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TerminatedNCT05681481BALLAD+Updated Feb 25, 2026Results posted

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

A Phase 3 interventional study of efgartigimod PH20 SC and Prednisone in Bullous Pemphigoid, sponsored by argenx. Terminated at 40 sites in 17 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-02-25.

Sponsored by argenx · Phase 3, Interventional, and Treatment

Why this study was terminated
Sponsor decision
Phase
Phase 3
Study type
Interventional
Enrollment
64
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Bullous Pemphigoid

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03

In context

Pemphigoid, Bullous

73 studies on the registry are indexed under Pemphigoid, Bullous; 11 are open to participants now.

This study's enrollment of 64 is above the median of 29 across 43 interventional studies indexed under Pemphigoid, Bullous.

Browse Pemphigoid, Bullous studies →

Lead sponsor

argenx is the lead sponsor of 87 studies on the registry; 33 are open to participants now.

Of its 22 completed or terminated interventional studies of FDA-regulated products, 16 (73%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
64 participants (actual)

Study arms

  • Experimental
    efgartigimod PH20 SC

    participants receiving efgartigimod PH20 SC on top of Prednisone

    Biological: efgartigimod PH20 SC · Drug: Prednisone

Interventions

  • Biologicalefgartigimod PH20 SC

    Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

  • DrugPrednisone

    Oral Prednisone

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: Up to 56 weeks

  2. Number of Participants Who Discontinued Treatment Because of Safety Concerns

    Time frame: Up to 56 weeks

Secondary outcomes

  1. 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.

    Time frame: Up to 56 weeks

  2. 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.

    Time frame: Up to 56 weeks

  3. 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)

    Time frame: Up to 56 weeks

  4. 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

    Time frame: Up to 56 weeks

  5. 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.

    Time frame: Up to 56 weeks

  6. 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.

    Time frame: Up to 56 weeks

  7. 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

    Time frame: Up to 56 weeks

  8. 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

    Time frame: Up to 56 weeks

  9. 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.

    Time frame: Up to 56 weeks

  10. 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).

    Time frame: Up to 56 weeks

  11. 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)

    Time frame: Up to 56 weeks

  12. 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)

    Time frame: Up to 56 weeks

07

Results

Posted Feb 25, 2026

Participant flow

This study was conducted at 38 sites that enrolled participants in 17 countries. On 13 Jan 2025 the sponsor terminated the study early due to a lack of efficacy of efgartigimod when administered with concomitant OCS in the antecedent study (ARGX-113-2009).

Participant flow — Overall Study
MilestoneEfgartigimod PH20 SC
Started64
Completed17
Not completed47
Withdrew: Adverse event5
Withdrew: Death1
Withdrew: Lost to follow-up2
Withdrew: Participant-specified withdrawal criterion met1
Withdrew: Physician decision3
Withdrew: Study terminated by sponsor26
Withdrew: Withdrawal by subject7
Withdrew: Withdrawal of consent2

Outcome measures

PrimaryNumber 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.

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants With Treatment-emergent AEs, SAEs and AESIs
ParticipantsEfgartigimod PH20 SC
AE40
SAE13
AESI25
PrimaryNumber of Participants Who Discontinued Treatment Because of Safety Concerns
Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Who Discontinued Treatment Because of Safety Concerns
ParticipantsEfgartigimod PH20 SC
Number of Participants Who Discontinued Treatment Because of Safety Concerns4
SecondaryNumber 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.

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Achieving CRoff for ≥ 8 Weeks
ParticipantsEfgartigimod PH20 SC
Number of Participants Achieving CRoff for ≥ 8 Weeks3
SecondaryNumber 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.

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Achieving CRoff or PRoff for ≥ 8 Weeks
ParticipantsEfgartigimod PH20 SC
Number of Participants Achieving CRoff or PRoff for ≥ 8 Weeks3
SecondaryNumber 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)

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Achieving CRmin for ≥ 8 Weeks
ParticipantsEfgartigimod PH20 SC
Number of Participants Achieving CRmin for ≥ 8 Weeks4
SecondaryNumber 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

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Achieving Complete Remission While Off Both Oral Corticosteroids and Efgartigimod PH20 SC for ≥ 8 Weeks
ParticipantsEfgartigimod PH20 SC
Number of Participants Achieving Complete Remission While Off Both Oral Corticosteroids and Efgartigimod PH20 SC for ≥ 8 Weeks10
SecondaryNumber 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.

