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

A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

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).

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 98
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Australia
  • Fitzroy, Australia, 3065
    • Investigator site 36 - AU0610013
  • Kogarah, Australia, 2217
    • Investigator site 27 - AU0610006
  • Woolloongabba, Australia, 4102
    • Investigator site 125 - AU0610019
• Bulgaria
  • Sofia, Bulgaria, 1407
    • Investigator site 28 - BG3590018
  • Sofia, Bulgaria, 1431
    • Investigator site 14 - BG3590010
  • Stara Zagora, Bulgaria, 6003
    • Investigator site 15 - BG3590020
• China
  • Beijing, China, 100006
    • Investigator site 111 - CN0860064
  • Beijing, China
    • Investigator site 91 - CN0860017
  • Chengdu, China
    • Investigator site 95 - CN0860018
  • Chongqing, China, 400016
    • Investigator site 116 - CN0860027
  • Fujian, China, 350004
    • Investigator site 108 - CN0860023
  • Guangzhou, China, 510091
    • Investigator site 107 - CN0860021
  • Guanzhou, China, 510080
    • Investigator site 110 - CN0860053
  • Hefei, China
    • Investigator site 128 - CN0860097
  • Nanchang, China, 330000
    • Investigator site 124 - CN0860098
  • Nanyang, China
    • Investigator site 94 - CN0860066
  • Shanghai, China, 200435
    • Investigator site 123 - CN0860095
  • Shanghai, China, 200025
    • Investigator site 127 - CN860020
  • Wuhan, China, 430031
    • Investigator site 109 - CN0860025
  • Zhengzhou, China, 450003
    • Investigator site 126 - CN0860026
  • Ürümqi, China, 830054
    • Investigator site 122 - CN0860065
• Croatia
  • Split, Croatia, 21000
    • Investigator site 22 - HR3850003
  • Zagreb, Croatia, 10000
    • Investigator site 16 - HR3850002
  • Zagreb, Croatia, 10000
    • Investigator site 37 - HR3850001
• Czechia
  • Brno, Czechia, 656 91
    • Investigator site 97 - CZ4200015
  • Hradec Králové, Czechia, 500 05
    • Investigator site 118 - CZ4200016
  • Plzen-Bory, Czechia, 13, 301 00
    • Investigator site 117 - CZ4200013
  • Prague, Czechia, 180 81
    • Investigator site 96 - CZ4200012
• France
  • Nice, France, 06202
    • Investigator site 38 - FR0330040
  • Rouen, France, 76031
    • Investigator site 29 - FR0330029
• Germany
  • Berlin, Germany, 10117
    • Investigator site 46 - DE0490039
  • Dresden, Germany, 01307
    • Investigator site 54 - DE0490030
  • Düsseldorf, Germany, 40225
    • Investigator site 80 - DE0490041
  • Erlangen, Germany, 91054
    • Investigator site 57 - DE0490046
  • Essen, Germany, 45147
    • Investigator site 51 - DE0490008
  • Frankfurt am Main, Germany, 60590
    • Investigator site 52 - DE0490024
  • Freiburg im Breisgau, Germany, 79104
    • Investigator site 53 - DE0490023
  • Kiel, Germany, 24105
    • Investigator site 56 - DE0490028
  • Marburg, Germany, 35043
    • Investigator site 45 - DE0490001
  • München, Germany, 85006
    • Investigator site 75 - DE0490047
  • Würzburg, Germany, 97080
    • Investigator site 55 - DE0490026
• Greece
  • Athens, Greece, 11525
    • Investigator site 60 - GE0300004
  • Athens, Greece, 11525
    • Investigator site 62 - GE0300006
