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

An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long Term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

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.

Study Overview

• Status: Terminated
• Conditions: Bullous Pemphigoid
• Intervention / Treatment: Biological: efgartigimod PH20 SC; Drug: Prednisone

• Study Type: Interventional
• Enrollment (Actual): 64
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Kogarah, Australia, 2217
    • Premier Specialists
• Bulgaria
  • Sofia, Bulgaria, 1431
    • Diagnostic and Consulting Center Aleksandrovska EOOD
• China
  • Chengdu, China, 610041
    • West China Hospital of Sichuan University
  • Chongqing, China, 400016
    • The First Affiliated Hospital of Chongqing Medical University
  • Shanghai, China, 200025
    • Ruijin Hospital Shanghai Jiaotong University School of Medicine
• Croatia
  • Zagreb, Croatia, 10000
    • Poliklinika Solmed
• Czechia
  • Prague, Czechia, 180 00
    • Fakultní nemocnice Bulovka
• Germany
  • Berlin, Germany, 10117
    • Charité - Universitätsmedizin Berlin
  • Dresden, Germany, 01307
    • Universitatsklinikum Carl Gustav Carus an der TU Dresden
  • Düsseldorf, Germany, 40225
    • Universitätsklinikum Düsseldorf
  • Kiel, Germany, 24105
    • Universitatsklinikum Schleswig-Holstein
  • München, Germany, 80337
    • LMU Klinikum der Universität
  • Würzburg, Germany, 97080
    • Universitätsklinikum Würzburg
• Greece
  • Athens, Greece, 16121
    • Hospital of Venereal and Skin Diseases A.Syggros
  • Thessaloniki, Greece, 54643
    • Hospital Of Skin And Venereal Diseases of Thessaloniki
• Hungary
  • Budapest, Hungary, 1085
    • Semmelweis Egyetem
• Israel
  • Ramat Gan, Israel, 5262100
    • Sheba Medical Center - PPDS
• Italy
  • Catania, Italy, 95123
    • Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
  • Florence, Italy, 50122
    • Azienda USL Toscana Centro - Ospidale Piero Palagi
  • Florence, Italy, 50125
    • Azienda Sanitaria Di Firenze
  • Genova, Italy, 16132
    • Ospedale Policlinico San Martino
  • Milan, Italy, 20122
    • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
  • Pavia, Italy, 27100
    • Fondazione IRCCS Policlinico San Matteo di Pavia
  • Roma, Italy, 00167
    • IDI IRCCS - Istituto Dermopatico dell'Immacolata
  • Rome, Italy, 00168
    • Fondazione Policlinico Universitario A. Gemelli
• Japan
  • Sapporo, Japan, 060-8648
    • Hokkaido University Hospital
• Netherlands
  • Groningen, Netherlands, 9713 GZ
    • Universitair Medisch Centrum Groningen
• Serbia
  • Belgrade, Serbia, 11000
    • University Clinical Center of Serbia - PPDS
• Slovakia
  • Bratislava, Slovakia, 821 06
    • Univerzitna nemocnica Bratislava
  • Trnava, Slovakia, 91702
    • Fakultna Nemocnica Trnava
• Spain
  • Granada, Spain, 18016
    • Hospital Universitario Clínico San Cecilio
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre
  • Valencia, Spain, 46017
    • Hospital Universitario Doctor Peset
• United Kingdom
  • London, United Kingdom, SE1 9RT
    • Guy's and St Thomas' NHS Foundation Trust
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Medical Dermatology Specialists
  • California
    • Fountain Valley, California, United States, 92708
      • First OC Dermatology
  • Florida
    • Miami, Florida, United States, 33173
      • Miami Dermatology And Laser Institute
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Hospital
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Saint Louis University
  • Ohio
    • Fairborn, Ohio, United States, 45324
      • Wright State Physicians

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: efgartigimod PH20 SC; participants receiving efgartigimod PH20 SC on top of Prednisone	Biological: efgartigimod PH20 SC; Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer; Drug: Prednisone; Oral Prednisone

