A phase 2/3 study of efgartigimod PH20 SC in adult participants with bullous pemphigoid

JRCT ID: jRCT2011220047

Registered date:26/03/2023

Basic Information

• Recruitment status: Suspended
• Health condition(s) or Problem(s) studied: Bullous pemphigoid(BP)
• Date of first enrollment: 26/03/2023
• 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,UK,Japan,Serbia,Japan,Israel,Japan,USA,Japan,China,Japan,Australia,Japan,Latvia,Japan,Romania,Japan,Slovakia,Japan,Czech Republic,Japan
• Study type: Interventional
• Intervention(s): ARGX-113 PH20 SC will be administered by subcutaneous injection on day 1 (baseline; week 0) and day 8 (week 1) at a dose of 2000 mg (via 2 separate 1000 mg injections), followed by weekly injections of ARGX-113 PH20 SC 1000 mg from week 2 through week 36 of the treatment period.

Outcome(s)

• Primary Outcome: Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for >=8 weeks at week 36.

Key inclusion & exclusion criteria

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

Include criteria

*The participant is willing and able to do the following:

a. understand the requirements of the study
b. provide written informed consent
c. comply with the study protocol procedures.

* The participant is male or female and has reached the age of consent at the time of signing the informed consent form (ICF).

* Participants have clinical signs of BP.

* Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
a) Male participants:
- Must agree to use an acceptable method of contraception from the time that the ICF is signed until the date of their last dose of IMP.

b) Female participants:
- Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before study intervention can be administered.

- WOCBP must agree to use at least one of the contraception methods identified in the protocol from the time that the ICF is signed until the date of their last dose of IMP.

Exclude criteria

Other forms of pemphigoid or other autoimmune bullous diseases (AIBDs).

*Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior to the baseline visit

*Use of BP treatments other than oral corticosteroids (OCS), topical corticosteroids (TCS), conventional immunosuppressants or dapsone.

*Known contraindication to OCS therapy

*Active or chronic infection at screening

*Positive COVID-19 test result at screening (testing performed if required per local regulations).

*History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for >=3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study:

- Basal cell or squamous cell skin cancer
- Carcinoma in situ of the cervix
- Carcinoma in situ of the breast
- Incidental histological finding of prostate cancer

*Clinical evidence of other significant serious diseases, have had a recent surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the patient at undue risk

*Use of an investigational product within 3 months before the first dose of IMP

*Previously participated in a clinical study with efgartigimod

*Known hypersensitivity to any of the components of the administered treatments

*Positive serum test at screening for an active infection:

- HBV
- HCV
- HIV

*Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse

*Pregnant or lactating females and those who intend to become pregnant during the study

Related Information

• Primary Sponsor: Ujiie Hideyuki