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

A Randomized, Double-Blinded, Placebo-Controlled Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients With Pemphigus (Vulgaris or Foliaceus)

This is a prospective, multicenter, randomized, double-blinded, placebo-controlled trial to investigate the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK, and PD of efgartigimod PH20 SC in adult participants aged from 18 years with PV or PF. The trial comprises a screening period of up to 3 weeks, a treatment period of up to 30 weeks, and an 8-week follow-up period for participants who do not enroll into the open-label extension (OLE) trial ARGX-113-1905. The primary objective of the ARGX-113-1904 trial is to demonstrate the efficacy of subcutaneous administration of efgartigimod co-formulated with recombinant human hyaluronidase PH20 (Efgartigimod PH20 SC) compared to placebo in the treatment of participants with Pemphigus Vulgaris (PV). Secondary objectives are to also demonstrate the efficacy of efgartigimod PH20 SC in the treatment of participants with Pemphigus Foliaceus (PF), and to demonstrate early onset of action and a prednisone-sparing effect. After confirmation of eligibility, participants will be randomized in a 2: 1 ratio to receive efgartigimod PH20 SC or placebo

Study Overview

• Status: Completed
• Conditions: Pemphigus Vulgaris; Pemphigus Foliaceus
• Intervention / Treatment: Other: Placebo; Biological: efgartigimod PH20 SC; Drug: prednisone

• Study Type: Interventional
• Enrollment (Actual): 222
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Sabine Coppieters, MD; Phone Number: +1 857-350-4834; Email: clinicaltrials@argenx.com

Study Locations

• Australia
  • Melbourne, Australia, 3065
    • Investigator site 103 - AU0610013
  • New South Wales
    • Sydney, New South Wales, Australia, 2217
      • Investigator site 24 - AU0610006
  • Victoria
    • Parkville, Victoria, Australia, 3050
      • Investigator site 5 - AU0610007
• Bulgaria
  • Pleven, Bulgaria, 5800
    • Investigator site 30 - BG350012
  • Plovdiv, Bulgaria, 4000
    • Investigator site 31 - BG3590013
  • Sofia, Bulgaria, 1431
    • Investigator site 4 - BG3590010
  • Sofia, Bulgaria, 1510
    • Investigator site 2 - BG3590009
  • Sofia, Bulgaria, 1606
    • Investigator site 13 - BG3590011
• China
  • Beijing, China, 100034
    • Investigator site 110 - CN0860017
  • Chendu, China, 610000
    • Investigator site 111 - CN0860018
  • Chongqing, China, 400042
    • Investigator site 131 - CH0860027
  • Fujian, China, 350005
    • Investigator site 118 - CN0860023
  • Guangzhou, China, 510000
    • Investigator site 120 - CN0860022
  • Guangzhou, China, 51000
    • Investigator site 128 - CH0860053
  • Guanzhou, China, 510000
    • Investigator site 109 - CN0860021
  • Nanjing, China
    • Investigator site 119 - CN0860024
  • Shanghai, China, 200025
    • Investigator site 112 - CN0860020
  • Shanghai, China, 200040
    • Investigator site 108 - CN0860016
  • Wuhan, China, 430022
    • Investigator site 113 - CN0860025
  • Wuhan, China, 430022
    • Investigator site 123 - CN0860019
  • Zhengzhou, China, 450008
    • Investigator site 129 - CH0860026
• France
  • Bobigny, France, 93000
    • Investigator site 34 - FR0330028
  • La Tronche, France, 38700
    • Investigator site 33 - FR0330027
  • Rouen, France, 76031
    • Investigator site 46 - FR0330029
  • Saint-Étienne, France, 42055
    • Investigator site 32 - FR0330026
• Georgia
  • Tbilisi, Georgia, 0159
    • Investigator site 63 - GE9950014
  • Tbilisi, Georgia, 0162
    • Investigator site 35 - GE9950013
  • Tbilisi, Georgia, 0179
    • Investigator site 36 - GE9950015
  • Tbilisi, Georgia, 0160
    • Investigator site 132 - GE9950030
• Germany
  • Berlin, Germany, 10117
    • Investigator site 64 - DE0490029
  • Dresden, Germany, 01307
    • Investigator site 48 - DE0490030
  • Frankfurt am main, Germany, 60590
    • Investigator site 49 - DE0490024
  • Freiburg, Germany, 79104
    • Investigator site 47 - DE0490023
  • Kiel, Germany, 24105
    • Investigator site 38 - DE0490028
  • Lübeck, Germany, 23538
    • Investigator site 37 - DE0490002
  • Marburg, Germany, 35043
    • Investigator site 68 - DE0490001
  • Tübingen, Germany, 72076
    • Investigator site 25 - DE0490025
  • Ulm, Germany, 89081
    • Investigator site 79 - DE0490027
  • Würzburg, Germany, 97080
    • Investigator site 21 - DE0490026
• Greece
  • Athens, Greece, 11525
    • Investigator site 40 - GR0300004
  • Athens, Greece, 16121
    • Investigator site 51 - GR0300006
  • Athens, Greece, 16121
    • Investigator site 69 - GR0300001
  • Chaïdári, Greece, 12462
    • Investigator site 39 - GR0300003
  • Thessaloníki, Greece, 54643
    • Investigator site 50 - GR0300002
  • Thessaloníki, Greece, 56429
    • Investigator site 41 - GR0300005
• Hungary
  • Budapest, Hungary, 1085
    • Investigator site 133 - HU0360023
  • Debrecen, Hungary, 4032
    • Investigator site 22 - HU0360003
  • Pécs, Hungary, 7632
    • Investigator site 14 - HU0360001
  • Szeged, Hungary, 6720
    • Investigator site 42 - HU0360002
• India
  • Ahmedabad, India, 380016
    • Investigator site 80 - IN0910002
  • Chandigarh, India, 160012
    • Investigator site 100 - IN0910001
  • Lucknow, India, 226005
    • Investigator site 90 - IN0910004
  • Nagpur, India, 440003
    • Investigator site 91 - IN0910003
• Israel
  • Tel Aviv, Israel, 64239
    • Investigator site 12 - ISR9720002
• Italy
  • Catania, Italy, 95123
    • Investigator site 104 - IT0390039
  • Firenze, Italy, 50125
    • Investigator site 52 - IT0390031
  • Genova, Italy, 16132
    • Investigator site 92 - IT0390030
  • Perugia, Italy, 06129
    • Investigator site 70 - IT0390038
  • Roma, Italy, 00168
    • Investigator site 43 - IT390005
  • Siena, Italy, 53100
    • Investigator site 71 - IT0390040
  • Lazio
    • Roma, Lazio, Italy, 00167
      • Investigator site 11 - IT0390006
• Japan
  • Aichi, Japan, 480-1195
    • Investigator site 94 - JP0810046
  • Hiroshima, Japan, 734-8551
    • Investigator site 81 - JP0810040
  • Kurume, Japan, 830-001
    • Investigator site 85 - JP0810050
  • Kōfu, Japan, 400-8506
    • Investigator site 82 - JP0810042
  • Okayama, Japan, 700-8558
    • Investigator site 84 - JP0810047
  • Okayama, Japan, 701-0192
    • Investigator site 93 - JP0810041
  • Osaka, Japan, 545-8586
    • Investigator site 86 - JP0810049
  • Sapporo, Japan, 060-8648
    • Investigator site 74 - JP0810045
  • Sendai, Japan, 980-8574
    • Investigator site 124 - JP0810067
  • Tokyo, Japan, 113-8431
    • Investigator site 83 - JP0810043
• Poland
  • Katowice, Poland, 40-081
    • Investigator site 26 - PL0480027
  • Poznań, Poland, 60-369
    • Investigator site 95 - PL0480036
  • Rzeszów, Poland, 35-055
    • Investigator site 27 - PL0480025
  • Wrocław, Poland, 50-566
    • Investigator site 28 - PL0480028
  • Łódź, Poland, 90-647
    • Investigator site 72 - PL0480032
• Romania
  • Bucharest, Romania, 011216
    • Investigator site 106 - RO0400013
  • Cluj-Napoca, Romania, 400006
    • Investigator site 105 - RO0400014
  • Iaşi, Romania, 700111
    • Investigator site 107 - RO0400015
• Russian Federation
  • Chelyabinsk, Russian Federation, 454092
    • Investigator site 54 - RU0070035
  • Ekaterinburg, Russian Federation, 620076
    • Investigator site 58 - RU0070033
  • Kazan, Russian Federation, 420111
    • Investigator site 57 - RU0070029
  • Krasnodar, Russian Federation, 350020
    • Investigator site 55 - RU0070030
  • Rostov-on-Don, Russian Federation, 344002
    • Investigator site 53 - RU0070032
  • Saint Petersburg, Russian Federation, 191123
    • Investigator site 56 - RU0070031
  • Saint Petersburg, Russian Federation, 197022
    • Investigator site 65 - RU0070034
  • Saratov, Russian Federation, 410012/410028
    • Investigator site 66 - RU0070028
• Serbia
  • Belgrad, Serbia, 11000
    • Investigator site 116 - RS3810011
  • Belgrade, Serbia, 11000
    • Investigator site 122 - RS3810010
  • Niš, Serbia, 18000
    • Investigator site 115 - RS3810012
  • Novi Sad, Serbia, 21000
    • Investigator site 114 - RS3810009
• Spain
  • Barcelona, Spain, 08907
    • Investigator site 29 - ES0340026
  • Barcelona, Spain, 8036
    • Investigator site 15 - ES0340032
  • Granada, Spain, 18016
    • Investigator site 130 - ES0340053
  • Madrid, Spain, 28007
    • Investigator site 67 - ES0340034
  • Madrid, Spain, 28034
    • Investigator site 10 - ES0340025
  • Madrid, Spain, 28041
    • Invetistigator site 8 - ES0340029
  • Madrid, Spain, 28046
    • Investigator site 6 - ES0340027
  • Málaga, Spain, 28009
    • Investigator site 134 - ES0340057
  • Pamplona, Spain, 31008
    • Investigator site 23 - ES0340031
  • Sevilla, Spain, 41013
    • Investigator site 7 - ES0340028
• Turkey
  • Gaziantep, Turkey, 27310
    • Investigator site 76 - TR0900020
  • Istanbul, Turkey, 34098
    • Investigator site 75 - TR0900012
  • Istanbul, Turkey, 34722
    • Investigator site 87 - TR0900011
• Ukraine
  • Dnipro, Ukraine, 49074
    • Investigator site 89 - UA3800017
  • Ivano-Frankivs'k, Ukraine, 76018
    • Investigator site 45 - UA3800023
  • Kyiv, Ukraine, 4050
    • Investigator site 16 - UA3800020
  • Kyiv, Ukraine, 4209
    • Investigator site 18 - UA3800019
  • Lviv, Ukraine, 79013
    • Investigator site 62 - UA3800021
  • Zaporizhzhia, Ukraine, 69063
    • Investigator site 17 - UA3800018
• United Kingdom
  • Birmingham, United Kingdom, B15 2GW
    • Investigator site 117 - UK0440021
  • Bristol, United Kingdom, BS2 8HW
    • Investigator site 96 - UK0440022
  • Southampton, United Kingdom, SO16 6YD
    • Investigator site 135 - GB0440037
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Investigator site 77 - US0010086
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Investigator site 97 - US0010091
  • California
    • Redwood City, California, United States, 94063
      • Investigator site 121 - US0010092
  • Colorado
    • Castle Rock, Colorado, United States, 80109
      • Investigator site 125 - US0010153
  • Florida
    • Boca Raton, Florida, United States, 33428
      • Investigator site 2 - US0010087
    • Miami, Florida, United States, 33173
      • Investigator site 99 - US0010117
    • Orlando, Florida, United States, 32827
      • Investigator site 78 - US0010109
  • Indiana
    • West Lafayette, Indiana, United States, 47906
      • Investigator site 127 - US0010155
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Investigator site 61 - US0010090
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Investigator site 102 - US0010098
  • New York
    • Buffalo, New York, United States, 14203-1070
      • Investigator site 19 - US0010088
    • New York, New York, United States, 10128
      • Investigator site 136 - US0010196
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Investigator site 60 - US0010096
  • Ohio
    • Cleveland, Ohio, United States, 44106-1716
      • Investigator site 20 - US0010094
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Investigator site 73 - US00100
    • Philadelphia, Pennsylvania, United States, 19140
      • Investigator site 101 - US0010097
  • Texas
    • Dallas, Texas, United States, 75246
      • Investigator site 98 - US0010107
    • Dripping Springs, Texas, United States, 78620
      • Investigator site 1 - US0010084
    • Houston, Texas, United States, 77004
      • Investigator site 126 - US0010182
    • Houston, Texas, United States, 77008
      • Investigator site 88 - US0010114
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Investigator site 59 - US0010106