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Achieving CR or PR While Off Both OCS and Efgartigimod PH20 SC for ≥ 8 Weeks
ParticipantsEfgartigimod PH20 SC
Number of Participants Achieving CR or PR While Off Both OCS and Efgartigimod PH20 SC for ≥ 8 Weeks10
SecondaryDuration 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.

Time frame:
Up to 56 weeks
Reported as:
Median · days
Duration of Sustained Remission
daysEfgartigimod PH20 SC
Duration of Sustained RemissionNA (54.0 to —)
SecondaryNumber 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

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Who Relapsed
ParticipantsEfgartigimod PH20 SC
Number of Participants Who Relapsed37
SecondaryTime 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

Time frame:
Up to 56 weeks
Reported as:
Median · days
Time to Relapse
daysEfgartigimod PH20 SC
Time to Relapse169.0 (108.0 to 301.0)
SecondaryBPDAI 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.

Time frame:
Up to 56 weeks
Reported as:
Mean · Percent change
BPDAI Activity Score, Percent Change From Baseline to Last Assessment
Percent changeEfgartigimod PH20 SC
BPDAI Activity Score, Percent Change From Baseline to Last Assessment0.15 ± 118.361
SecondaryIGA-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).

Time frame:
Up to 56 weeks
Reported as:
Number · Participants
IGA-BP Score at Last Assessment
ParticipantsEfgartigimod PH20 SC
0 - Clear28
1 - Almost clear8
2 - Mild19
3 - Moderate4
4 - Severe4
SecondaryItch 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)

Time frame:
Up to 56 weeks
Reported as:
Mean · score on a scale
Itch NRS 24-hour Average Score, Change From Baseline to Last Assessment
score on a scaleEfgartigimod PH20 SC
Itch NRS 24-hour Average Score, Change From Baseline to Last Assessment0.3 ± 3.07
SecondaryNumber 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)

Time frame:
Up to 56 weeks
Reported as:
Count of participants · Participants
Number of Participants Who Failed Treatment
ParticipantsEfgartigimod PH20 SC
Number of Participants Who Failed Treatment0

Adverse events

Collected over Up to 56 weeks. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Efgartigimod PH20 SC1/50 (2%)13/50 (26%)38/50 (76%)
Most frequent serious events
Showing 10 of 19
Most frequent serious events
EventEfgartigimod PH20 SC
AnaemiaBlood and lymphatic system disorders1/50
Cardiac failureCardiac disorders1/50
Abdominal distensionGastrointestinal disorders1/50
Condition aggravatedGeneral disorders1/50
General physical health deteriorationGeneral disorders1/50
Oedema peripheralGeneral disorders1/50
AppendicitisInfections and infestations1/50
Gastroenteritis viralInfections and infestations1/50
Herpes zosterInfections and infestations1/50
PneumoniaInfections and infestations1/50
Most frequent other events
Most frequent other events
EventEfgartigimod PH20 SC
ArthralgiaMusculoskeletal and connective tissue disorders5/50
Back painMusculoskeletal and connective tissue disorders5/50
Injection site paraesthesiaGeneral disorders4/50
Oedema peripheralGeneral disorders4/50
NasopharyngitisInfections and infestations4/50
Urinary tract infectionInfections and infestations4/50
DiarrhoeaGastrointestinal disorders3/50
NauseaGastrointestinal disorders3/50
InsomniaPsychiatric disorders3/50
PruritusSkin and subcutaneous tissue disorders3/50

Baseline characteristics

Age, Continuous
Age, Continuous(years)Efgartigimod PH20 SC
Mean70.6 ± 10.17
Sex: Female, Male
Sex: Female, Male(Participants)Efgartigimod PH20 SC
Female48
Male16
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Efgartigimod PH20 SC
Asian10
White52
Other2
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Efgartigimod PH20 SC
Hispanic or Latino1
Not Hispanic or Latino63
08

