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)

NCT04598477 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: ARGX-113-1905
• Study Type: interventional
• Target: 183
• Locations: 21 countries
• Sites: 127

Brief 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. 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

Conditions

Pemphigus Vulgaris; Pemphigus Foliaceus

Outcome Measures

Primary Outcomes (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).

  Up to 60 weeks

Secondary Outcomes (10)

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

  Up to 60 weeks

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

  Up to 60 weeks

• Time to DC in Participants With PV and PF

  Up to 52 weeks

• Time to CR in Participants With PV and PF

  Up to 52 weeks

• Time to CRmin in Participants With PV and PF

  Up to 52 weeks

Study Arms (1)

efgartigimod PH20 SC

EXPERIMENTAL

patients receiving efgartigimod PH20 SC on top of prednisone

Biological: efgartigimod PH20 SC; Drug: prednisone

Interventions

efgartigimod PH20 SC BIOLOGICAL

Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer

efgartigimod PH20 SC

prednisone DRUG

Oral prednisone tablets

efgartigimod PH20 SC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• argenx: lead

Study Sites (127)

Investigator site 115 - US0010086

Birmingham, Alabama, 35233, United States

Location

Investigator site 89 - US0010091

Scottsdale, Arizona, 85259, United States

Location

Investigator site 124 - US0010092

Redwood City, California, 94063, United States

Location

Investigator site 1 - US0010087

Boca Raton, Florida, 33428, United States

Location

Investigator site 90 - US0010117

Miami, Florida, 33173, United States

Location

Investigator site 91 - US0010109

Orlando, Florida, 32827, United States

Location

Investigator site 126 - US0010090

Minneapolis, Minnesota, 55455, United States

Location

Investigator site 111 - US0010098

St Louis, Missouri, 63110, United States

Location

Investigator site 10 - US0010088

Buffalo, New York, 14203-1070, United States

Location

Investigator site 76 - US0010096

Durham, North Carolina, 27710, United States

Location

Investigator site 30 - US0010094

Cleveland, Ohio, 44106-1716, United States

Location

Investigator site 84 - US0010089

Philadelphia, Pennsylvania, 19104, United States

Location

Investigator site 103 - US0010097

Philadelphia, Pennsylvania, 19140, United States

Location

Investigator site 125 - US0010107

Dallas, Texas, 75246, United States

Location

Investigator site 87 - US0010084

Dripping Springs, Texas, 78620, United States

Location

Investigator site 88 - US0010114

Houston, Texas, 77008, United States

Location

Investigator site 44 - US0010106

Norfolk, Virginia, 23502, United States

Location

Investigator site 15 - AU0610006

Sydney, New South Wales, 2217, Australia

Location

Investigator site 11 - AU0610007

Parkville, Victoria, 3050, Australia

Location

Investigator site 92 - AU0610013

Melbourne, 3065, Australia

Location

Investigator site 17 - BG3590012

Pleven, 5800, Bulgaria

Location

Investigator site 18 - BG3590013

Plovdiv, 4000, Bulgaria

Location

Investigator site 2 - BG3590010

Sofia, 1431, Bulgaria

Location

Investigator site 16 - BG3590009

Sofia, 1510, Bulgaria

Location

Investigator site 3 - BG3590011

Sofia, 1606, Bulgaria

Location

Investigator site 101 - CN0860017

Beijing, 100034, China

Location

Investigator site 107 - CN0860018

Chengdu, 610000, China

Location

Investigator site 118 - CH0860027

Chongqing, 400042, China

Location

Investigator site 120 - CH0860023

Fuzhou, 35005, China

Location

Investigator site 119 - CH0860022

Guangzhou, 510000, China

Location

Investigator site 116 - CH0860053