Participation Criteria

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 is male or female, and aged from 18 years at the time of signing the informed consent form (ICF).
3. The participant has a clinical diagnosis of PV (mucosal, cutaneous, mucocutaneous) or PF which has been confirmed by cutaneous histology, positive direct immunofluorescence (IF), and positive indirect IF and/or enzyme-linked immunosorbent assay (ELISA).

4. The participant meets one of the following profiles:

   1. Newly diagnosed disease with PDAI ≥15 at baseline and naïve to treatment
   2. Newly diagnosed disease with PDAI ≥15 while receiving a first course of oral prednisone (or equivalent). According to clinical judgment, the participant has shown no significant improvement of PV or PF signs for at least 2 weeks before baseline and is considered fit to start prednisone treatment at 0.5 mg/kg qd at baseline.
   3. Experiencing flare with PDAI ≥15, a maximum of 4 years since diagnosis, and off prednisone therapy ± a conventional immunosuppressant (e.g., azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil) or dapsone. Note: conventional immunosuppressants and dapsone must be discontinued before baseline.
   4. Experiencing flare with PDAI ≥15, a maximum of 4 years since diagnosis, and receiving a tapered dose of oral prednisone (or the equivalent), provided that prednisone has been given at stable dose ± a conventional immunosuppressant for at least 2 weeks and patients are fit to start prednisone treatment at 0.5 mg/kg qd at baseline.

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

   1. Male participants: Male participants must agree to use acceptable method of contraception, and not donate sperm from signing the ICF until the end of the study.

   2. Female participants: Women of childbearing potential must:

      • have a negative serum pregnancy test at screening and negative urine pregnancy test at baseline before the IMP can be administered.
      • agree to use a highly effective or acceptable contraception method, which should be maintained at minimum until after the last dose of IMP
6. For Japanese participants enrolled in sites in Japan only: A Japanese participant is defined as a participant whose parents and 4 grandparents are Japanese, and who has Japanese nationality, was born in Japan, has not lived outside of Japan for a total of >10 years, and currently lives in Japan.

Exclusion Criteria:

1. Participant has a confirmed diagnosis of paraneoplastic pemphigus, drug-induced pemphigus, pemphigus vegetans, pemphigus erythematosus, or any other non-PV/non-PF autoimmune blistering disease.
2. Participants with mild disease severity as defined by PDAI <15 at baseline.
3. Participants who show a significant improvement of PV or PF in the period from screening to baseline according to clinical judgment (eg, the patient has achieved DC or a substantial reduction in PDAI activity score during screening period).
4. The participant has been administered therapy(ies) other than oral prednisone or conventional immunosuppressants (e.g., azathioprine, cyclophosphamide, methotrexate, mycophenolate mofetil) or dapsone within 2 months before the baseline visit and that can affect clinical disease activity. For example, excluded medications are intravenous methylprednisolone, dapsone, sulfasalazine, tetracyclines, nicotinamide at doses above the recommended daily allowance (RDA)/dietary reference intake (DRI), plasmapheresis/ plasma exchange, immunoadsorption, and IVIg.
5. Use of any monoclonal antibody (including rituximab or another anti-CD20 biologic) within 6 months before the baseline visit.
6. Known hypersensitivity to any of the components of the administered treatments.
7. The participant has a known contraindication to oral prednisone.
8. The participant has a history of refractory disease, as defined by a failure to respond to first-line and second-line therapies

9. Participants who have a history of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before first IMP administration. Participants with any of 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
10. 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 patient at undue risk.
11. Pregnant and lactating women and those intending to become pregnant during the trial.
12. Current or history (i.e. within 12 months of screening) of alcohol, drug, or medication abuse.
13. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of PV or PF or put the participant at undue risk.
14. The participant has a Karnofsky Performance score <60%.
15. Vaccination with live viral vaccines within 28 days prior to randomization.
16. The participant has clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection.
17. Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B Virus, Hepatitis C Virus , HIV.
18. The participant has total immunoglobulin G (IgG) <6 g/L at screening.
19. The participant has previously participated in a trial with efgartigimod and has received at least one administration of IMP.
20. Use of an investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to first IMP administration

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

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
Experimental: placebo; patients receiving placebo on top of prednisone	Other: Placebo; Subcutaneous injection of placebo; Drug: prednisone; Oral prednisone tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Pemphigus Vulgaris (PV) participants who achieve complete clinical remission (CR) on minimal Prednisone therapy	Proportion of Pemphigus Vulgaris participants who achieve Clinical Remission on minimal Prednisone therapy	30 weeks treatment period