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Achieving CRoff for ≥ 8 Weeks	CRoff = complete remission while receiving efgartigimod PH20 SC and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.	Up to 56 weeks
Number of Participants Achieving CRoff or PRoff for ≥ 8 Weeks	CRoff / PRoff = complete or partial remission while receiving efgartigimod PH20 SC and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus. Partial remission is defined as the presence of only new transient lesions.	Up to 56 weeks
Number of Participants Achieving CRmin for ≥ 8 Weeks	Minimal oCRmin = complete remission while being on minimal dose of OCS for ≥ 8 weeks. OCS = oral corticosteroid. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus Minimal OCS therapy is defined as ≤0.10 mg/kg/day of prednisone (or an equivalent dose of another oral corticosteroid)	Up to 56 weeks
Number of Participants Achieving Complete Remission While Off Both Oral Corticosteroids and Efgartigimod PH20 SC for ≥ 8 Weeks	CR = complete remission; OCS = oral corticosteroids; Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus	Up to 56 weeks
Number of Participants Achieving CR or PR While Off Both OCS and Efgartigimod PH20 SC for ≥ 8 Weeks	CR = complete remission; PR = partial remission; OCS = oral corticosteroids Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus. Partial remission is defined as the presence of only new transient lesions.	Up to 56 weeks
Duration of Sustained Remission	Sustained remission is defined as healing of lesions with no nontransient lesions (ie, BPDAI activity score of 0) and absence of pruritus while the participant was off concurrent BP therapy (and, for participants enrolled prior to protocol amendment 2, efgartigimod PH20 SC) for ≥8 weeks. New lesions that heal within 1 week or pruritus lasting <1 week and clearing without treatment were not considered to change the condition of sustained remission.	Up to 56 weeks
Number of Participants Who Relapsed	Relapse is defined as the appearance of 3 or more new lesions a month or at least 1 large lesion that did not heal within 1 week, or extension of established lesions or daily pruritus in a participant who had achieved CDA (Control of disease activity): the point at which new lesions cease to form and established lesions begin to heal, and pruritic symptoms start to abate	Up to 56 weeks
Time to Relapse	Relapse is defined as the appearance of 3 or more new lesions a month or at least 1 large lesion that did not heal within 1 week, or extension of established lesions or daily pruritus in a participant who had achieved CDA (Control of disease activity): the point at which new lesions cease to form and established lesions begin to heal, and pruritic symptoms start to abate	Up to 56 weeks
BPDAI Activity Score, Percent Change From Baseline to Last Assessment	The Bullous Pemphigoid Disease Area Index (BPDAI) is an internationally validated tool to objectively measure disease activity. The BPDAI differentiates scores for skin (erosions/blisters and urticaria/erythema) and mucous membrane activity in several anatomical locations. BPDAI activity scores range from 0 to 360, with a higher score representing more severe disease.	Up to 56 weeks
IGA-BP Score at Last Assessment	The Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) is a tool used to asses BP disease activity and severity. The IGA-BP categorizes the severity of BP on a numerical scale of 0 (clear) to 4 (severe).	Up to 56 weeks
Itch NRS 24-hour Average Score, Change From Baseline to Last Assessment	The Itch Numerical Rating Scale (NRS) is used to indicate pruritic symptoms of BP. The score varies between 0 (best outcome) to 10 (worst outcome)	Up to 56 weeks
Number of Participants Who Failed Treatment	Treatment failure is defined as the absence of CDA despite receiving efgartigimod PH20 SC with escalated dosages of prednisone (or equivalent OCS)	Up to 56 weeks

Collaborators and Investigators

• Sponsor: argenx

Study record dates

Study Major Dates

• Study Start (Actual): March 22, 2023
• Primary Completion (Actual): March 20, 2025
• Study Completion (Actual): March 20, 2025

Study Registration Dates

• First Submitted: December 8, 2022
• First Submitted That Met QC Criteria: December 27, 2022
• First Posted (Actual): January 12, 2023

Study Record Updates

• Last Update Posted (Actual): February 25, 2026
• Last Update Submitted That Met QC Criteria: February 6, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Autoimmune Diseases; Immune System Diseases; Skin Diseases; Skin Diseases, Vesiculobullous; Skin and Connective Tissue Diseases; Pemphigoid, Bullous; Polycyclic Compounds; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Pregnadienediols; Prednisone
• Other Study ID Numbers: ARGX-113-2010; 2024-515832-59-00 (Ctis)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes