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)

NCT ID: NCT04598477

Last Updated: 2025-03-30

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

183 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-07-15

Study Completion Date

2024-03-25

Brief Summary

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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

Detailed Description

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Conditions

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Pemphigus Vulgaris Pemphigus Foliaceus

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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efgartigimod PH20 SC

patients receiving efgartigimod PH20 SC on top of prednisone

Group Type EXPERIMENTAL

efgartigimod PH20 SC

Intervention Type BIOLOGICAL

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

prednisone

Intervention Type DRUG

Oral prednisone tablets

Interventions

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efgartigimod PH20 SC

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

Intervention Type BIOLOGICAL

prednisone

Oral prednisone tablets

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Investigator site 115 - US0010086

Birmingham, Alabama, United States

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Investigator site 89 - US0010091

Scottsdale, Arizona, United States

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Investigator site 124 - US0010092

Redwood City, California, United States

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Investigator site 1 - US0010087

Boca Raton, Florida, United States

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Investigator site 90 - US0010117

Miami, Florida, United States

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Investigator site 91 - US0010109

Orlando, Florida, United States

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Investigator site 126 - US0010090

Minneapolis, Minnesota, United States

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Investigator site 111 - US0010098

St Louis, Missouri, United States

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Investigator site 10 - US0010088

Buffalo, New York, United States

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Investigator site 76 - US0010096

Durham, North Carolina, United States

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Investigator site 30 - US0010094

Cleveland, Ohio, United States

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Investigator site 84 - US0010089

Philadelphia, Pennsylvania, United States

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Investigator site 103 - US0010097

Philadelphia, Pennsylvania, United States

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Investigator site 125 - US0010107

Dallas, Texas, United States

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Investigator site 87 - US0010084

Dripping Springs, Texas, United States

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Investigator site 88 - US0010114

Houston, Texas, United States

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Investigator site 44 - US0010106

Norfolk, Virginia, United States

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Investigator site 15 - AU0610006

Sydney, New South Wales, Australia

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Investigator site 11 - AU0610007

Parkville, Victoria, Australia

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Investigator site 92 - AU0610013

Melbourne, , Australia

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Investigator site 17 - BG3590012

Pleven, , Bulgaria

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Investigator site 18 - BG3590013

Plovdiv, , Bulgaria

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Investigator site 2 - BG3590010

Sofia, , Bulgaria

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Investigator site 16 - BG3590009

Sofia, , Bulgaria

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Investigator site 3 - BG3590011

Sofia, , Bulgaria

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Investigator site 101 - CN0860017

Beijing, , China

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Investigator site 107 - CN0860018

Chengdu, , China

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Investigator site 118 - CH0860027

Chongqing, , China

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Investigator site 120 - CH0860023

Fuzhou, , China

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Investigator site 119 - CH0860022

Guangzhou, , China

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Investigator site 116 - CH0860053

Guangzhou, , China

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Investigator site 100 - CN0860021

Guanzhou, , China

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Investigator site 113 - CN0860024

Nanjing, , China

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Investigator site 102 - CN0860020

Shanghai, , China

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Investigator site 99 - CN0860016

Shanghai, , China

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Investigator site 112 - CN0860019

Wuhan, , China

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Investigator site 121 - CH0860025

Wuhan, , China

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Investigator site 117 - CH0860026

Zhengzhou, , China

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Investigator site 77 - FR0330028

Bobigny, , France

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Investigator site 60 - FR0330027

La Tronche, , France

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Investigator site 108 - FR0330029

Rouen, , France

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Investigator site 51 - FR0330026

Saint-Etienne, , France

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Investigator site 78 - GE9950014

Tbilisi, , Georgia

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Investigator site 127 - GE9950030

Tbilisi, , Georgia

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Investigator site 32 - GE9950013

Tbilisi, , Georgia

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Investigator site 31 - GE9950015

Tbilisi, , Georgia

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Investigator site 45 - DE0490029

Berlin, , Germany

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Investigator site 34 - DE0490030

Dresden, , Germany

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Investigator site 33 - DE0490024

Frankfurt am Main, , Germany

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Investigator site 53 - DE0490023

Freiburg im Breisgau, , Germany

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Investigator site 35 - DE0490028

Kiel, , Germany

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Investigator site 20 - DE0490002

Lübeck, , Germany

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Investigator site 52 - DE0490001

Marburg, , Germany

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Investigator site 19 - DE0490025

Tübingen, , Germany

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Investigator site 93 - DE0490027

Ulm, , Germany

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Investigator site 4 - DE0490026

Würzburg, , Germany

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Investigator site 21 - GR030004

Athens, , Greece

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Investigator site 37 - GR030006

Athens, , Greece

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Investigator site 54 - GR0300001

Athens, , Greece

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Investigator site 22 - GR030003

Chaïdári, , Greece

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Investigator site 36 - GR0300002

