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) (ADDRESS+)

March 28, 2025 updated by: argenx

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

183

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Melbourne, Australia, 3065
        • Investigator site 92 - AU0610013
    • New South Wales
      • Sydney, New South Wales, Australia, 2217
        • Investigator site 15 - AU0610006
    • Victoria
      • Parkville, Victoria, Australia, 3050
        • Investigator site 11 - AU0610007
  • Bulgaria
      • Pleven, Bulgaria, 5800
        • Investigator site 17 - BG3590012
      • Plovdiv, Bulgaria, 4000
        • Investigator site 18 - BG3590013
      • Sofia, Bulgaria, 1431
        • Investigator site 2 - BG3590010
      • Sofia, Bulgaria, 1510
        • Investigator site 16 - BG3590009
      • Sofia, Bulgaria, 1606
        • Investigator site 3 - BG3590011
  • China
      • Beijing, China, 100034
        • Investigator site 101 - CN0860017
      • Chengdu, China, 610000
        • Investigator site 107 - CN0860018
      • Chongqing, China, 400042
        • Investigator site 118 - CH0860027
      • Fuzhou, China, 35005
        • Investigator site 120 - CH0860023
      • Guangzhou, China, 510000
        • Investigator site 119 - CH0860022
      • Guangzhou, China, 51000
        • Investigator site 116 - CH0860053
      • Guanzhou, China, 510000
        • Investigator site 100 - CN0860021
      • Nanjing, China, 210042
        • Investigator site 113 - CN0860024
      • Shanghai, China, 200025
        • Investigator site 102 - CN0860020
      • Shanghai, China, 200040
        • Investigator site 99 - CN0860016
      • Wuhan, China, 430022
        • Investigator site 112 - CN0860019
      • Wuhan, China, 430022
        • Investigator site 121 - CH0860025
      • Zhengzhou, China, 450008
        • Investigator site 117 - CH0860026
  • France
      • Bobigny, France, 93000
        • Investigator site 77 - FR0330028
      • La Tronche, France, 38700
        • Investigator site 60 - FR0330027
      • Rouen, France, 76031
        • Investigator site 108 - FR0330029
      • Saint-Étienne, France, 42055
        • Investigator site 51 - FR0330026
  • Georgia
      • Tbilisi, Georgia, 0159
        • Investigator site 78 - GE9950014
      • Tbilisi, Georgia, 0160
        • Investigator site 127 - GE9950030
      • Tbilisi, Georgia, 0162
        • Investigator site 32 - GE9950013
      • Tbilisi, Georgia, 0179
        • Investigator site 31 - GE9950015
  • Germany
      • Berlin, Germany, 10117
        • Investigator site 45 - DE0490029
      • Dresden, Germany, 01307
        • Investigator site 34 - DE0490030
      • Frankfurt am main, Germany, 60590
        • Investigator site 33 - DE0490024
      • Freiburg, Germany, 79104
        • Investigator site 53 - DE0490023
      • Kiel, Germany, 24105
        • Investigator site 35 - DE0490028
      • Lübeck, Germany, 23538
        • Investigator site 20 - DE0490002
      • Marburg, Germany, 35043
        • Investigator site 52 - DE0490001
      • Tübingen, Germany, 72076
        • Investigator site 19 - DE0490025
      • Ulm, Germany, 89081
        • Investigator site 93 - DE0490027
      • Würzburg, Germany, 97080
        • Investigator site 4 - DE0490026
  • Greece
      • Athens, Greece, 11525
        • Investigator site 21 - GR030004
      • Athens, Greece, 16121
        • Investigator site 37 - GR030006
      • Athens, Greece, 16121
        • Investigator site 54 - GR0300001
      • Chaïdári, Greece, 12462
        • Investigator site 22 - GR030003
      • Thessaloníki, Greece, 54643
        • Investigator site 36 - GR0300002
      • Thessaloníki, Greece, 56429
        • Investigator site 23 - GR030005
  • Hungary
      • Debrecen, Hungary, 4032
        • Investigator site 6 - HU0360003
      • Pécs, Hungary, 7632
        • Investigator site 5 - HU0360001
      • Szeged, Hungary, 6720
        • Investigator site 24 - HU0360002
  • India
      • Ahmedabad, India, 380016
        • Investigator site 65 - IN0910002
      • Chandigarh, India, 160012
        • Investigator site 94 - IN0910001
      • Lucknow, India, 226005
        • Investigator site 79 - IN0910004
      • Nagpur, India, 440003
        • Investigator site 80 - IN0910003
  • Israel
      • Tel Aviv, Israel, 64239
        • Investigator site 85 - IL9720002
  • Italy
      • Catania, Italy, 95123
        • Investigator site 95 - IT0390039
      • Firenze, Italy, 50125
        • Investigator site 38 - IT0390031
      • Genova, Italy, 16132
        • Investigator site 81 - IT0390030
      • Perugia, Italy, 06129
        • Investigator site 55 - IT0390038
      • Roma, Italy, 00167
        • Investigator site 12 - IT0390006
      • Roma, Italy, 00168
        • Investigator site 25 - IT-0390005
      • Siena, Italy, 53100
        • Investigator site 61 - IT0390040
  • Japan
      • Aichi, Japan, 480-1195
        • Investigator site 82 - JP0810046
      • Hiroshima, Japan, 734-8551
        • Investigator site 68 - JP0810040
      • Kurume, Japan, 830-001
        • Investigator site 69 - JP0810050
      • Kōfu, Japan, 400-8506
        • Investigator site 66 - JP0810042
      • Okayama, Japan, 700-8558
        • Investigator site 73 - JP0810047