  • Athens, Greece, 16121
    • Investigator site 58 - GR0300001
  • Chaïdári, Greece, 12462
    • Investigator site 76 - GR030003
  • Thessaloniki, Greece, 54643
    • Investigator site 61 - GE0300002
  • Thessaloniki, Greece, 56429
    • Investigator site 59 - GR0300005
• Hungary
  • Budapest, Hungary, 1085
    • Investigator site 26 - HU0360023
  • Debrecen, Hungary, 4032
    • Investigator site 11 - HU0360003
  • Pécs, Hungary, 7632
    • Investigator site 7 - HU0360008
• Israel
  • Afula, Israel, 1834111
    • Investigator site 39 - IL9720003
  • Haifa, Israel, 3109601
    • Investigator site 63 - IL9720018
  • Ramat Gan, Israel, 5262100
    • Investigator site 41 - IL9720001
  • Tel Aviv, Israel, 64239
    • Investigator site 40 - IL9720002
• Italy
  • Bologna, Italy, 40138
    • Investigator site 65 - IT0390055
  • Brescia, Italy, 25132
    • Investigator site 43 - IT0390060
  • Catania, Italy, 95123
    • Investigator site 78 - IT0390039
  • Florence, Italy, 50122
    • Investigator site 47 - IT0390031
  • Florence, Italy, 50122
    • Investigator site 86 - IT0390067
  • Genova, Italy, 16132
    • Investigator site 81 - IT0390030
  • Milan, Italy, 20122
    • Investigator site 77 - IT0390062
  • Parma, Italy, 43126
    • Investigator site 119 - IT0390066
  • Pavia, Italy, 27100
    • Investigator site 64 - IT0390061
  • Roma, Italy, 00167
    • Investigator site 23 - IT0390006
  • Roma, Italy, 00168
    • Investigator site 42 - IT0390005
  • Siena, Italy, 53100
    • Investigator site 30 - IT0390040
• Japan
  • Kumamoto, Japan, 860-8556
    • Investigator site 103 - JP0810070
  • Kurume, Japan, 830-0011
    • Investigator site 99 - JP0810050
  • Maebashi, Japan, 371-8511
    • Investigator site 104 - JP0810071
  • Nagakute, Japan, 480-1195
    • Investigator site 100 - JP0810046
  • Niigata, Japan, 951-8520
    • Investigator site 129 - JP0810073
  • Osaka, Japan, 545-8586
    • Investigator site 101 - JP0810049
  • Sapporo, Japan, 060-8648
    • Investigator site 112 - JP0810045
  • Sendai, Japan, 980-8574
    • Investigator site 105 - JP0810067
  • Tokyo, Japan, 113-8431
    • Investigator site 98 - JP0810043
  • Yokohama, Japan, 236-0004
    • Investigator site 106 - JP0810068
  • Ōsaka-sayama, Japan, 589-8511
    • Investigator site 102 - JP0810069
• Latvia
  • Riga, Latvia, 1001
    • Investigator site 87 - LV3710005
  • Riga, Latvia, 1003
    • Investigator site 82 - LV3710003
  • Riga, Latvia, 1003
    • Investigator site 88 - LV3710004
• Netherlands
  • Groningen, Netherlands, 9713
    • Investigator site 66 - NL0310015
• Poland
  • Lodz, Poland, 90-647
    • Investigator site 17 - PL0480047
  • Rzeszów, Poland, 35055
    • Investigator site 79 - PL0480025
  • Warsaw, Poland, 00-716
    • Investigator site 83 - PL0480050
  • Wroclaw, Poland, 50-220
    • Investigator site 18 - PL0480048
  • Wroclaw, Poland, 51-685
    • Investigator site 19 - PL0480046
• Romania
  • Cluj-Napoca, Romania, 400006
    • Investigator site 90 - RO0400014
  • Iași, Romania, 700111
    • Investigator site 89 - RO0400015
• Serbia
  • Belgrade, Serbia, 110000
    • Investigator 68 - RS381011
  • Belgrade, Serbia, 11040
    • Investigator site 84 - RS3810010
  • Niš, Serbia, 18000
    • Investigator site 67 - RS3810012
  • Novi Sad, Serbia, 21000
    • Investigator site 69 - RS3810009
• Slovakia
  • Bratislava, Slovakia, 4210002
    • Investigator site 113 - SK4210002
  • Košice, Slovakia, 040 11
    • Investigator site 120 - SK4210004
  • Trnava, Slovakia, 91702
    • Investigator site 114 - SK4210003
• Spain
  • Badalona, Spain, 08916
    • Investigator site 32 - ES0340050
  • Barcelona, Spain, 08003
    • Investigator 24 - ES0340051
  • Granada, Spain, 18016
    • Investigator site 8 - ES0340053
  • Madrid, Spain, 28034
    • Investigator site 12 - ES0340025
  • Madrid, Spain, 28041
    • Investigator 20 - ES0340029
  • Manises, Spain, 46940
    • Investigator site 49 - ES0340058
  • Mieres, Spain, 33611
    • Investigator site 48 - ES0340059
  • Málaga, Spain, 29009
    • Investigator site 31 - ES0340057
  • Seville, Spain, 41009
    • Investigator site 9 - ES0340052
  • Valencia, Spain, 46017
    • Investigator site 70 - ES0340061
• United Kingdom
  • Bristol, United Kingdom, BS2 8HW
    • Investigator site 33 - UK0440022
  • London, United Kingdom
    • Investigator site 71 - UK0440036
  • Southampton, United Kingdom, SO16 6YD
    • Investigator site 44 - UK0440037
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Investigator site 74 - US0010178
  • California
    • Fountain Valley, California, United States, 92708
      • Investigator site 6 - US0010138
    • Redwood City, California, United States, 94063
      • Investigator site 121 - US0010092
    • Santa Monica, California, United States, 90404
      • Investigator site 72 - US0010186
  • Colorado
    • Castle Rock, Colorado, United States, 80109
      • Investigator site 10 - US0010153
  • Florida
    • Boca Raton, Florida, United States, 33428
      • Investigator site 2 - US0010087
    • Clearwater, Florida, United States, 33756
      • Investigator site 21 - US0010152
    • Miami, Florida, United States, 33173
      • Investigator site 1 - US0010017
  • Indiana
    • West Lafayette, Indiana, United States, 47906
      • Investigator site 13 - US0010155
  • Kentucky
    • Louisville, Kentucky, United States, 40217
      • Investigator site 35 - US0010156
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Investigator site 50 - US0010149
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • Investigator site 115 - US0010157
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Investigator site 73 - US0010098
  • New Hampshire
    • Lebanon, New Hampshire, United States, 03756
      • Investigator site 85 - US0010159
  • New York
    • Buffalo, New York, United States, 14203
      • Investigator site 5 - US0010088
    • New York, New York, United States, 10016
      • Investigator site 93 - US0010169
  • Ohio
    • Fairborn, Ohio, United States, 45324
      • Investigator site 4 - US0010137
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • Investigator site 25 - US0010158
  • Texas
    • Houston, Texas, United States, 77004
      • Investigator site 34 - US0010182
  • Utah
    • Murray, Utah, United States, 84107
      • Investigator site 92 - US0010150
  • West Virginia
    • Morgantown, West Virginia, United States, 26505
      • Investigator site 3 - US0010151