Study locations

40 sites
  • Medical Dermatology Specialists
    Phoenix, Arizona 85006, United States
  • First OC Dermatology
    Fountain Valley, California 92708, United States
  • Miami Dermatology and Laser Institute
    Miami, Florida 33173, United States
  • University of Michigan Hospital
    Ann Arbor, Michigan 48109, United States
  • Saint Louis University
    St Louis, Missouri 63110, United States
  • Wright State Physicians
    Fairborn, Ohio 45324, United States
  • Premier Specialists
    Kogarah, 2217, Australia
  • Diagnostic and Consulting Center Aleksandrovska EOOD
    Sofia, 1431, Bulgaria
  • West China Hospital of Sichuan University
    Chengdu, 610041, China
  • The First Affiliated Hospital of Chongqing Medical University
    Chongqing, 400016, China
  • Ruijin Hospital Shanghai Jiaotong University School of Medicine
    Shanghai, 200025, China
  • Poliklinika Solmed
    Zagreb, 10000, Croatia
  • Fakultni nemocnice Bulovka
    Prague, 180 00, Czechia
  • Charité - Universitätsmedizin Berlin
    Berlin, 10117, Germany
  • Universitätsklinikum Carl Gustav Carus an der TU Dresden
    Dresden, 01307, Germany
  • Universitatsklinikum Dusseldorf
    Düsseldorf, 40225, Germany
  • Universitatsklinikum Schleswig-Holstein
    Kiel, 24105, Germany
  • LMU Klinikum der Universität
    München, 80337, Germany
  • Universitätsklinikum Würzburg
    Würzburg, 97080, Germany
  • Hospital of Venereal and Skin Diseases A.Syggros
    Athens, 16121, Greece
  • Hospital Of Skin And Venereal Diseases of Thessaloniki
    Thessaloniki, 54643, Greece
  • Semmelweis Egyetem
    Budapest, 1085, Hungary
  • Sheba Medical Center - PPDS
    Ramat Gan, 5262100, Israel
  • Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
    Catania, 95123, Italy
  • Azienda USL Toscana Centro - Ospidale Piero Palagi
    Florence, 50122, Italy
  • Azienda Sanitaria Di Firenze
    Florence, 50125, Italy
  • Ospedale Policlinico San Martino
    Genova, 16132, Italy
  • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
    Milan, 20122, Italy
  • Fondazione IRCCS Policlinico San Matteo di Pavia
    Pavia, 27100, Italy
  • IDI IRCCS - Istituto Dermopatico dell'Immacolata
    Roma, 00167, Italy
  • Fondazione Policlinico Universitario A. Gemelli
    Rome, 00168, Italy
  • Hokkaido University Hospital
    Sapporo, 060-8648, Japan
  • Universitair Medisch Centrum Groningen
    Groningen, 9713 GZ, Netherlands
  • University Clinical Center of Serbia - PPDS
    Belgrade, 11000, Serbia
  • Univerzitna nemocnica Bratislava
    Bratislava, 821 06, Slovakia
  • Fakultna nemocnica Trnava
    Trnava, 91702, Slovakia
  • Hospital Universitario Clínico San Cecilio
    Granada, 18016, Spain
  • Hospital Universitario 12 de Octubre
    Madrid, 28041, Spain
  • Hospital Universitario Doctor Peset
    Valencia, 46017, Spain
  • Guy's and St Thomas' NHS Foundation Trust
    London, SE1 9RT, United Kingdom
09

References and documents

Study documents

  • Study protocol · May 2, 2024
  • Statistical analysis plan · Apr 29, 2025

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 25, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05681481
Lead sponsor
argenx
Responsible party
Sponsor
First posted
Jan 12, 2023
Start date
Mar 22, 2023
Primary completion
Mar 20, 2025
Completion
Mar 20, 2025
Results posted
Feb 25, 2026
Last update
Feb 25, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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