      • Okayama, Japan, 701-0192
        • Investigator site 70 - JP0810041
      • Osaka, Japan, 545-8586
        • Investigator site 71 - JP0810049
      • Sapporo, Japan, 060-8648
        • Investigator site 72 - JP0810045
      • Sendai, Japan, 980-8574
        • Investigator site 114 - JP0810067
      • Tokyo, Japan, 113-8431
        • Investigator site 67 - JP0810043
  • Poland
      • Katowice, Poland, 40-081
        • Investigator site 27 - PL0480027
      • Poznań, Poland, 60-369
        • Investigator site 86 - PL0480036
      • Rzeszów, Poland, 35-055
        • Investigator site 28 - PL0480025
      • Wrocław, Poland, 50-566
        • Investigator site 26 - PL0480028
      • Łódź, Poland, 90-647
        • Investigator site 56 - PL0480032
  • Romania
      • Bucharest, Romania, 011216
        • Investigator site 97 - RO0400013
      • Cluj-Napoca, Romania, 400006
        • Investigator 96 - RO0400014
      • Iaşi, Romania, 700111
        • Investigator site 98 - RO0400015
  • Russian Federation
      • Chelyabinsk, Russian Federation, 454092
        • Investigator site 40 - RU0070035
      • Ekaterinburg, Russian Federation, 620076
        • Investigator site 41 - RU0070033
      • Kazan, Russian Federation, 420111
        • Investigator site 48 - RU0070029
      • Krasnodar, Russian Federation, 350020
        • Investigator site 49 - RU0070030
      • Rostov-on-Don, Russian Federation, 344002
        • Investigator site 39 - RU0070032
      • Saint Petersburg, Russian Federation, 191123
        • Investigator site 46 - RU0070031
      • Saint Petersburg, Russian Federation, 197022
        • Investigator site 50 - RU0070034
      • Saratov, Russian Federation, 410012/410028
        • Investigator site 47 - RU0070028
  • Serbia
      • Belgrade, Serbia, 11000
        • Investigator site 109 - RS3810011
      • Belgrade, Serbia, 11000
        • Investigator site 110 - RS3810010
      • Niš, Serbia, 18000
        • Investigator site 105 - RS3810012
      • Novi Sad, Serbia, 21000
        • Investigator site 104 - RS3810009
  • Spain
      • Barcelona, Spain, 08907
        • Investigator site 62 - ES0340026
      • Barcelona, Spain, 8036
        • Investigator site 13 - ES0340032
      • Granada, Spain, 18016
        • Investigator site 122 - ES0340053
      • Madrid, Spain, 28007
        • Investigator site 59 - ES0340034
      • Madrid, Spain, 28034
        • Investigator site 42 - ES0340025
      • Madrid, Spain, 28041
        • Investigator site 7 - ES0340029
      • Madrid, Spain, 28046
        • Investigator site 57 - ES0340027
      • Sevilla, Spain, 41013
        • Investigator site 58 - ES0340028
  • Turkey
      • Gaziantep, Turkey, 27310
        • Investigator site 64 - TR0900020
      • Istanbul, Turkey, 34098
        • Investigator site 63 - TR0900012
      • Istanbul, Turkey, 34722
        • Investigator site 74 - TR0900011
  • Ukraine
      • Dnipro, Ukraine, 49074
        • Investigator site 75 - UA3800017
      • Ivano-Frankivs'k, Ukraine, 76018
        • Investigator site 29 - UA3800023
      • Kyiv, Ukraine, 04050
        • Investigator site 14 - UA3800020
      • Kyiv, Ukraine, 4209
        • Investigator site 8 - UA3800019
      • Lviv, Ukraine, 79013
        • Investigator site 43 - UA3800021
      • Zaporizhzhia, Ukraine, 69063
        • Investigator site 9 - UA3800018
  • United Kingdom
      • Birmingham, United Kingdom, B15 2GW
        • Investigator site 106 - UK0440021
      • Bristol, United Kingdom, BS2 8HW
        • Investigator site 83 - UK0440022
      • Southampton, United Kingdom, SO16 6YD
        • Investigator site 123 - UK0440037
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Investigator site 115 - US0010086
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Investigator site 89 - US0010091
    • California
      • Redwood City, California, United States, 94063
        • Investigator site 124 - US0010092
    • Florida
      • Boca Raton, Florida, United States, 33428
        • Investigator site 1 - US0010087
      • Miami, Florida, United States, 33173
        • Investigator site 90 - US0010117
      • Orlando, Florida, United States, 32827
        • Investigator site 91 - US0010109
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Investigator site 126 - US0010090
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Investigator site 111 - US0010098
    • New York
      • Buffalo, New York, United States, 14203-1070
        • Investigator site 10 - US0010088
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Investigator site 76 - US0010096
    • Ohio
      • Cleveland, Ohio, United States, 44106-1716
        • Investigator site 30 - US0010094
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Investigator site 84 - US0010089
      • Philadelphia, Pennsylvania, United States, 19140
        • Investigator site 103 - US0010097
    • Texas
      • Dallas, Texas, United States, 75246
        • Investigator site 125 - US0010107
      • Dripping Springs, Texas, United States, 78620
        • Investigator site 87 - US0010084
      • Houston, Texas, United States, 77008
        • Investigator site 88 - US0010114
    • Virginia
      • Norfolk, Virginia, United States, 23502
        • Investigator site 44 - US0010106