Thessaloniki, , Greece

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Investigator site 23 - GR030005

Thessaloniki, , Greece

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Investigator site 6 - HU0360003

Debrecen, , Hungary

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Investigator site 5 - HU0360001

Pécs, , Hungary

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Investigator site 24 - HU0360002

Szeged, , Hungary

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Investigator site 65 - IN0910002

Ahmedabad, , India

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Investigator site 94 - IN0910001

Chandigarh, , India

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Investigator site 79 - IN0910004

Lucknow, , India

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Investigator site 80 - IN0910003

Nagpur, , India

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Investigator site 85 - IL9720002

Tel Aviv, , Israel

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Investigator site 95 - IT0390039

Catania, , Italy

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Investigator site 38 - IT0390031

Florence, , Italy

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Investigator site 81 - IT0390030

Genova, , Italy

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Investigator site 55 - IT0390038

Perugia, , Italy

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Investigator site 12 - IT0390006

Roma, , Italy

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Investigator site 25 - IT-0390005

Roma, , Italy

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Investigator site 61 - IT0390040

Siena, , Italy

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Investigator site 82 - JP0810046

Aichi, , Japan

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Investigator site 68 - JP0810040

Hiroshima, , Japan

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Investigator site 66 - JP0810042

Kofu, , Japan

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Investigator site 69 - JP0810050

Kurume, , Japan

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Investigator site 73 - JP0810047

Okayama, , Japan

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Investigator site 70 - JP0810041

Okayama, , Japan

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Investigator site 71 - JP0810049

Osaka, , Japan

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Investigator site 72 - JP0810045

Sapporo, , Japan

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Investigator site 114 - JP0810067

Sendai, , Japan

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Investigator site 67 - JP0810043

Tokyo, , Japan

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Investigator site 27 - PL0480027

Katowice, , Poland

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Investigator site 56 - PL0480032

Lodz, , Poland

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Investigator site 86 - PL0480036

Poznan, , Poland

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Investigator site 28 - PL0480025

Rzeszów, , Poland

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Investigator site 26 - PL0480028

Wroclaw, , Poland

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Investigator site 97 - RO0400013

Bucharest, , Romania

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Investigator 96 - RO0400014

Cluj-Napoca, , Romania

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Investigator site 98 - RO0400015

Iași, , Romania

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Investigator site 40 - RU0070035

Chelyabinsk, , Russia

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Investigator site 48 - RU0070029

Kazan', , Russia

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Investigator site 49 - RU0070030

Krasnodar, , Russia

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Investigator site 39 - RU0070032

Rostov-on-Don, , Russia

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Investigator site 46 - RU0070031

Saint Petersburg, , Russia

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Investigator site 50 - RU0070034

Saint Petersburg, , Russia

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Investigator site 47 - RU0070028

Saratov, , Russia

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Investigator site 41 - RU0070033

Yekaterinburg, , Russia

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Investigator site 109 - RS3810011

Belgrade, , Serbia

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Investigator site 110 - RS3810010

Belgrade, , Serbia

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Investigator site 105 - RS3810012

Niš, , Serbia

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Investigator site 104 - RS3810009

Novi Sad, , Serbia

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Investigator site 62 - ES0340026

Barcelona, , Spain

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Investigator site 13 - ES0340032

Barcelona, , Spain

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Investigator site 122 - ES0340053

Granada, , Spain

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Investigator site 59 - ES0340034

Madrid, , Spain

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Investigator site 42 - ES0340025

Madrid, , Spain

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Investigator site 7 - ES0340029

Madrid, , Spain

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Investigator site 57 - ES0340027

Madrid, , Spain

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Investigator site 58 - ES0340028

Seville, , Spain

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Investigator site 64 - TR0900020

Gaziantep, , Turkey (Türkiye)

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Investigator site 63 - TR0900012

Istanbul, , Turkey (Türkiye)

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Investigator site 74 - TR0900011

Istanbul, , Turkey (Türkiye)

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Investigator site 75 - UA3800017

Dnipro, , Ukraine

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Investigator site 29 - UA3800023

Ivano-Frankivsk, , Ukraine

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Investigator site 14 - UA3800020

Kyiv, , Ukraine

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Investigator site 8 - UA3800019

Kyiv, , Ukraine

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Investigator site 43 - UA3800021

Lviv, , Ukraine

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Investigator site 9 - UA3800018

Zaporizhzhia, , Ukraine

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Investigator site 106 - UK0440021

Birmingham, , United Kingdom

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Investigator site 83 - UK0440022

Bristol, , United Kingdom

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Investigator site 123 - UK0440037

Southampton, , United Kingdom

Site Status

Countries

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United States Australia Bulgaria China France Georgia Germany Greece Hungary India Israel Italy Japan Poland Romania Russia Serbia Spain Turkey (Türkiye) Ukraine United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ARGX-113-1905

Identifier Type: -

Identifier Source: org_study_id

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