A Study to Assess the 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)

NCT04281472 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: ARGX-113-18022019-003076-39
• Study Type: interventional
• Target: 322
• Locations: 25 countries
• Sites: 216

Brief Summary

This is a Phase 2 study to evaluate the safety and efficacy of the subcutaneous formulation of efgartigimod in adults with CIDP.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Outcome Measures

Primary Outcomes (2)

• Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI)

  Up to 12 weeks during the open-label stage A

• Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline

  Up to 48 weeks during the randomized placebo-controlled stage B

Secondary Outcomes (24)

• Stage A: Time to Initial Confirmed Evidence of Clinical Improvement (ECI)

  Up to 12 weeks during the open-label stage A

• Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Adjusted INCAT Score

  Up to 12 weeks during the open-label stage A

• Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Medical Research Council (MRC) Sum Score

  Up to 12 weeks during the open-label stage A

• Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in I-RODS Disability Scores

  Up to 12 weeks during the open-label stage A

• Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in TUG Score

  Up to 12 weeks during the open-label stage A

Study Arms (2)

efgartigimod PH20 SC

EXPERIMENTAL

patients receiving efgartigimod PH20 SC in both stage A as stage B

Biological: efgartigimod PH20 SC in stage B

Placebo

PLACEBO COMPARATOR

patients receiving efgartigimod PH20 SC during stage A and receiving placebo in stage B

Biological: efgartigimod PH20 SC in stage B; Other: placebo in stage B

Interventions

efgartigimod PH20 SC in stage B BIOLOGICAL

Stage A: efgartigimod PH20 SC, Stage B: efgartigimod PH20 SC

Placebo; efgartigimod PH20 SC

placebo in stage B OTHER

Stage A: N/A, stage B: placebo

Placebo

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 (include 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)
• Male or female patient aged 18 years or older, at the time of signing the informed consent.
• Diagnosed with probable or definite CIDP according to criteria of the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS 2010), progressing or relapsing forms.
• CIDP Disease Activity Status (CDAS) score ≥2 at screening.
• INCAT score ≥2 at the first run-in visit (for patients entering run-in) or stage A baseline (for treatment-naïve patients with documented evidence for worsening on the total adjusted INCAT disability score within 3 months prior to screening). Patients with an INCAT score of 2 at trial entry must have this score exclusively from the leg disability score; for patients with an INCAT score of ≥3 at trial entry, there are no specific requirements for arm or leg scores.
• Fulfilling any of the following treatment conditions:
• Currently treated with pulsed corticosteroids, oral corticosteroids equivalent to prednisolone/prednisone ≤10mg/day, and/or IVIg or SCIg, if this treatment has been started within the last 5 years before screening, and the patient is willing to discontinue this treatment at the first run-in visit; or
• Without previous treatment (treatment-naive); or
• Treatment with corticosteroids and/or IVIg or SCIg discontinued at least 6 months prior to screening Note: Patients not treated with monthly or daily corticosteroids, IVIg or SCIg for at least 6 months prior to screening are considered as equal to treatment-naïve patients.
• Women of childbearing potential who have a negative pregnancy test at screening and a negative urine pregnancy test up to Stage A baseline.
• Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last dose of IMP

You may not qualify if:

• Pure sensory atypical CIDP (EFNS/PNS definition).
• Polyneuropathy of other causes, including the following: Multifocal motor neuropathy; Monoclonal gammopathy of uncertain significance with anti-myelin associated, glycoprotein immunoglobulin M (IgM) antibodies; Hereditary demyelinating neuropathy; Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes; Lumbosacral radiculoplexus neuropathy; Polyneuropathy most likely due to diabetes mellitus; Polyneuropathy most likely due to systemic illnesses; Drug- or toxin-induced polyneuropathy.
• Any other disease that could better explain the patient's signs and symptoms.
• Any history of myelopathy or evidence of central demyelination.
• Current or past history (within 12 months of screening) of alcohol, drug or medication abuse.
• Severe psychiatric disorder (such as severe depression, psychosis, bipolar disorder), history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the patient or could affect adherence with the trial protocol.
• Patients with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV): serologic panel test results indicative of an active (acute or chronic) infection; Active Hepatitis C Virus (HCV): serology positive for HCV-Ab; Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count ≤200 cells/mm3.
• Total IgG level \<6 g/L at screening.
• Treatment with the following: Within 3 months (or 5 half-lives of the drug, whichever is longer) before screening: plasma exchange or immunoadsorption, any concomitant Fc-containing therapeutic agents or other biological, or any other investigational product; Within 6 months before screening: rituximab, alemtuzumab, any other monoclonal antibody, cyclophosphamide, interferon, tumor necrosis factor-alpha inhibitors, fingolimod, methotrexate, azathioprine, mycophenolate, any other immunomodulating or immunosuppressive medications, and oral daily corticosteroids \>10 mg/day. Note: Patients using IVIg, SCIg, pulsed corticosteroids, and oral daily corticosteroids ≤10 mg/day can be included.
• Patients who (intend to) use prohibited medications and therapies (see protocol) during the trial.
• Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after last IMP administration.
• Patients with any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of CIDP.
• Patients who have a history of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first IMP administration. Patients with the following cancer can be included anytime: Adequately treated basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, or Incidental histological finding of Prostate cancer (TNM \[tumor, nodes, and metastases classification\] stage T1a or T1b).
• Patients who previously participated in a trial with efgartigimod and have received at least one administration of IMP.
• Patients with known medical history of hypersensitivity to any of the ingredients of IMP.

Sponsors & Collaborators

• argenx: lead

Study Sites (216)

Investigator site 0010065

Birmingham, Alabama, 35233-2110, United States

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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 0010004

Orange, California, 92868, United States

Location

Investigator site 0010190

Pomona, California, 91767-2009, United States

Location

Investigator site 0010160

Rancho Mirage, California, 92270, United States

Location

Investigator site 0010071

San Francisco, California, 94109, United States

Location

Investigator site 0010057

Centennial, Colorado, 80112, United States

Location

Investigator site 0010026

New Haven, Connecticut, 06520, 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 0010050

Orlando, Florida, 32806, United States

Location

Investigator site 0010172

Ormond Beach, Florida, 32174-3102, United States

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Investigator site 0010006

Tampa, Florida, 33612, United States

Location

Investigator site 0010125

Augusta, Georgia, 30912-3125, United States

Location

Investigator site 0010011

Iowa City, Iowa, 52242, United States

Location

Investigator site 0010015

Fairway, Kansas, 66205, United States

Location

Investigator site 0010147

Lexington, Kentucky, 40536, United States

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Investigator site 0010014

Detroit, Michigan, 48201, United States

Location

Investigator site 0010063

East Lansing, Michigan, 48824, United States

Location

Investigator site 0010052

Minneapolis, Minnesota, 55455-4800, United States

Location

Investigator site 0010028

Columbia, Missouri, 65212, United States

Location

Investigator site 0010070

New Brunswick, New Jersey, 08550, United States

Location

Investigator site 0010069

New York, New York, 06511, United States

Location

Investigator site 0010168

New York, New York, 10001, United States

Location

Investigator site 0010191

New York, New York, 10021, United States

Location

Investigator site 0010074

New York, New York, 10032, United States

Location

Investigator site 0010075

Patchogue, New York, 11772, United States

Location

Investigator site 0010003

Chapel Hill, North Carolina, 27517, United States

Location

Investigator site 0010077

Durham, North Carolina, 27710, United States

Location

Investigator site 0010051

Cincinnati, Ohio, 45267-0525, United States

Location

Investigator site 0010064

Columbus, Ohio, 43210, United States

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Investigator site 0010047

Philadelphia, Pennsylvania, 15213, United States

Location

Investigator site 0010007

Philadelphia, Pennsylvania, 19104, United States

Location

Investigator site 0010067

Pittsburgh, Pennsylvania, 15123, United States

Location

Investigator site 0010066

Austin, Texas, 78756, United States

Location

Investigator site 0010026

Houston, Texas, 77055-7421, United States

Location

Investigator site 0010009

San Antonio, Texas, 78229, United States

Location

Investigator site 0010076

Burlington, Vermont, 05401, United States

Location

Investigator site 0010007

Charlottesville, Virginia, 22903, United States

Location

Investigator site 0010061

Richmond, Virginia, 23298, United States

Location

Investigator site 0430009

Graz, 8036, Austria

Location

Investigator site 0430007

Innsbruck, 6020, Austria

Location

Investigator site 0430008

Linz, 4021, Austria

Location

Investigator site 0430006

Salzburg, 5020, Austria

Location

Investigator site 0430005

Vienna, 1090, Austria

Location

Investigator site 0320017

Brussels, 1090, Belgium

Location

Investigator site 0320019

Brussels, 1090, Belgium

Location

Investigator site 0320016

Edegem, 2650, Belgium

Location

Investigator site 0320009

Leuven, 3000, Belgium

Location

Investigator site 0320024

Liège, 4000, Belgium

Location

Investigator site 0320022

Woluwe-Saint-Lambert, 1200, Belgium

Location

Investigator site 3590007

Pleven, 5800, Bulgaria

Location

Investigator site 3590008

Sofia, 1113, Bulgaria

Location

Investigator site 3590009

Sofia, 1431, Bulgaria

Location

Investigator site 3590005

Sofia, 1680, Bulgaria

Location

Investigator site 0860033

Beijing, 100053, China

Location

Investigator site 0860030

Changchun, China

Location

Investigator site 0860041

Changsha, 410008, China

Location

Investigator site 0860036

Chengdu, China

Location

Investigator site 0860049

Chifeng, 024000, China

Location

Investigator site 0860038

Fuzhou, 350001, China

Location

Investigator site 0860050

Guanzhou, 510120, China

Location

Investigator site 0860032

Guanzhou, 510515, China

Location

Investigator site 0860045

Guiyang, 550000, China

Location

Investigator site 0860046

Hangzhou, China

Location

Investigator site 0860035

Hanzhou, 310003, China

Location

Investigator site 0860031

Jinan, 2500012, China

Location

Investigator site 0860063

Jining, China

Location

Investigator site 0860044

Nanchang, 330088, China

Location

Investigator Site 0860040

Nanchang, China

Location

Investigator site 0860051

Nanchang, China

Location

Investigator site 0860043

Nanjing, 210001, China

Location

Investigator site 0860043

Nanjing, China

Location

Investigator site 0860047

Shanghai, 200090, China

Location

Investigator site 0860028

Shanghai, China

Location

Investigator site 0860052

Shanghai, China

Location

Investigator site 0860042

Taiyuan, 030001, China

Location

Investigator site 0860029

Wuhan, 430040, China

Location

Investigator site 0860034

Wuhan, 430060, China

Location

Investigator site 0860048

Xi'an, 710038, China

Location

Investigator site 0860037

Xi'an, 710075, China

Location

Investigator site 0860054

Xiangyang, 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

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Investigator site 0450003

Odense, 5000, Denmark

Location

Investigator site 0330034

Angers, 49033, France

Location

Investigator site 0330013

Bordeaux, 33076, France

Location

Investigator site 0330033

Clermont-Ferrand, 63003, France

Location

Investigator site 0330025

Garches, 92380, France

Location

Investigator site 0330023

Le Kremlin-Bicêtre, 94275, France

Location

Investigator site 0330024

Limoges, 87042, France

Location

Investigator site 0330022

Nantes, 44093, France

Location

Investigator site 0330021

Nice, 06202, France

Location

Investigator site 0330035

Paris, 75013, France

Location

Investigator site 0330020

Strasbourg, 67098, France

Location

Investigator site 9950020

Kutaisi, 4600, Georgia

Location

Investigator site 9950005

Tbilisi, 0112, Georgia

Location

Investigator Site 9950002

Tbilisi, Georgia

Location

Investigator Site 9950003

Tbilisi, Georgia

Location

Investigator Site 9950004

Tbilisi, Georgia

Location

Investigator site 0490018

Berlin, 10117, Germany

Location

Investigator site 0490017

Berlin, Germany

Location

Investigator site 0490044

Bochum, 37075, Germany

Location

Investigator site 0490013

Cologne, 50937, Germany

Location

Investigator site 0490045

Essen, 45147, Germany

Location

Investigator site 0490021

Göttingen, Germany

Location

Investigator site 0490014

Hanover, Germany

Location

Investigator site 0490016

Kiel, 24105, Germany

Location

Investigator site 0490020

Leipzig, Germany

Location

Investigator site 0490019

Potsdam, 14471, Germany

Location

Investigator site 0490015

Regensburg, 93053, Germany

Location

Investigator site 0360017

Budapest, 1121, Hungary

Location

Investigator site 0360018

Kistarcsa, 1121, Hungary

Location

Investigator site 9720006

Holon, 58100, Israel

Location

Investigator site 9720005

Ramat Gan, 52621, Israel

Location

Investigator site 9720004

Tel Aviv, 6423906, Israel

Location

Investigator site 0390022

Brescia, Italy

Location

Investigator site 0390029

Florence, Italy

Location

Investigator site 0390024

Genova, Italy

Location

Investigator site 0390027

Messina, 98125, Italy

Location

Investigator site 0390003

Milan, Italy

Location

Investigator site 0390026

Milan, Italy

Location

Investigator site 0390007

Napoli, 80131, Italy

Location

Investigator site 0390023

Pisa, Italy

Location

Investigator site 0390008

Roma, Italy

Location

Investigator site 0390028

Siena, Italy

Location

Investigator site 0390042

Torino, 10126, Italy

Location

Investigator site 0810035

Bunkyō City, 113-8582, Japan

Location

Investigator site 0810002

Chiba, Japan

Location

Investigator site 0810034

Chūōku, Japan

Location

Investigator site 0810030

Fuchū, 183-0042, Japan

Location

Investigator site 0810031

Fukuoka, 812-8582, Japan

Location

Investigator site 0810065

Ginowan, 901-214, Japan

Location

Investigator site 0810066

Hakodate, 041-0821, Japan

Location

Investigator site 0810058

Hiroshima, 730-0011, Japan

Location

Investigator site 0810029

Kawaguchi, Japan

Location

Investigator site 0810062

Kawasaki, 2016-0015, Japan

Location

Investigator site 0810026

Kodaira, Japan

Location

Investigator site 0810061

Kyoto, 616-8255, Japan

Location

Investigator site 0810027

Mibu, Japan

Location

Investigator site 0810032

Nagoya, Japan

Location

Investigator site 0810003

Osaka, 565-0871, Japan

Location

Investigator site 0810007

Osaka, Japan

Location

Investigator site 0810028

Sagamihara, Japan

Location

Investigator site 0810033

Sapporo, 0608638, Japan

Location

Investigator site 0810037

Shinjuku-Ku, 160-8582, Japan

Location

Investigator site 0810063

Suita, 565-0871, Japan

Location

Investigator site 0810036

tabashi City, 173-8606, Japan

Location

Investigator site 0810064

Tokushima, 770-0042, Japan

Location

Investigator site 0810060

Yokohama, 236-0004, Japan

Location

Investigator site 3710001

Riga, 1038, Latvia

Location

Investigator site 0310010

Amsterdam, 1105, Netherlands

Location

Investigator site 0310011

Rotterdam, Netherlands

Location

Investigator site 0480019

Bialystok, 15-402, Poland

Location

Investigator site 0480023

Katowice, 40-650, Poland

Location

Investigator site 0480017

Krakow, 30-539, Poland

Location

Investigator site 0480024

Krakow, 31-202, Poland

Location

Investigator site 0480020

Lodz, 90-324, Poland

Location

Investigator site 0480018

Lublin, 20-090, Poland

Location

Investigator site 0480022

Warsaw, Poland

Location

Investigation site 0400002

Brasov, 500299, Romania

Location

Investigator site 0400001

Bucharest, 011302, Romania

Location

Investigator site 0400004

Constanța, 900591, Romania

Location

Investigator site 0400003

Timișoara, 300723, Romania

Location

Investigator site 0070017

Kazan', 420021, Russia

Location

Investigator site 0070023

Kazan', 420097, Russia

Location

Investigator site 0070016

Moscow, 117186, Russia

Location

Investigator site 0070020

Moscow, 117186, Russia

Location

Investigator site 0070018

Perm, 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 0340020

Alicante, 03010, Spain

Location

Investigator site 0340021

Badalona, 08041, Spain

Location

Investigator site 0340038

Barcelona, Spain

Location

Investigator site 0340019

Córdoba, 14011, Spain

Location

Investigator site 0340017

Madrid, 28007, Spain

Location

Investigator site 0340018

Madrid, 28040, Spain

Location

Investigator site 0340016

Seville, 41013, Spain

Location

Investigator site 8860014

Kaohsiung City, Taiwan

Location

Investigator site 8860015

Taichung, Taiwan

Location

Investigator site 8860013

Tainan, Taiwan

Location

Investigator site 8860011

Taipei, Taiwan

Location

Investigator site 8860012

Taipei, Taiwan

Location

Investigator site 8860016

Taipei, Taiwan

Location

Investigator site 8860017

Taoyuan District, Taiwan

Location

Investigator site 0900025

Bursa, Turkey (Türkiye)

Location

Investigator site 0900023

Istanbul, Turkey (Türkiye)

Location

Investigator site 0900021

Izmir, Turkey (Türkiye)

Location

Investigator site 0900022

Samsun, Turkey (Türkiye)

Location

Investigator site 0900024

Sarıçam, Turkey (Türkiye)

Location

Investigator site 3800012

Dnipro, 49069, Ukraine

Location

Investigator site 3800014

Dnipro, 79044, Ukraine

Location

Investigator site 3800010

Ivano-Frankivsk, 76008, Ukraine

Location

Investigator site 3800015

Kharkiv, 61058, Ukraine

Location

Investigator site 3800013

Kyiv, 21000, Ukraine

Location

Investigator site 380008

Lutsk, 43024, Ukraine

Location

Investigator site 380009

Vinnytsia, 21009, Ukraine

Location

Investigator site 3800015

Vinnytsia, Ukraine

Location

Investigator site 3800011

Zaporizhzhya, 69068, Ukraine

Location

Investigator site 0440017

Glasgow, United Kingdom

Location

Investigator site 0440015

Inverness, United Kingdom

Location

Investigator site 0440026

London, United Kingdom

Location

Investigator site 0440016

Oxford, United Kingdom

Location

Investigator site 0440018

Sheffield, United Kingdom

Location

Investigator site 0440019

Stoke-on-Trent, United Kingdom

Location

Investigator site 0440028

Tooting, United Kingdom

Location

Related Publications (1)

• Allen JA, Lin J, Basta I, Dysgaard T, Eggers C, Guptill JT, Gwathmey KG, Hewamadduma C, Hofman E, Hussain YM, Kuwabara S, Le Masson G, Leypoldt F, Chang T, Lipowska M, Lowe M, Lauria G, Querol L, Simu MA, Suresh N, Tse A, Ulrichts P, Van Hoorick B, Yamasaki R, Lewis RA, van Doorn PA; ADHERE Study Group. Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial. Lancet Neurol. 2024 Oct;23(10):1013-1024. doi: 10.1016/S1474-4422(24)00309-0.

  PMID: 39304241 DERIVED

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

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

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 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2020
• First Posted: February 24, 2020
• Study Start: April 15, 2020
• Primary Completion: May 11, 2023
• Study Completion: May 11, 2023
• Last Updated: August 20, 2024
• Results First Posted: August 20, 2024

Record last verified: 2024-07

Locations

HU (2); FR (10); GE (5); LA (1); PL (7); SE (3); IL (3); TW (7); DK (3); TU (5); CZ (1); JP (23); US (45); RU (8); GB (7); NL (2); BU (4); AU (5); IT (11); RO (4); ES (7); UA (9); DE (11); CN (27); BE (6)