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

JRCT ID: jRCT2011230060

Registered date:06/03/2024

Basic Information

• Recruitment status: Suspended
• Health condition(s) or Problem(s) studied: Bullous pemphigoid
• Date of first enrollment: 06/03/2024
• Target sample size: 12
• Countries of recruitment: Bulgaria,Japan,Croatia,Japan,France,Japan,Germany,Japan,Greece,Japan,Hungary,Japan,Italy,Japan,Netherlands,Japan,Poland,Japan,Spain,Japan,United Kingdom,Japan,Israel,Japan,Serbia,Japan,Australia,Japan,United States,Japan,China,Japan,Czech Republic,Japan,Canada,Japan,Latvia,Japan,Romania,Japan,Slovakia,Japan
• Study type: Interventional
• Intervention(s): 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.

Outcome(s)

Primary Outcome

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

Secondary Outcome

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

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

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.

Exclude criteria

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

Related Information

• Primary Sponsor: Ujiie Hideyuki
• Secondary ID(s): NCT05681481,2021-003063-10