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: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: argenx
• Treatments: Prednisone

Criteria

Inclusion Criteria:

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

1. understand the requirements of the study

2. provide written informed consent

3. comply with the study protocol procedures.

• The participant is male or female, and aged ≥18 years (≥20 years in Japan) 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:

1. Male participants:

• Must agree to use an acceptable method of contraception and not donate sperm from the time that the ICF is signed until they have received their last dose of IMP.

2. 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 a highly effective or acceptable contraception method until at least 90 days after they receive their last dose of IMP.

The full list of inclusion criteria can be found in the protocol.

Exclusion 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, conventional immunosuppressants or dapsone.

• Known contraindication to OCS therapy.

• Clinically significant uncontrolled active or chronic, bacterial, viral, or fungal infection at screening.

• Positive nasopharyngeal swab test for SARS-CoV-2 at screening.

• 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 EFG

• 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 or within 90 days after last dose of the IMP

The full list of exclusion criteria can be found in the protocol.