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An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD+)

A phase 3 study to evaluate the long-term safety, tolerability and efficacy of efgartigimod PH20 SC in adult participants with bullous pemphigoid. (BALLAD+)

Ujiie Hideyuki

Hokkaido University Hospital

Kita14, Nishi5, Kita-Ku, Sapporo-shi, Hokkaido

sayaka.kakoi@thermofisher.com

Yamaji Hideomi

PPD-SNBL K.K.

St. Lukes tower 12 F, 8-1, Akashi-cho, Chuo-ku, Tokyo

+81-90-6106-5298

hideomi.yamaji@thermofisher.com

Complete

Mar. 06, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

Participants can be included in the study only if all of the following criteria apply:
1. Has completed the week 36 visit of ARGX-113-2009
2. Is capable of providing signed informed consent, and complying with protocol requirements
3. Agrees to use contraceptive measures consistent with local regulations and the following:
a. Male participants
b. WOCBP must have a negative urine pregnancy test at
baseline before receiving IMP.

Participants will be excluded from the study if any of the following criteria apply:
1. Clinically significant disease, recent major surgery (within 3 months of baseline), or intention to have surgery during the study: or any other medical condition that, in the investigators opinion, would confound the results of the study or put the participant at undue risk
2. Known hypersensitivity to IMP or 1 of its excipients
3. Permanently discontinued IMP in ARGX-113-2009 due to an AE considered related to IMP and for whom the benefit/risk balance is not considered positive

Both

Bullous pemphigoid

A loading dose at the initiation of the treatment or Day1 and Day8 of the continuous treatment, followed by weekly maintenance doses of 1000 mg will be used.

- Incidence and severity of treatment-emergent AEs, SAEs, and AESIs
- Rate of treatment discontinuation because of safety concerns

Proportions of participants achieving:
- CR while off OCS for >=8 weeks
- CR or PR while off OCS for >=8 weeks
- CR while on minimal OCS therapy for >=8 weeks.
- CR while off both OCS and efgartigimod PH20 SC for >=8 weeks
- CR or PR while off both OCS and efgartigimod PH20 SC for >=8 weeks
Duration of sustained remission
Proportion of participants who relapse
Time to relapse
Incidence and severity of relapse
BPDAI activity scores, IGA-BP scores, and itch NRS over time
Rate of treatment failure

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