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal Prednisone therapy	Proportion of Pemphigus Vulgaris and Pemphigus Foliaceus participants who achieve complete clinical remission on minimal Prednisone therapy	30 weeks treatment period
Cumulative prednisone dose over the trial in Pemphigus Vulgaris participants	Cumulative prednisone dose over the trial in Pemphigus Vulgaris participants	Up to 30 weeks
Time to complete clinical remission in Pemphigus Vulgaris participants	Time to complete clinical remission in Pemphigus Vulgaris participants	Up to 30 weeks
Time to Disease Control (DC) in Pemphigus Vulgaris (PV) participants	Time to Disease Control in Pemphigus Vulgaris participants	Up to 30 weeks
Proportion of Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal prednisone therapy	Proportion of Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal prednisone therapy	30 weeks treatment period
Cumulative prednisone dose over the trial in Pemphigus Vulgaris and Pemphigus Foliaceus participants	Cumulative prednisone dose over the trial in Pemphigus Vulgaris and Pemphigus Foliaceus participants	Up to 30 weeks
Time to complete clinical remission in Pemphigus Vulgaris and Pemphigus Foliaceus participants	Time to complete clinical remission in Pemphigus Vulgaris and Pemphigus Foliaceus participants	Up to 30 weeks
Time to disease control in Pemphigus Vulgaris and Pemphigus Foliaceus participants	Time to disease control in Pemphigus Vulgaris and Pemphigus Foliaceus participants	Up to 30 weeks
Rate of treatment failure	Rate of treatment failure	Up to 30 weeks
Rate of flare	Rate of flare	Up to 30 weeks
Pemphigus Disease Area Index at each visit	Pemphigus Disease Area Index at each visit	Up to 41 weeks
Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)	Incidence of Treatment-Emergent Adverse Events, Adverse Events of Special Interest, and Serious Adverse Events	Up to 41 weeks
Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)	Severity of Treatment-Emergent Adverse Events, Adverse Events of Special Interest, and Serious Adverse Events	Up to 41 weeks
Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS)	Composite Glucocorticoid Toxicity Index comprising the Aggregate Improvement Score and the Cumulative Worsening Score	Up to 30 weeks
EuroQol Five-Dimension Five-Level Scale (EQ-5D-5L) score	EuroQol Five-Dimension Five-Level Scale score	30 weeks treatment period
Autoimmune Bullous Disease Quality of Life (ABQOL) score	Autoimmune Bullous Disease Quality of Life score	30 weeks treatment period
Efgartigimod serum concentrations	Efgartigimod serum concentrations	Up to 38 weeks
Total immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels	Total immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels	Up to 41 weeks
Anti desmoglein-1 and -3 autoantibodies serum levels	Anti desmoglein-1 and -3 autoantibodies serum levels	Up to 41 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod PH20 SC	Incidence of anti-drug antibodies to efgartigimod PH20 SC	Up to 38 weeks
Prevalence of anti-drug antibodies (ADA) to efgartigimod PH20 SC	Prevalence of anti-drug antibodies to efgartigimod PH20 SC	Up to 38 weeks
Incidence of antibodies produced against recombinant human hyaluronidase (rHuPH20) (plasma levels)	Incidence of antibodies produced against recombinant human hyaluronidase (rHuPH20) (plasma levels)	Up to 31 weeks
Prevalence of antibodies produced against recombinant human hyaluronidase (rHuPH20)	Prevalence of antibodies produced against recombinant human hyaluronidase (rHuPH20)	Up to 31 weeks
Number of participants or caregivers completing the self-administration training	Number of participants or caregivers completing the self-administration training	Up to 41 weeks
Percentage of participants or caregivers completing the self-administration training	Percentage of participants or caregivers completing the self-administration training	Up to 41 weeks
Number of participants or caregivers determined by the site staff to be sufficiently competent to self-administer efgartigimod PH20 SC	Number of participants or caregivers determined by the site staff to be sufficiently competent to self-administer efgartigimod PH20 SC	Up to 41 weeks
Percentage of participants or caregivers determined by the site staff to be sufficiently competent to self-administer efgartigimod PH20 SC	Percentage of participants or caregivers determined by the site staff to be sufficiently competent to self-administer efgartigimod PH20 SC	Up to 41 weeks
Number of participants or caregivers that self-administer efgartigimod PH20 SC under site staff supervision	Number of participants or caregivers that self-administer efgartigimod PH20 SC under site staff supervision	Up to 41 weeks
Percentage of participants or caregivers that self-administer efgartigimod PH20 SC under site staff supervision	Percentage of participants or caregivers that self-administer efgartigimod PH20 SC under site staff supervision	Up to 41 weeks

Collaborators and Investigators

• Sponsor: argenx

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2020
• Primary Completion (Actual): August 22, 2023
• Study Completion (Actual): August 22, 2023

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): September 22, 2023
• Last Update Submitted That Met QC Criteria: September 21, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Immune System Diseases; Autoimmune Diseases; Skin Diseases, Vesiculobullous; Pemphigus; Physiological Effects of Drugs; Anti-Inflammatory Agents; Antineoplastic Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Prednisone
• Other Study ID Numbers: ARGX-113-1904

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No