Participation Criteria

Eligibility Criteria

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

Description

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.

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 (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

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

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
Placebo Comparator: placebo PH20 SC; participants receiving placebo PH20 SC on top of Prednisone	Drug: Prednisone; Oral Prednisone; Other: placebo; Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With CRoff at Week 36	CRoff = complete remission while receiving efgartigimod PH20 SC or placebo 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.	at week 36

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative OCS Dose	OCS = oral corticosteroid	up to week 36
Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36	IGA-BP = Investigator Global Assessment of Bullous Pemphigoid. IGA-BP scores range from 0-4, with a higher score representing severe BP. OCS = oral corticosteroid	at week 36
Number of Participants With CDA Who Remained Free of Relapse Through Week 36	Control of disease activity (CDA) is defined as the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate. 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.	up to week 36
Number of Participants With CRmin at Week 36	CRmin = complete remission receiving minimal oral corticosteroids for at least 8 weeks. Minimal oral corticosteroid (OCS) therapy is defined as ≤0.1 mg/kg/day of prednisone (or equivalent).	at week 36
Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score	Itch NRS = Itch Numerical Rating Scale. The Itch NRS scores range from 0-10 with 10 representing the worst imaginable itch.	up to week 36
Changes From Baseline in the BPDAI Total Activity Score	BPDAI = Bullous Pemphigoid Disease Area Index (BPDAI) Total Activity Score is a tool to objectively measure disease activity. BPDAI scores range from 0 to 360 with a higher score indicating more severe disease (max 240 for total skin activity and 120 for mucosal activity).	up to week 36
Time to CDA	CDA = control of disease activity, the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate.	up to week 36
Time to CR	CR = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus.	up to week 36
Time to CRmin	CRmin = complete remission, the absence of new lesions, complete healing of existing lesions, and absence of pruritus while being on minimal dose of OCS for ≥8 Weeks. OCS = oral corticosteroid. Minimal OCS therapy is defined as an oral prednisone dosage of ≤0.10 mg/kg/day (or equivalent).	up to week 36
Time to CRoff/PRoff	CRoff= complete remission (the absence of new lesions, complete healing of existing lesions, and absence of pruritus) while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. PRoff = partial remission (the presence of only new transient lesions) while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks.	up to week 36
Time to CRoff	CRoff is defined as the absence of new lesions, complete healing of existing lesions, and absence of pruritus while being off OCS therapy for ≥8 weeks.	up to week 36
Time From CDA to Achieve Relapse	CDA is defined as the point at which new lesions ceased to form, established lesions began to heal, and pruritic symptoms started to abate. 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.	up to week 36
Number of Participants Who Receive Rescue Therapy Before Week 36		at week 36
The AIS From the GTI	The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) can range from -346 to 439 with a higher score representing greater corticosteroid toxicity. If a study drug is effective at lowering greater corticosteroid toxicity over time, the score will be lower.	up to week 36
The CWS From the GTI	The Cumulative Worsening Score (CWS) from the Glucocorticoid Toxicity Index (GTI) can range from 0 to 439 with a higher score representing greater corticosteroid toxicity.	up to week 36
EQ-5D-5L VAS Scores Over Time	The EuroQol 5-Dimension 5-Level Visual Analog Scale (EQ-5D-5L VAS) scores range from 0-100 with 0 representing the worst health.	up to week 36
DLQI Scores Over Time	Dermatology Life Quality Index (DLQI) assess the participant's perception of the impact of skin diseases on different aspects of their health-related QoL. Scores range from 0 to 30 with a higher score representing a worse quality of life.	up to week 36
ABQoL Scores Over Time	Autoimmune Bullous Disease Quality of Life Index (ABQoL) questionnaire assesses the impact of Autoimmune Bullous Disease and their therapies on the daily life of patients. Scores range from 0 to 51 with a higher score representing a worse quality of life.	up to week 36
Efgartigimod Serum Concentrations		up to week 36
Percent Change of Total IgG Serum Levels From Baseline Over Time		up to 36 weeks
Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time		up to 36 weeks
Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20		up to 46 weeks
Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC		up to week 32

Collaborators and Investigators

• Sponsor: argenx

Study record dates

Study Major Dates

• Study Start (Actual): June 9, 2022
• Primary Completion (Actual): September 13, 2024
• Study Completion (Actual): September 13, 2024

Study Registration Dates

• First Submitted: February 14, 2022
• First Submitted That Met QC Criteria: February 23, 2022
• First Posted (Actual): March 4, 2022

Study Record Updates

• Last Update Posted (Estimated): October 23, 2025
• Last Update Submitted That Met QC Criteria: October 8, 2025
• Last Verified: September 1, 2025

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-113-2009

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.: No