A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)

NCT04280718 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: ARGX-113-19022019-003107-35
• Study Type: interventional
• Target: 229
• Locations: 24 countries
• Sites: 147

Brief Summary

This is the open-label extension study of phase II ARGX-113-1802 to evaluate the long-term safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP. Patients already stabilized on efgartigimod PH20 SC will also have the opportunity to participate in a sub study to explore less frequent dosing of efgartigimod PH20 SC.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events and serious adverse events

  Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study

Secondary Outcomes (16)

• Change from baseline over time of the adjusted INCAT score

  Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study

• Change from baseline over time of the MRC Sum score

  Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study

• Change from baseline over time of I-RODS disability scores

  Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study

• Change from baseline over time of mean grip strength

  Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study

• Change from baseline over time of TUG score

  Up to 48 weeks per cycle (each cycle is 48 weeks) until the end of the study

Study Arms (1)

efgartigimod PH20 SC

EXPERIMENTAL

Patients treated with efgartigimod PH20 SC

Biological: Efgartigimod PH20 SC

Interventions

Efgartigimod PH20 SC BIOLOGICAL

Subcutaneous administration of efgartigimod

Also known as: ARGX-113

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, 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) of this trial.
• Male or female patient with one of the following options:
• Have completed the Week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or
• Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or
• Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or
• Have completed the Week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment.
• Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.
• Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last administration of IMP.

You may not qualify if:

• Week-48/ED visit in the ARGX-113-1802 trial or the Week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP.
• Pregnant and lactating women and those intending to become pregnant during the trial.
• Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or patients who (intend to) use prohibited medications (see protocol) and therapies during the trial, or any other reason which could confound the results of the trial or put the patient at undue risk.

Sponsors & Collaborators

• argenx: lead

Study Sites (147)

Investigator site 0010065

Birmingham, Alabama, 35233-2110, United States

Location

Investigator site 0010013

Phoenix, Arizona, 85018, United States

Location

Investigator site 0010055

Scottsdale, Arizona, 85028, United States

Location

Investigator site 0010032

Carlsbad, California, 92011, United States

Location

Investigator site 10190

Pomona, California, 91767-2009, United States

Location

Investigator site 0010160

Rancho Mirage, California, 92270-4150, United States

Location

Investigator site 0010071

San Francisco, California, 94109, United States

Location

Investigator site 0010057

Centennial, Colorado, 80112, United States

Location

Investigator site 0010072

Boca Raton, Florida, 33487, United States

Location

Investigator site 0010144

Coral Springs, Florida, 33067-4640, United States

Location

Investigator site 0010023

Jacksonville, Florida, 32209, United States

Location

Investigator site 0010068

Maitland, Florida, 32751, United States

Location

Investigator site 0010059

Miami, Florida, 33136, United States

Location

Investigator site 0010006

Tampa, Florida, 33612, United States

Location

Investigator site 0010011

Iowa City, Iowa, 52242, United States

Location

Investigator site 0010015

Fairway, Kansas, 66205, United States

Location

Investigator site 10147

Lexington, Kentucky, 40536, United States

Location

Investigator site 10168

New York, New York, 10016, United States

Location

Investigator site 0010003

Chapel Hill, North Carolina, 27517, United States

Location

Investigator site 0010064

Columbus, Ohio, 43210, United States

Location

Investigator site 0010007

Philadelphia, Pennsylvania, 19107, United States

Location

Investigator site 0010047

Philadelphia, Pennsylvania, 19126, United States

Location

Investigator Site 0010066

Austin, Texas, 78756, United States

Location

Investigator site 0010009

San Antonio, Texas, 78229, United States

Location

Investigator site 0010061

Richmond, Virginia, 23298, United States

Location

Investigator site 0430007

Innsbruck, Austria

Location

Investigator site 0430008

Linz, 4021, Austria

Location

Investigator site 0430005

Vienna, 1090, Austria

Location

Investigator site 0320016

Edegem, Belgium

Location

Investigator site 0320009

Leuven, 3000, Belgium

Location

Investigator site 320024

Liège, 4000, Belgium

Location

Investigator site 320022

Woluwe-Saint-Lambert, 1200, Belgium

Location

Investigator site 3590007

Pleven, 5800, Bulgaria

Location

Investigator site 3590005

Sofia, 1113, Bulgaria

Location

Investigator site 3590008

Sofia, 1431, Bulgaria

Location

Investigator site 3590006

Sofia, 1680, Bulgaria

Location

Investigator site 0860033

Beijing, China

Location

Investigator site 860041

Changsha, China

Location

Investigator site 0860036

Chengdu, China

Location

Investigator site 860049

Chifeng, 024000, China

Location

Investigator site 0860038

Fuzhou, China

Location

Investigator site 0860050

Guangzhou, 510120, China

Location

Investigator site 0860032

Guanzhou, China

Location

Investigator site 0860045

Guiyang, China

Location

Investigator site 0860035

Hangzhou, 310003, China

Location

Investigator site 0860031

Jinan, 250012, China

Location

Investigator site 0860040

Nanchang, 33008, China

Location

Investigator site 0860043

Nanjing, China

Location

Investigator site 0860028

Shanghai, China

Location

Investigator site 860047

Shanghai, China

Location

Investigator site 0860042

Tianjin, China

Location

Investigator site 0860034

Wuhan, 430040, China

Location

Investigator site 0860029

Wuhan, 430060, China

Location

Investigator site 0860048

Xi'an, 710038, China

Location

Investigator site 0860054

Xianyang, 712000, China

Location

Investigator site 4200010

Hradec Králové, 500-03, Czechia

Location

Investigator site 0450002

Aarhus, 8200, Denmark

Location

Investigator site 0450001

Copenhagen, 2100, Denmark

Location

Investigator site 0450003

Odense, 5000, Denmark

Location

Investigator site 00330034

Angers, France

Location

Investigator site 0330013

Bordeaux, 33076, France

Location

Investigator site 330033

Clermont-Ferrand, 63003, France

Location

Investigator site 0330023

Le Kremlin-Bicêtre, 94275, France

Location

Investigator site 0330024

Limoges, 87042, France

Location

Investigator site 330022

Nantes, 44093, France

Location

Investigator site 0330021

Nice, 06202, France

Location

Investigator site 0330035

Paris, France

Location

Investigator site 0330020

Strasbourg, 67098, France

Location

Investigator site 9950020

Kutaisi, 4600, Georgia

Location

Investigator site 9950004

Tbilisi, 0112, Georgia

Location

Investigator site 9950005

Tbilisi, 0112, Georgia

Location

Investigator site 9950002

Tbilisi, Georgia

Location

Investigator Site 9950003

Tbilisi, Georgia

Location

Investigator site 490044

Bochum, 44791, Germany

Location

Investigator site 0490013

Cologne, Germany

Location

Investigator site 490045

Essen, 45147, Germany

Location

Investigator site 490021

Göttingen, 37075, Germany

Location

Investigator site 0490016

Kiel, 24105, Germany

Location

Investigator site 0490019

Potsdam, Germany

Location

Investigator site 9720004

Tel Aviv, 6423906, Israel

Location

Investigator site 0390022

Brescia, Italy

Location

Investigator site 390029

Florence, 50139, Italy

Location

Investigator site 0390024

Genova, 16132, Italy

Location

Investigator site 390027

Messina, 98125, Italy

Location

Investigator site 0390003

Milan, 20122, Italy

Location

Investigator site 0390026

Milan, Italy

Location

Investigator site 0390023

Pisa, 56125, Italy

Location

Investigator site 0390008

Roma, 00189, Italy

Location

Investigator site 0390042

Torino, Italy

Location

Investigator site 0810035

Bunkyō City, Japan

Location

Investigator site 0810002

Chiba, Japan

Location

Investigator site 0810030

Fuchū, 183-0042, Japan

Location

Investigator site 0810031

Fukuoka, 812-8582, Japan

Location

Investigator site 0810065

Ginowan, Japan

Location

Investigator site 0810066

Hakodate, Japan

Location

Investigator site 0810058

Hiroshima, Japan

Location

Investigator site 0810029

Kawagoe, Japan

Location

Investigator site 0810026

Kodaira, 187-8551, Japan

Location

Investigator site 810061

Kyoto, 616-8255, Japan

Location

Investigator site 0810027

Mibu, 321-0293, Japan

Location

Investigator site 0810032

Nagoya, 466-8560, Japan

Location

Investigator site 0810003

Osaka, 565-0871, Japan

Location

Investigator site 0810007

Osaka, 589-8511, Japan

Location

Investigator site 0810063

Suita, 565-0871, Japan

Location

Investigator site 0810036

tabashi City, 173-8606, Japan

Location

Investigator site 0810064

Tokushima, Japan

Location

Investigator site 0810060

Yokohama, Japan

Location

Investigator site 31

Riga, 1038, Latvia

Location

Investigator site 0310010

Amsterdam, 1105, Netherlands

Location

Investigator site 0310011

Rotterdam, 3015, Netherlands

Location

Investigator site 0480024

Krakow, 31-202, Poland

Location

Investigator site 0480018

Krakow, 31-539, Poland

Location

Investigator site 0480020

Lodz, 90-324, Poland

Location

Investigator Site 0480017

Lublin, 20-093, Poland

Location

Investigator site 0480022

Warsaw, 07-097, Poland

Location

Investigator site 040002

Brasov, 500299, Romania

Location

Investigator site 040001

Bucharest, 011302, Romania

Location

Investigator site 040004

Constanța, 900591, Romania

Location

Investigator site 040003

Timișoara, 300723, Romania

Location

Investigator site 070017

Kazan', 420021, Russia

Location

Investigator site 0070023

Kazan', 420097, Russia

Location

Investigator site 0070020

Moscow, 117186, Russia

Location

Investigator site 70021

Moscow, 117186, Russia

Location

Investigator site 0070019

Rostov-on-Don, 344022, Russia

Location

Investigator site 0070014

Saint Petersburg, 194354, Russia

Location

Investigator site 0070021

Saransk, 430032, Russia

Location

Investigator site 3810001

Belgrade, 11000, Serbia

Location

Investigator site 3810003

Belgrade, 11000, Serbia

Location

Investigator site 3810004

Kragujevac, Serbia

Location

Investigator site 0340021

Badalona, 08035, Spain

Location

Investigator site 0340038

Barcelona, Spain

Location

Investigator site 0340018

Madrid, 28040, Spain

Location

Investigator site 8860013

Tainan, 701, Taiwan

Location

Investigator site 8860012

Taipei, 116, Taiwan

Location

Investigator site 8860016

Taipei, 116, Taiwan

Location

Investigator site 8860017

Taoyuan District, 333, Taiwan

Location

Investigator site 900025

Bursa, 16059, Turkey (Türkiye)

Location

Investigator site 900021

Izmir, Turkey (Türkiye)

Location

Investigator site 900022

Samsun, 55239, Turkey (Türkiye)

Location

Investigator Site 3800012

Dnipro, 49069, Ukraine

Location

Investigator Site 3800010

Ivano-Frankivsk, 76008, Ukraine

Location

Investigator site 3100013

Kyiv, 02000, Ukraine

Location

Investigator site 3800008

Lutsk, 43024, Ukraine

Location

Investigator site 3800011

Zaporizhzhya, 69068, Ukraine

Location

Investigator site 440026

London, United Kingdom

Location

Investigator site 0440016

Oxford, United Kingdom

Location

Investigator site 0440018

Sheffield, United Kingdom

Location

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Interventions

efgartigimod alfa

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2020
• First Posted: February 21, 2020
• Study Start: September 18, 2020
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): April 30, 2027
• Last Updated: June 29, 2025

Record last verified: 2025-06

Locations

ES (3); UA (5); FR (9); GB (3); IL (1); IT (9); RU (7); AU (3); TU (3); SE (3); DE (6); US (25); DK (3); JP (18); TW (4); GE (5); LA (1); NL (2); PL (5); RO (4); BU (4); CN (19); BE (4); CZ (1)