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. 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).
  2. The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.

  3. Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating in clinical trials and:

    1. 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.

    2. Female participants

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

Exclusion Criteria:

  1. Pregnant and lactating women and those intending to become pregnant during the trial.
  2. 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.
  3. Known hypersensitivity to any of the components of the administered treatments.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: efgartigimod PH20 SC
patients receiving efgartigimod PH20 SC on top of prednisone
Biological: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
Drug: prednisone
Oral prednisone tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
Time Frame: Up to 60 weeks
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).
Up to 60 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin
Time Frame: Up to 60 weeks
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.
Up to 60 weeks
Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin
Time Frame: Up to 60 weeks
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.
Up to 60 weeks
Time to DC in Participants With PV and PF
Time Frame: Up to 52 weeks
Disease Control (DC) defined as absence of new lesions and the start of healing of established lesions
Up to 52 weeks
Time to CR in Participants With PV and PF
Time Frame: Up to 52 weeks
CR (Complete clinical remission) defined as the absence of new lesions and complete healing of established lesions
Up to 52 weeks
Time to CRmin in Participants With PV and PF
Time Frame: Up to 52 weeks
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.
Up to 52 weeks
Time to CRoff in Participants With PV and PF
Time Frame: Up to 52 weeks
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.
Up to 52 weeks
Time to Flare After CRmin in Participants With PV and PF
Time Frame: Up to 52 weeks
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.
Up to 52 weeks
Rate of Treatment Failure in Participants With PV and PF
Time Frame: Up to 52 weeks
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.
Up to 52 weeks
Number of Flares in Participants With PV and PF
Time Frame: Up to 60 weeks
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.
Up to 60 weeks
Normalized Cumulative Prednisone Dose in Participants With PV and PF
Time Frame: Up to 60 weeks
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
Up to 60 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

argenx

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 15, 2021

Primary Completion (Actual)

March 25, 2024

Study Completion (Actual)

March 25, 2024

Study Registration Dates

First Submitted

October 8, 2020

First Submitted That Met QC Criteria

October 16, 2020

First Posted (Actual)

October 22, 2020

Study Record Updates

Last Update Posted (Actual)

March 30, 2025

Last Update Submitted That Met QC Criteria

March 28, 2025

Last Verified

March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ARGX-113-1905

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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