A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

NCT ID: NCT05267600

Last Updated: 2025-10-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 98 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-06-09
• Study Completion Date: 2024-09-13

Brief 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 efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP. This study intends to demonstrate that efgartigimod 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 that intends to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC in participants with BP.
• Part B of the study is a phase 3 evaluation that 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 efgartigimod PH20 SC for the treatment of participants with BP (futility analysis).

Conditions

Bullous Pemphigoid

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

efgartigimod PH20 SC

participants receiving efgartigimod PH20 SC on top of Prednisone

Group Type: EXPERIMENTAL

efgartigimod PH20 SC

Intervention Type: BIOLOGICAL

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

Prednisone

Intervention Type: DRUG

Oral Prednisone

placebo PH20 SC

participants receiving placebo PH20 SC on top of Prednisone

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: OTHER

Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Prednisone

Intervention Type: DRUG

Oral Prednisone

Interventions

efgartigimod PH20 SC

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

Intervention Type: BIOLOGICAL

placebo

Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Intervention Type: OTHER

Prednisone

Oral Prednisone

Intervention Type: DRUG

Eligibility 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 has reached the age of consent at the time of signing the informed consent form (ICF).
• Participants have clinical signs of BP.
• Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and: Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before study intervention can be administered.

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 (TCS), conventional immunosuppressants or dapsone.
• Known contraindication to OCS therapy
• Active, chronic or latent 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 or prevent participants from complying with protocol requirements
• Use of an investigational product within 3 months before the first dose of IMP
• Previously participated in a clinical study with efgartigimod or currently participating in another interventional clinical study
• 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 as assessed by the investigator
• Pregnant or lactating females and those who intend to become pregnant during the study
• Live or live-attenuated vaccine received <4 weeks before baseline visit

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

argenx

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigator site 74 - US0010178

Phoenix, Arizona, United States

Investigator site 6 - US0010138

Fountain Valley, California, United States

Investigator site 121 - US0010092

Redwood City, California, United States

Investigator site 72 - US0010186

Santa Monica, California, United States

Investigator site 10 - US0010153

Castle Rock, Colorado, United States

Investigator site 2 - US0010087

Boca Raton, Florida, United States

Investigator site 21 - US0010152

Clearwater, Florida, United States

Investigator site 1 - US0010017

Miami, Florida, United States

Investigator site 13 - US0010155

West Lafayette, Indiana, United States

Investigator site 35 - US0010156

Louisville, Kentucky, United States

Investigator site 50 - US0010149

Ann Arbor, Michigan, United States

Investigator site 115 - US0010157

Jackson, Mississippi, United States

Investigator site 73 - US0010098

St Louis, Missouri, United States

Investigator site 85 - US0010159

Lebanon, New Hampshire, United States

Investigator site 5 - US0010088

Buffalo, New York, United States

Investigator site 93 - US0010169

New York, New York, United States

Investigator site 4 - US0010137

Fairborn, Ohio, United States

Investigator site 25 - US0010158

Pittsburgh, Pennsylvania, United States

Investigator site 34 - US0010182

Houston, Texas, United States

Investigator site 92 - US0010150

Murray, Utah, United States

Investigator site 3 - US0010151

Morgantown, West Virginia, United States

Investigator site 36 - AU0610013

Fitzroy, , Australia

Investigator site 27 - AU0610006

Kogarah, , Australia

Investigator site 125 - AU0610019

Woolloongabba, , Australia

Investigator site 28 - BG3590018

Sofia, , Bulgaria

Investigator site 14 - BG3590010

Sofia, , Bulgaria

Investigator site 15 - BG3590020

Stara Zagora, , Bulgaria

Investigator site 111 - CN0860064

Beijing, , China

Investigator site 91 - CN0860017

Beijing, , China

Investigator site 95 - CN0860018

Chengdu, , China

Investigator site 116 - CN0860027

Chongqing, , China

Investigator site 108 - CN0860023

Fujian, , China

Investigator site 107 - CN0860021

Guangzhou, , China

Investigator site 110 - CN0860053

Guanzhou, , China

Investigator site 128 - CN0860097

Hefei, , China

Investigator site 124 - CN0860098

Nanchang, , China

Investigator site 94 - CN0860066

Nanyang, , China

Investigator site 127 - CN860020

Shanghai, , China

Investigator site 123 - CN0860095

Shanghai, , China

Investigator site 122 - CN0860065

Ürümqi, , China

Investigator site 109 - CN0860025

Wuhan, , China

Investigator site 126 - CN0860026

Zhengzhou, , China

Investigator site 22 - HR3850003

Split, , Croatia

Investigator site 16 - HR3850002

Zagreb, , Croatia

Investigator site 37 - HR3850001

Zagreb, , Croatia

Investigator site 97 - CZ4200015

Brno, , Czechia

Investigator site 118 - CZ4200016

Hradec Králové, , Czechia

Investigator site 117 - CZ4200013

Plzen-Bory, , Czechia

Investigator site 96 - CZ4200012

Prague, , Czechia

Investigator site 38 - FR0330040

Nice, , France

Investigator site 29 - FR0330029

Rouen, , France

Investigator site 46 - DE0490039

Berlin, , Germany

Investigator site 54 - DE0490030

Dresden, , Germany

Investigator site 80 - DE0490041

Düsseldorf, , Germany

Investigator site 57 - DE0490046

Erlangen, , Germany

Investigator site 51 - DE0490008

Essen, , Germany

Investigator site 52 - DE0490024

Frankfurt am Main, , Germany

Investigator site 53 - DE0490023

Freiburg im Breisgau, , Germany

Investigator site 56 - DE0490028

Kiel, , Germany

Investigator site 45 - DE0490001

Marburg, , Germany

Investigator site 75 - DE0490047

München, , Germany

Investigator site 55 - DE0490026

Würzburg, , Germany

Investigator site 60 - GE0300004

Athens, , Greece

Investigator site 62 - GE0300006

Athens, , Greece

Investigator site 58 - GR0300001

Athens, , Greece

Investigator site 76 - GR030003

Chaïdári, , Greece

Investigator site 61 - GE0300002

Thessaloniki, , Greece

Investigator site 59 - GR0300005

Thessaloniki, , Greece

Investigator site 26 - HU0360023

Budapest, , Hungary

Investigator site 11 - HU0360003

Debrecen, , Hungary

Investigator site 7 - HU0360008

Pécs, , Hungary

Investigator site 39 - IL9720003

Afula, , Israel

Investigator site 63 - IL9720018

Haifa, , Israel

Investigator site 41 - IL9720001

Ramat Gan, , Israel

Investigator site 40 - IL9720002

Tel Aviv, , Israel

Investigator site 65 - IT0390055

Bologna, , Italy

Investigator site 43 - IT0390060

Brescia, , Italy

Investigator site 78 - IT0390039

Catania, , Italy

Investigator site 47 - IT0390031

Florence, , Italy

Investigator site 86 - IT0390067

Florence, , Italy

Investigator site 81 - IT0390030

Genova, , Italy

Investigator site 77 - IT0390062

Milan, , Italy

Investigator site 119 - IT0390066

Parma, , Italy

Investigator site 64 - IT0390061

Pavia, , Italy

Investigator site 23 - IT0390006

Roma, , Italy

Investigator site 42 - IT0390005

Roma, , Italy

Investigator site 30 - IT0390040

Siena, , Italy

Investigator site 103 - JP0810070

Kumamoto, , Japan

Investigator site 99 - JP0810050

Kurume, , Japan

Investigator site 104 - JP0810071

Maebashi, , Japan

Investigator site 100 - JP0810046

Nagakute, , Japan

Investigator site 129 - JP0810073

Niigata, , Japan

Investigator site 101 - JP0810049

Osaka, , Japan

Investigator site 102 - JP0810069

Ōsaka-sayama, , Japan

Investigator site 112 - JP0810045

Sapporo, , Japan

Investigator site 105 - JP0810067

Sendai, , Japan

Investigator site 98 - JP0810043

Tokyo, , Japan

Investigator site 106 - JP0810068

Yokohama, , Japan

Investigator site 87 - LV3710005

Riga, , Latvia

Investigator site 82 - LV3710003

Riga, , Latvia

Investigator site 88 - LV3710004

Riga, , Latvia

Investigator site 66 - NL0310015

Groningen, , Netherlands

Investigator site 17 - PL0480047

Lodz, , Poland

Investigator site 79 - PL0480025

Rzeszów, , Poland

Investigator site 83 - PL0480050

Warsaw, , Poland

Investigator site 18 - PL0480048

Wroclaw, , Poland

Investigator site 19 - PL0480046

Wroclaw, , Poland

Investigator site 90 - RO0400014

Cluj-Napoca, , Romania

Investigator site 89 - RO0400015

Iași, , Romania

Investigator 68 - RS381011

Belgrade, , Serbia

Investigator site 84 - RS3810010

Belgrade, , Serbia

Investigator site 67 - RS3810012

Niš, , Serbia

Investigator site 69 - RS3810009

Novi Sad, , Serbia

Investigator site 113 - SK4210002

Bratislava, , Slovakia

Investigator site 120 - SK4210004

Košice, , Slovakia

Investigator site 114 - SK4210003

Trnava, , Slovakia

Investigator site 32 - ES0340050

Badalona, , Spain

Investigator 24 - ES0340051

Barcelona, , Spain

Investigator site 8 - ES0340053

Granada, , Spain

Investigator site 12 - ES0340025

Madrid, , Spain

Investigator 20 - ES0340029

Madrid, , Spain

Investigator site 49 - ES0340058

Manises, , Spain

Investigator site 31 - ES0340057

Málaga, , Spain

Investigator site 48 - ES0340059

Mieres, , Spain

Investigator site 9 - ES0340052

Seville, , Spain

Investigator site 70 - ES0340061

Valencia, , Spain

Investigator site 33 - UK0440022

Bristol, , United Kingdom

Investigator site 71 - UK0440036

London, , United Kingdom

Investigator site 44 - UK0440037

Southampton, , United Kingdom

Countries

United States; Australia; Bulgaria; China; Croatia; Czechia; France; Germany; Greece; Hungary; Israel; Italy; Japan; Latvia; Netherlands; Poland; Romania; Serbia; Slovakia; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

ARGX-113-2009

Identifier Type: -

Identifier Source: org_study_id