Guangzhou, 51000, China

Location

Investigator site 100 - CN0860021

Guanzhou, 510000, China

Location

Investigator site 113 - CN0860024

Nanjing, 210042, China

Location

Investigator site 102 - CN0860020

Shanghai, 200025, China

Location

Investigator site 99 - CN0860016

Shanghai, 200040, China

Location

Investigator site 112 - CN0860019

Wuhan, 430022, China

Location

Investigator site 121 - CH0860025

Wuhan, 430022, China

Location

Investigator site 117 - CH0860026

Zhengzhou, 450008, China

Location

Investigator site 77 - FR0330028

Bobigny, 93000, France

Location

Investigator site 60 - FR0330027

La Tronche, 38700, France

Location

Investigator site 108 - FR0330029

Rouen, 76031, France

Location

Investigator site 51 - FR0330026

Saint-Etienne, 42055, France

Location

Investigator site 78 - GE9950014

Tbilisi, 0159, Georgia

Location

Investigator site 127 - GE9950030

Tbilisi, 0160, Georgia

Location

Investigator site 32 - GE9950013

Tbilisi, 0162, Georgia

Location

Investigator site 31 - GE9950015

Tbilisi, 0179, Georgia

Location

Investigator site 45 - DE0490029

Berlin, 10117, Germany

Location

Investigator site 34 - DE0490030

Dresden, 01307, Germany

Location

Investigator site 33 - DE0490024

Frankfurt am Main, 60590, Germany

Location

Investigator site 53 - DE0490023

Freiburg im Breisgau, 79104, Germany

Location

Investigator site 35 - DE0490028

Kiel, 24105, Germany

Location

Investigator site 20 - DE0490002

Lübeck, 23538, Germany

Location

Investigator site 52 - DE0490001

Marburg, 35043, Germany

Location

Investigator site 19 - DE0490025

Tübingen, 72076, Germany

Location

Investigator site 93 - DE0490027

Ulm, 89081, Germany

Location

Investigator site 4 - DE0490026

Würzburg, 97080, Germany

Location

Investigator site 21 - GR030004

Athens, 11525, Greece

Location

Investigator site 37 - GR030006

Athens, 16121, Greece

Location

Investigator site 54 - GR0300001

Athens, 16121, Greece

Location

Investigator site 22 - GR030003

Chaïdári, 12462, Greece

Location

Investigator site 36 - GR0300002

Thessaloniki, 54643, Greece

Location

Investigator site 23 - GR030005

Thessaloniki, 56429, Greece

Location

Investigator site 6 - HU0360003

Debrecen, 4032, Hungary

Location

Investigator site 5 - HU0360001

Pécs, 7632, Hungary

Location

Investigator site 24 - HU0360002

Szeged, 6720, Hungary

Location

Investigator site 65 - IN0910002

Ahmedabad, 380016, India

Location

Investigator site 94 - IN0910001

Chandigarh, 160012, India

Location

Investigator site 79 - IN0910004

Lucknow, 226005, India

Location

Investigator site 80 - IN0910003

Nagpur, 440003, India

Location

Investigator site 85 - IL9720002

Tel Aviv, 64239, Israel

Location

Investigator site 95 - IT0390039

Catania, 95123, Italy

Location

Investigator site 38 - IT0390031

Florence, 50125, Italy

Location

Investigator site 81 - IT0390030

Genova, 16132, Italy

Location

Investigator site 55 - IT0390038

Perugia, 06129, Italy

Location

Investigator site 12 - IT0390006

Roma, 00167, Italy

Location

Investigator site 25 - IT-0390005

Roma, 00168, Italy

Location

Investigator site 61 - IT0390040

Siena, 53100, Italy

Location

Investigator site 82 - JP0810046

Aichi, 480-1195, Japan

Location

Investigator site 68 - JP0810040

Hiroshima, 734-8551, Japan

Location

Investigator site 66 - JP0810042

Kofu, 400-8506, Japan

Location

Investigator site 69 - JP0810050

Kurume, 830-001, Japan

Location

Investigator site 73 - JP0810047

Okayama, 700-8558, Japan

Location

Investigator site 70 - JP0810041

Okayama, 701-0192, Japan

Location

Investigator site 71 - JP0810049

Osaka, 545-8586, Japan

Location

Investigator site 72 - JP0810045

Sapporo, 060-8648, Japan

