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Pemphigus VulgarisPhase 3ADDRESS+NCT04598477

A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)

A Phase 3 study testing efgartigimod PH20 SC and prednisone in people with Pemphigus Vulgaris and Pemphigus Foliaceus, run by argenx at 127 sites in 21 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This was a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pemphigus Vulgaris, Pemphigus Foliaceus
What is being tested?
efgartigimod PH20 SC and prednisone
Could I receive a placebo?
No, everyone receives an active treatment
How many people
183 participants
Where
127 sitesin 21 countries
Study dates
15 Jul 2021to 25 Mar 2024
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

  • Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits).
  • The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
  • Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating in clinical trials and:
  • Male participants:
  • Male participants must agree to use an acceptable method of contraception as described in the protocol, from signing the ICF until the last dose of the study drug.
  • Female participants
Show all 9Show fewer
  • Women of childbearing potential (WOCBP) must:
  • have a negative urine pregnancy test at baseline before the IMP can be administered,
  • agree to use a highly effective or acceptable contraception method (as described in the protocol), which should be maintained at minimum until after the last dose of IMP

Who cannot join

  • Pregnant and lactating women and those intending to become pregnant during the trial.
  • Participants with clinical evidence of other significant serious disease or participants who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk.
  • Known hypersensitivity to any of the components of the administered treatments.

Trial locations and contacts

127 sites in 21 countries.

