends to provide proof of concept
for the therapeutic activity of EFG in participants with BP.
- Part B of the study is a phase 3 evaluation which intends to confirm the results
obtained from part A in a separate, larger group of participants with BP.
An interim analysis will be performed during part A (on data obtained through week 26 for all
Part A participants) to assess the primary endpoint and several secondary endpoints, confirm
the appropriate sample size for part B of the study, and determine whether the efficacy
results observed through week 26 of part A warrant continued study of EFG PH20 SC for the
treatment of participants with BP (futility analysis).
Other than differences in main goals, endpoints, and statistical analyses, parts A and B are
identical in schedule, structure, assessments, and conduct.