Location

Investigator site 114 - JP0810067

Sendai, 980-8574, Japan

Location

Investigator site 67 - JP0810043

Tokyo, 113-8431, Japan

Location

Investigator site 27 - PL0480027

Katowice, 40-081, Poland

Location

Investigator site 56 - PL0480032

Lodz, 90-647, Poland

Location

Investigator site 86 - PL0480036

Poznan, 60-369, Poland

Location

Investigator site 28 - PL0480025

Rzeszów, 35-055, Poland

Location

Investigator site 26 - PL0480028

Wroclaw, 50-566, Poland

Location

Investigator site 97 - RO0400013

Bucharest, 011216, Romania

Location

Investigator 96 - RO0400014

Cluj-Napoca, 400006, Romania

Location

Investigator site 98 - RO0400015

Iași, 700111, Romania

Location

Investigator site 40 - RU0070035

Chelyabinsk, 454092, Russia

Location

Investigator site 48 - RU0070029

Kazan', 420111, Russia

Location

Investigator site 49 - RU0070030

Krasnodar, 350020, Russia

Location

Investigator site 39 - RU0070032

Rostov-on-Don, 344002, Russia

Location

Investigator site 46 - RU0070031

Saint Petersburg, 191123, Russia

Location

Investigator site 50 - RU0070034

Saint Petersburg, 197022, Russia

Location

Investigator site 47 - RU0070028

Saratov, 410012/410028, Russia

Location

Investigator site 41 - RU0070033

Yekaterinburg, 620076, Russia

Location

Investigator site 109 - RS3810011

Belgrade, 11000, Serbia

Location

Investigator site 110 - RS3810010

Belgrade, 11000, Serbia

Location

Investigator site 105 - RS3810012

Niš, 18000, Serbia

Location

Investigator site 104 - RS3810009

Novi Sad, 21000, Serbia

Location

Investigator site 62 - ES0340026

Barcelona, 08907, Spain

Location

Investigator site 13 - ES0340032

Barcelona, 8036, Spain

Location

Investigator site 122 - ES0340053

Granada, 18016, Spain

Location

Investigator site 59 - ES0340034

Madrid, 28007, Spain

Location

Investigator site 42 - ES0340025

Madrid, 28034, Spain

Location

Investigator site 7 - ES0340029

Madrid, 28041, Spain

Location

Investigator site 57 - ES0340027

Madrid, 28046, Spain

Location

Investigator site 58 - ES0340028

Seville, 41013, Spain

Location

Investigator site 64 - TR0900020

Gaziantep, 27310, Turkey (Türkiye)

Location

Investigator site 63 - TR0900012

Istanbul, 34098, Turkey (Türkiye)

Location

Investigator site 74 - TR0900011

Istanbul, 34722, Turkey (Türkiye)

Location

Investigator site 75 - UA3800017

Dnipro, 49074, Ukraine

Location

Investigator site 29 - UA3800023

Ivano-Frankivsk, 76018, Ukraine

Location

Investigator site 14 - UA3800020

Kyiv, 04050, Ukraine

Location

Investigator site 8 - UA3800019

Kyiv, 4209, Ukraine

Location

Investigator site 43 - UA3800021

Lviv, 79013, Ukraine

Location

Investigator site 9 - UA3800018

Zaporizhzhia, 69063, Ukraine

Location

Investigator site 106 - UK0440021

Birmingham, B15 2GW, United Kingdom

Location

Investigator site 83 - UK0440022

Bristol, BS2 8HW, United Kingdom

Location

Investigator site 123 - UK0440037

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Pemphigus

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Limitations and Caveats

Study was terminated prematurely by the Sponsor.

Results Point of Contact

• Title: Regulatory Manager
• Organization: Argenx

regulatory@argenx.com

Please email:

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2020
• First Posted: October 22, 2020
• Study Start: July 15, 2021
• Primary Completion: March 25, 2024
• Study Completion: March 25, 2024
• Last Updated: March 30, 2025
• Results First Posted: March 30, 2025

Record last verified: 2025-03

Locations

RO (3); UA (6); IL (1); IN (4); SE (4); PL (5); FR (4); ES (8); GB (3); HU (3); US (17); AU (3); DE (10); GE (4); RU (8); IT (7); BU (5); GR (6); JP (10); CN (13); TU (3)