127 sites in 21 countries

United States17 sites

  • Investigator site 115 - US0010086

    Birmingham, Alabama, 35233

  • Investigator site 1 - US0010087

    Boca Raton, Florida, 33428

  • Investigator site 10 - US0010088

    Buffalo, New York, 14203-1070

  • Investigator site 30 - US0010094

    Cleveland, Ohio, 44106-1716

  • Investigator site 125 - US0010107

    Dallas, Texas, 75246

  • Investigator site 87 - US0010084

    Dripping Springs, Texas, 78620

  • Investigator site 76 - US0010096

    Durham, North Carolina, 27710

  • Investigator site 88 - US0010114

    Houston, Texas, 77008

  • Investigator site 90 - US0010117

    Miami, Florida, 33173

  • Investigator site 126 - US0010090

    Minneapolis, Minnesota, 55455

  • Investigator site 44 - US0010106

    Norfolk, Virginia, 23502

  • Investigator site 91 - US0010109

    Orlando, Florida, 32827

  • Investigator site 84 - US0010089

    Philadelphia, Pennsylvania, 19104

  • Investigator site 103 - US0010097

    Philadelphia, Pennsylvania, 19140

  • Investigator site 124 - US0010092

    Redwood City, California, 94063

  • Investigator site 89 - US0010091

    Scottsdale, Arizona, 85259

  • Investigator site 111 - US0010098

    St Louis, Missouri, 63110

Other trials in United States

China13 sites

  • Investigator site 101 - CN0860017

    Beijing, 100034

  • Investigator site 107 - CN0860018

    Chengdu, 610000

  • Investigator site 118 - CH0860027

    Chongqing, 400042

  • Investigator site 120 - CH0860023

    Fuzhou, 35005

  • Investigator site 119 - CH0860022

    Guangzhou, 510000

  • Investigator site 116 - CH0860053

    Guangzhou, 51000

  • Investigator site 100 - CN0860021

    Guanzhou, 510000

  • Investigator site 113 - CN0860024

    Nanjing, 210042

  • Investigator site 102 - CN0860020

    Shanghai, 200025

  • Investigator site 99 - CN0860016

    Shanghai, 200040

  • Investigator site 112 - CN0860019

    Wuhan, 430022

  • Investigator site 121 - CH0860025

    Wuhan, 430022

  • Investigator site 117 - CH0860026

    Zhengzhou, 450008

Other trials in China

Germany10 sites

  • Investigator site 45 - DE0490029

    Berlin, 10117

  • Investigator site 34 - DE0490030

    Dresden, 01307

  • Investigator site 33 - DE0490024

    Frankfurt am Main, 60590

  • Investigator site 53 - DE0490023

    Freiburg im Breisgau, 79104

  • Investigator site 35 - DE0490028

    Kiel, 24105

  • Investigator site 20 - DE0490002

    Lübeck, 23538

  • Investigator site 52 - DE0490001

    Marburg, 35043

  • Investigator site 19 - DE0490025

    Tübingen, 72076

  • Investigator site 93 - DE0490027

    Ulm, 89081

  • Investigator site 4 - DE0490026

    Würzburg, 97080

Other trials in Germany

Japan10 sites

  • Investigator site 82 - JP0810046

    Aichi, 480-1195

  • Investigator site 68 - JP0810040

    Hiroshima, 734-8551

  • Investigator site 66 - JP0810042

    Kofu, 400-8506

  • Investigator site 69 - JP0810050

    Kurume, 830-001

  • Investigator site 73 - JP0810047

    Okayama, 700-8558

  • Investigator site 70 - JP0810041

    Okayama, 701-0192

  • Investigator site 71 - JP0810049

    Osaka, 545-8586

  • Investigator site 72 - JP0810045

    Sapporo, 060-8648

  • Investigator site 114 - JP0810067

    Sendai, 980-8574

  • Investigator site 67 - JP0810043

    Tokyo, 113-8431

Other trials in Japan

Russia8 sites

  • Investigator site 40 - RU0070035

    Chelyabinsk, 454092

  • Investigator site 48 - RU0070029

    Kazan', 420111

  • Investigator site 49 - RU0070030

    Krasnodar, 350020

  • Investigator site 39 - RU0070032

    Rostov-on-Don, 344002

  • Investigator site 46 - RU0070031

    Saint Petersburg, 191123

  • Investigator site 50 - RU0070034

    Saint Petersburg, 197022

  • Investigator site 47 - RU0070028

    Saratov, 410012/410028

  • Investigator site 41 - RU0070033

    Yekaterinburg, 620076

Other trials in Russia

Spain8 sites

  • Investigator site 62 - ES0340026

    Barcelona, 08907

  • Investigator site 13 - ES0340032

    Barcelona, 8036

  • Investigator site 122 - ES0340053

    Granada, 18016

  • Investigator site 59 - ES0340034

    Madrid, 28007

  • Investigator site 42 - ES0340025

    Madrid, 28034

  • Investigator site 7 - ES0340029

    Madrid, 28041

  • Investigator site 57 - ES0340027

    Madrid, 28046

  • Investigator site 58 - ES0340028

    Seville, 41013

Other trials in Spain

Italy7 sites

  • Investigator site 95 - IT0390039

    Catania, 95123

  • Investigator site 38 - IT0390031

    Florence, 50125

  • Investigator site 81 - IT0390030

    Genova, 16132

  • Investigator site 55 - IT0390038

    Perugia, 06129

  • Investigator site 12 - IT0390006

    Roma, 00167

  • Investigator site 25 - IT-0390005

    Roma, 00168

  • Investigator site 61 - IT0390040

    Siena, 53100

Other trials in Italy

Greece6 sites

  • Investigator site 21 - GR030004

    Athens, 11525

  • Investigator site 37 - GR030006

    Athens, 16121

  • Investigator site 54 - GR0300001

    Athens, 16121

  • Investigator site 22 - GR030003

    Chaïdári, 12462

  • Investigator site 36 - GR0300002

    Thessaloniki, 54643

  • Investigator site 23 - GR030005

    Thessaloniki, 56429

Other trials in Greece

Ukraine6 sites

  • Investigator site 75 - UA3800017

    Dnipro, 49074

  • Investigator site 29 - UA3800023

    Ivano-Frankivsk, 76018

  • Investigator site 14 - UA3800020

    Kyiv, 04050

  • Investigator site 8 - UA3800019

    Kyiv, 4209

  • Investigator site 43 - UA3800021

    Lviv, 79013

  • Investigator site 9 - UA3800018

    Zaporizhzhya, 69063

Other trials in Ukraine

Bulgaria5 sites

  • Investigator site 17 - BG3590012

    Pleven, 5800

  • Investigator site 18 - BG3590013

    Plovdiv, 4000

  • Investigator site 2 - BG3590010

    Sofia, 1431

  • Investigator site 16 - BG3590009

    Sofia, 1510

  • Investigator site 3 - BG3590011

    Sofia, 1606

Other trials in Bulgaria

Poland5 sites

  • Investigator site 27 - PL0480027

    Katowice, 40-081

  • Investigator site 56 - PL0480032

    Lodz, 90-647

  • Investigator site 86 - PL0480036

    Poznan, 60-369

  • Investigator site 28 - PL0480025

    Rzeszów, 35-055

  • Investigator site 26 - PL0480028

    Wroclaw, 50-566

Other trials in Poland

France4 sites

  • Investigator site 77 - FR0330028

    Bobigny, 93000

  • Investigator site 60 - FR0330027

    La Tronche, 38700

  • Investigator site 108 - FR0330029

    Rouen, 76031

  • Investigator site 51 - FR0330026

    Saint-Etienne, 42055

Other trials in France

Georgia4 sites

  • Investigator site 78 - GE9950014

    Tbilisi, 0159

  • Investigator site 127 - GE9950030

    Tbilisi, 0160

  • Investigator site 32 - GE9950013

    Tbilisi, 0162

  • Investigator site 31 - GE9950015

    Tbilisi, 0179

Other trials in Georgia

India4 sites

