A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

NCT05681481 | Terminated Phase 3 Results FDA Drug

• 2 other identifiers: ARGX-113-20102024-515832-59-00
• Study Type: interventional
• Target: 64
• Locations: 17 countries
• Sites: 40

Brief Summary

The purpose of this study is to evaluate the safety of efgartigimod PH20 SC over a longer period of time in adult participants with moderate-to-severe bullous pemphigoid (BP) who have completed ARGX-113-2009 study. The study will also evaluate the efficacy of efgartigimod PH20 SC. Eligible participants can roll over from the main study (ARGX-113-2009) to this open-label extension study (ARGX-113-2010). The study consists of a treatment period of up to 48 weeks in which participants could receive efgartigimod PH20 SC according to their clinical status. After the first 5 visits, the participants will visit the study centres at least once every 4 weeks. The participants who are not receiving efgartigimod PH20 SC (after the main study or currently on the study), will enter an observation period with study visits at least once every 8 weeks. If the participant relapses, they can re-enter the treatment period where they will receive efgartigimod PH20 SC. The treatment and observation period is followed by a follow-up period of 8 weeks. Oral or topical corticosteroids can be administered at the investigator's discretion.

Conditions

Bullous Pemphigoid

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment-emergent AEs, SAEs and AESIs

  Adverse events, Serious Adverse event and Adverse events of special interest. Adverse events in the 'Infections and infestations' SOC were defined as AESIs because efgartigimod causes a transient reduction in total IgG levels.

  Up to 56 weeks

• Number of Participants Who Discontinued Treatment Because of Safety Concerns

  Up to 56 weeks

Secondary Outcomes (12)

• Number of Participants Achieving CRoff for ≥ 8 Weeks

  Up to 56 weeks

• Number of Participants Achieving CRoff or PRoff for ≥ 8 Weeks

  Up to 56 weeks

• Number of Participants Achieving CRmin for ≥ 8 Weeks

  Up to 56 weeks

• Number of Participants Achieving Complete Remission While Off Both Oral Corticosteroids and Efgartigimod PH20 SC for ≥ 8 Weeks

  Up to 56 weeks

• Number of Participants Achieving CR or PR While Off Both OCS and Efgartigimod PH20 SC for ≥ 8 Weeks

  Up to 56 weeks

Study Arms (1)

efgartigimod PH20 SC

EXPERIMENTAL

participants receiving efgartigimod PH20 SC on top of Prednisone

Biological: efgartigimod PH20 SC; Drug: Prednisone

Interventions

efgartigimod PH20 SC BIOLOGICAL

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

efgartigimod PH20 SC

Prednisone DRUG

Oral Prednisone

efgartigimod PH20 SC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Has completed the week 36 visit of ARGX-113-2009
• Is capable of providing signed informed consent and complying with protocol requirements
• Agrees to use contraceptive measures consistent with local regulations and the following: Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving the study drug and must use one of the contraception methods described in the protocol from signing the ICF until the last dose of the study drug

You may not qualify if:

• Clinically significant disease, recent major surgery (within 3 months of baseline), or intends to have surgery during the study; or any other medical condition that, in the investigator's opinion would confound the results of the study or put the participant at undue risk
• Known hypersensitivity to the study drug or 1 of its excipients
• Permanently discontinued IMP in ARGX-113-2009 due to an adverse event (AE) considered related to the study drug and for whom the benefit/risk balance is not considered positive

Sponsors & Collaborators

• argenx: lead

Study Sites (40)

Medical Dermatology Specialists

Phoenix, Arizona, 85006, United States

Location

First OC Dermatology

Fountain Valley, California, 92708, United States

Location

Miami Dermatology and Laser Institute

Miami, Florida, 33173, United States

Location

University of Michigan Hospital

Ann Arbor, Michigan, 48109, United States

Location

Saint Louis University

St Louis, Missouri, 63110, United States

Location

Wright State Physicians

Fairborn, Ohio, 45324, United States

Location

Premier Specialists

Kogarah, 2217, Australia

Location

Diagnostic and Consulting Center Aleksandrovska EOOD

Sofia, 1431, Bulgaria

Location

West China Hospital of Sichuan University

Chengdu, 610041, China

Location

The First Affiliated Hospital of Chongqing Medical University

Chongqing, 400016, China

Location

Ruijin Hospital Shanghai Jiaotong University School of Medicine

Shanghai, 200025, China

Location

Poliklinika Solmed

Zagreb, 10000, Croatia

Location

Fakultni nemocnice Bulovka

Prague, 180 00, Czechia

Location

Charité - Universitätsmedizin Berlin

Berlin, 10117, Germany

Location

Universitätsklinikum Carl Gustav Carus an der TU Dresden

Dresden, 01307, Germany

Location

Universitatsklinikum Dusseldorf

Düsseldorf, 40225, Germany

Location

Universitatsklinikum Schleswig-Holstein

Kiel, 24105, Germany

Location

LMU Klinikum der Universität

München, 80337, Germany

Location

Universitätsklinikum Würzburg

Würzburg, 97080, Germany

Location

Hospital of Venereal and Skin Diseases A.Syggros

Athens, 16121, Greece

Location

Hospital Of Skin And Venereal Diseases of Thessaloniki

Thessaloniki, 54643, Greece

Location

Semmelweis Egyetem

Budapest, 1085, Hungary

Location

Sheba Medical Center - PPDS

Ramat Gan, 5262100, Israel

Location

Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele

Catania, 95123, Italy

Location

Azienda USL Toscana Centro - Ospidale Piero Palagi

Florence, 50122, Italy

Location

Azienda Sanitaria Di Firenze

Florence, 50125, Italy

Location

Ospedale Policlinico San Martino

Genova, 16132, Italy

Location

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Fondazione IRCCS Policlinico San Matteo di Pavia

Pavia, 27100, Italy

Location

IDI IRCCS - Istituto Dermopatico dell'Immacolata

Roma, 00167, Italy

Location

Fondazione Policlinico Universitario A. Gemelli

Rome, 00168, Italy

Location

Hokkaido University Hospital

Sapporo, 060-8648, Japan

Location

Universitair Medisch Centrum Groningen

Groningen, 9713 GZ, Netherlands

Location

University Clinical Center of Serbia - PPDS

Belgrade, 11000, Serbia

Location

Univerzitna nemocnica Bratislava

Bratislava, 821 06, Slovakia

Location

Fakultna nemocnica Trnava

Trnava, 91702, Slovakia

Location

Hospital Universitario Clínico San Cecilio

Granada, 18016, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario Doctor Peset

Valencia, 46017, Spain

Location

Guy's and St Thomas' NHS Foundation Trust

London, SE1 9RT, United Kingdom

Location

MeSH Terms

Conditions

Pemphigoid, Bullous

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Results Point of Contact

• Title: Regulatory Manager
• Organization: argenx BV

regulatory@argenx.com

+32 93103400

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2022
• First Posted: January 12, 2023
• Study Start: March 22, 2023
• Primary Completion: March 20, 2025
• Study Completion: March 20, 2025
• Last Updated: February 25, 2026
• Results First Posted: February 25, 2026

Record last verified: 2026-02

Locations

ES (3); US (6); DE (6); CR (1); IL (1); CZ (1); IT (8); NL (1); SE (1); SL (2); GB (1); JP (1); HU (1); CN (3); GR (2); AU (1); BU (1)