Bullous PemphigoidPhase 3BALLAD+NCT05681481
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 study testing efgartigimod PH20 SC and Prednisone in people with Bullous Pemphigoid, run by argenx at 40 sites in 17 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bullous Pemphigoid
- What is being tested?
- efgartigimod PH20 SC and Prednisone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 40 sitesin 17 countries
- Study dates
- 22 Mar 2023to 20 Mar 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
40 sites in 17 countries.
40 sites in 17 countries
Italy8 sites
Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele
Catania, 95123
Azienda USL Toscana Centro - Ospidale Piero Palagi
Florence, 50122
Azienda Sanitaria Di Firenze
Florence, 50125
Ospedale Policlinico San Martino
Genova, 16132
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, 20122
Fondazione IRCCS Policlinico San Matteo di Pavia
Pavia, 27100
IDI IRCCS - Istituto Dermopatico dell'Immacolata
Roma, 00167
Fondazione Policlinico Universitario A. Gemelli
Rome, 00168
United States6 sites
University of Michigan Hospital
Ann Arbor, Michigan, 48109
Wright State Physicians
Fairborn, Ohio, 45324
First OC Dermatology
Fountain Valley, California, 92708
Miami Dermatology and Laser Institute
Miami, Florida, 33173
Medical Dermatology Specialists
Phoenix, Arizona, 85006
Saint Louis University
St Louis, Missouri, 63110
Germany6 sites
Charité - Universitätsmedizin Berlin
Berlin, 10117
Universitätsklinikum Carl Gustav Carus an der TU Dresden
Dresden, 01307
Universitatsklinikum Dusseldorf
Düsseldorf, 40225
Universitatsklinikum Schleswig-Holstein
Kiel, 24105
LMU Klinikum der Universität
München, 80337
Universitätsklinikum Würzburg
Würzburg, 97080
China3 sites
West China Hospital of Sichuan University
Chengdu, 610041
The First Affiliated Hospital of Chongqing Medical University
Chongqing, 400016
Ruijin Hospital Shanghai Jiaotong University School of Medicine
Shanghai, 200025
Spain3 sites
Hospital Universitario Clínico San Cecilio
Granada, 18016
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitario Doctor Peset
Valencia, 46017
Greece2 sites
Hospital of Venereal and Skin Diseases A.Syggros
Athens, 16121
Hospital Of Skin And Venereal Diseases of Thessaloniki
Thessaloniki, 54643
Slovakia2 sites
Univerzitna nemocnica Bratislava
Bratislava, 821 06
Fakultna nemocnica Trnava
Trnava, 91702
Bulgaria1 site
Diagnostic and Consulting Center Aleksandrovska EOOD
Sofia, 1431
Netherlands1 site
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ
United Kingdom1 site
Guy's and St Thomas' NHS Foundation Trust
London, SE1 9RT
About this study
Official title: 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.
- Conditions
- Bullous Pemphigoid
- Being tested
- efgartigimod PH20 SC, Prednisone
- Medical subject headings
- Pemphigoid, Bullous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efgartigimod PH20 SCBiological
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer
PrednisoneDrug
Oral Prednisone
| Study group | Role | What they receive |
|---|---|---|
| efgartigimod PH20 SC | Experimental | participants receiving efgartigimod PH20 SC on top of Prednisone Receives: Biological: efgartigimod PH20 SC; Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 56 weeks
Number of Participants Who Discontinued Treatment Because of Safety Concerns
Measured: Up to 56 weeks
Secondary outcomes (12)
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.
Measured: 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.
Measured: 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)
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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.
Measured: 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).
Measured: 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)
Measured: 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)
Measured: Up to 56 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2023
- Study start22 Mar 2023
- Primary completion20 Mar 2025
- Study completion20 Mar 2025
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Bullous PemphigoidStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05681481
- Sponsor's protocol ID
- ARGX-113-2010
- Other IDs
- 2024-515832-59-00
- Acronym
- BALLAD+
- Status last verified
- Feb 2026
- First submitted
- 8 Dec 2022
- Last updated
- 25 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05681481
- Acronym / sponsor ID
- BALLAD+ / ARGX-113-2010
- Conditions
- Bullous Pemphigoid
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 64 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 22 Mar 2023 (actual)
- Primary completion
- 20 Mar 2025 (actual)
- Study completion
- 20 Mar 2025 (actual)
- First posted
- 12 Jan 2023 (actual)
- Results
- Posted 25 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| efgartigimod PH20 SC | Experimental | Biological: efgartigimod PH20 SC, Drug: Prednisoneparticipants receiving efgartigimod PH20 SC on top of Prednisone |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| efgartigimod PH20 SCSubcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer | Biological | efgartigimod PH20 SC |
| PrednisoneOral Prednisone | Drug | efgartigimod PH20 SC |
Primary outcome
Number of Participants With Treatment-emergent AEs, SAEs and AESIs
Time frame: Up to 56 weeks
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.
Number of Participants Who Discontinued Treatment Because of Safety Concerns
Time frame: Up to 56 weeks
12 secondary outcomes
Number of Participants Achieving CRoff for ≥ 8 Weeks
Time frame: Up to 56 weeks
Number of Participants Achieving CRoff or PRoff for ≥ 8 Weeks
Time frame: Up to 56 weeks
Number of Participants Achieving CRmin for ≥ 8 Weeks
Time frame: Up to 56 weeks
Number of Participants Achieving Complete Remission While Off Both Oral Corticosteroids and Efgartigimod PH20 SC for ≥ 8 Weeks
Time frame: Up to 56 weeks
Number of Participants Achieving CR or PR While Off Both OCS and Efgartigimod PH20 SC for ≥ 8 Weeks
Time frame: Up to 56 weeks
Duration of Sustained Remission
Time frame: Up to 56 weeks
Number of Participants Who Relapsed
Time frame: Up to 56 weeks
Time to Relapse
Time frame: Up to 56 weeks
BPDAI Activity Score, Percent Change From Baseline to Last Assessment
Time frame: Up to 56 weeks
IGA-BP Score at Last Assessment
Time frame: Up to 56 weeks
Itch NRS 24-hour Average Score, Change From Baseline to Last Assessment
Time frame: Up to 56 weeks
Number of Participants Who Failed Treatment
Time frame: Up to 56 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
40 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing efgartigimod PH20 SC and Prednisone in people with Bullous Pemphigoid, run by argenx at 40 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in Italy, United States, Germany, China, Spain and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent AEs, SAEs and AESIs (time frame: Up to 56 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2026.