  • Investigator site 65 - IN0910002

    Ahmedabad, 380016

  • Investigator site 94 - IN0910001

    Chandigarh, 160012

  • Investigator site 79 - IN0910004

    Lucknow, 226005

  • Investigator site 80 - IN0910003

    Nagpur, 440003

Other trials in India

Serbia4 sites

  • Investigator site 109 - RS3810011

    Belgrade, 11000

  • Investigator site 110 - RS3810010

    Belgrade, 11000

  • Investigator site 105 - RS3810012

    Niš, 18000

  • Investigator site 104 - RS3810009

    Novi Sad, 21000

Other trials in Serbia

Australia3 sites

  • Investigator site 92 - AU0610013

    Melbourne, 3065

  • Investigator site 11 - AU0610007

    Parkville, Victoria, 3050

  • Investigator site 15 - AU0610006

    Sydney, New South Wales, 2217

Other trials in Australia

Hungary3 sites

  • Investigator site 6 - HU0360003

    Debrecen, 4032

  • Investigator site 5 - HU0360001

    Pécs, 7632

  • Investigator site 24 - HU0360002

    Szeged, 6720

Other trials in Hungary

Romania3 sites

  • Investigator site 97 - RO0400013

    Bucharest, 011216

  • Investigator 96 - RO0400014

    Cluj-Napoca, 400006

  • Investigator site 98 - RO0400015

    Iași, 700111

Other trials in Romania

Turkey (Türkiye)3 sites

  • Investigator site 64 - TR0900020

    Gaziantep, 27310

  • Investigator site 63 - TR0900012

    Istanbul, 34098

  • Investigator site 74 - TR0900011

    Istanbul, 34722

Other trials in Turkey (Türkiye)

United Kingdom3 sites

  • Investigator site 106 - UK0440021

    Birmingham, B15 2GW

  • Investigator site 83 - UK0440022

    Bristol, BS2 8HW

  • Investigator site 123 - UK0440037

    Southampton, SO16 6YD

Other trials in United Kingdom

Israel1 site

  • Investigator site 85 - IL9720002

    Tel Aviv, 64239

Other trials in Israel

About this study

Official title: An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus

This was a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provided extension of efgartigimod PH20 SC treatment and retreatment options for participants who had been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to the placebo arm in trial ARGX-113-1904. The participants could also receive concomitant prednisone therapy. Investigators could increase or decrease the prednisone dose based on protocol-specified criteria.

Trial ARGX-113-1905 evaluated the ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CR or CRmin, and the ability to treat flare; it also assessed patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial.

Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration

Being tested
efgartigimod PH20 SC, prednisone
Medical subject headings
Pemphigus

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 using rHuPH20 (PH20) as a permeation enhancer

  • prednisoneDrug

    Oral prednisone tablets

Study groups in NCT04598477
Study groupRoleWhat they receive
efgartigimod PH20 SCExperimentalpatients 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.

  1. Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)

    Incidence rates were calculated as 100 × n/PYFU. PYFU=participant-years of follow-up. The safety data sets includes participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF).

    Measured: Up to 60 weeks

Secondary outcomes (10)

  1. Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin

    CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

    Measured: Up to 60 weeks

  2. Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin

    CRmin (complete clinical remission on minimal prednisone therapy) defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

    Measured: Up to 60 weeks

  3. Time to DC in Participants With PV and PF

    Disease Control (DC) defined as absence of new lesions and the start of healing of established lesions

    Measured: Up to 52 weeks

  4. Time to CR in Participants With PV and PF

    CR (Complete clinical remission) defined as the absence of new lesions and complete healing of established lesions

    Measured: Up to 52 weeks

  5. Time to CRmin in Participants With PV and PF

    CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

    Measured: Up to 52 weeks

  6. Time to CRoff in Participants With PV and PF

    Complete remission off therapy (CRoff) is defined as the absence of new and established lesions completely healed while the patient is receiving no prednisone therapy for at least 8 weeks.

    Measured: Up to 52 weeks

  7. Time to Flare After CRmin in Participants With PV and PF

    CRmin defined as defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.

    Measured: Up to 52 weeks

  8. Rate of Treatment Failure in Participants With PV and PF

    The absence of DC with oral prednisone 1.5 mg/kg/day for a minimum of 3 weeks, or absence of DC due to prednisone-related SAE, or flare before CRmin resulting in withdrawal of the participant.

    Measured: Up to 52 weeks

  9. Number of Flares in Participants With PV and PF

    A flare is defined as the appearance of 3 or more new lesions in a 4-week period that do not heal spontaneously within 1 week or the extension, of established lesions in a participant who had achieved DC.

    Measured: Up to 60 weeks

  10. Normalized Cumulative Prednisone Dose in Participants With PV and PF

    Normalized Cumulative prednisone dose (NCPD, mg/kg/day) is the average daily intake of all weight-adjusted prednisone doses received during the study, taking into account the number of days in study

    Measured: Up to 60 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Oct 2020
  2. Study start15 Jul 2021
  3. Primary completion25 Mar 2024
  4. Study completion25 Mar 2024

Why it stopped: Based on the lack of observed efficacy in the primary study ARGX-113-1904, the sponsor decided to discontinue the open-label extension study.

Resources

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
183 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04598477
Sponsor's protocol ID
ARGX-113-1905
Acronym
ADDRESS+
Status last verified
Mar 2025
First submitted
8 Oct 2020
Last updated
30 Mar 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing efgartigimod PH20 SC and prednisone in people with Pemphigus Vulgaris and Pemphigus Foliaceus, run by argenx at 127 sites in 21 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 9 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 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 127 sites in United States, China, Germany, Japan, Russia and 16 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 30 Mar 2025). 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?

Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) (time frame: Up to 60 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04598477.

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 30 Mar 2025.

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