AzTrials
AzTrials

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase 2ADHERENCT04281472

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)

A Phase 2 study testing efgartigimod PH20 SC in stage B in people with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), run by argenx at 216 sites in 25 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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

Who is it for?
Ages 18 years and older
What is being tested?
efgartigimod PH20 SC in stage B
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
322 participants
Where
216 sitesin 25 countries
Study dates
15 Apr 2020to 11 May 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
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  • 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

Who cannot join

  • 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.
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  • 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 received a live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, sub-unit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.
  • 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.
  • Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or any other reason which could confound the results of the trial or put the patient at undue risk.

Trial locations and contacts

216 sites in 25 countries.

216 sites in 25 countries

United States45 sites

  • Investigator site 0010125

    Augusta, Georgia, 30912-3125

  • Investigator site 0010066

    Austin, Texas, 78756

  • Investigator site 0010065

    Birmingham, Alabama, 35233-2110

  • Investigator site 0010072

    Boca Raton, Florida, 33487

  • Investigator site 0010076

    Burlington, Vermont, 05401

  • Investigator Site 0010032

    Carlsbad, California, 92011

  • Investigator site 0010057

    Centennial, Colorado, 80112

  • Investigator site 0010003

    Chapel Hill, North Carolina, 27517

  • Investigator site 0010007

    Charlottesville, Virginia, 22903

  • Investigator site 0010051

    Cincinnati, Ohio, 45267-0525

  • Investigator site 0010028

    Columbia, Missouri, 65212

  • Investigator site 0010064

    Columbus, Ohio, 43210

  • Investigator site 0010144

    Coral Springs, Florida, 33067-4640

  • Investigator site 0010014

    Detroit, Michigan, 48201

  • Investigator site 0010077

    Durham, North Carolina, 27710

  • Investigator site 0010063

    East Lansing, Michigan, 48824

  • Investigator site 0010015

    Fairway, Kansas, 66205

  • Investigator site 0010026

    Houston, Texas, 77055-7421

  • Investigator site 0010011

    Iowa City, Iowa, 52242

  • Investigator site 0010023

    Jacksonville, Florida, 32209

  • Investigator site 0010147

    Lexington, Kentucky, 40536

  • Investigator Site 0010068

    Maitland, Florida, 32751

  • Investigator site 0010059

    Miami, Florida, 33136

  • Investigator site 0010052

    Minneapolis, Minnesota, 55455-4800

  • Investigator site 0010070

    New Brunswick, New Jersey, 08550

  • Investigator site 0010026

    New Haven, Connecticut, 06520

  • Investigator site 0010069

    New York, New York, 06511

  • Investigator site 0010168

    New York, New York, 10001

  • Investigator site 0010191

    New York, New York, 10021

  • Investigator site 0010074

    New York, New York, 10032

  • Investigator site 0010004

    Orange, California, 92868

  • Investigator site 0010050

    Orlando, Florida, 32806

  • Investigator site 0010172

    Ormond Beach, Florida, 32174-3102

  • Investigator site 0010075

    Patchogue, New York, 11772

  • Investigator site 0010047

    Philadelphia, Pennsylvania, 15213

  • Investigator site 0010007

    Philadelphia, Pennsylvania, 19104

  • Investigator site 0010013

    Phoenix, Arizona, 85018

  • Investigator site 0010067

    Pittsburgh, Pennsylvania, 15123

  • Investigator site 0010190

    Pomona, California, 91767-2009

  • Investigator site 0010160

    Rancho Mirage, California, 92270

  • Investigator site 0010061

    Richmond, Virginia, 23298

  • Investigator site 0010009

    San Antonio, Texas, 78229

  • Investigator site 0010071

    San Francisco, California, 94109

  • Investigator site 0010055

    Scottsdale, Arizona, 85028

  • Investigator site 0010006

    Tampa, Florida, 33612

Other trials in United States

China27 sites

  • Investigator site 0860033

    Beijing, 100053

  • Investigator site 0860030

    Changchun

  • Investigator site 0860041

    Changsha, 410008

  • Investigator site 0860036

    Chengdu

  • Investigator site 0860049

    Chifeng, 024000

  • Investigator site 0860038

    Fuzhou, 350001

  • Investigator site 0860050

    Guanzhou, 510120

  • Investigator site 0860032

    Guanzhou, 510515

  • Investigator site 0860045

    Guiyang, 550000

  • Investigator site 0860046

    Hangzhou

  • Investigator site 0860035

    Hanzhou, 310003

  • Investigator site 0860031

    Jinan, 2500012

  • Investigator site 0860063

    Jining

  • Investigator site 0860044

    Nanchang, 330088

  • Investigator Site 0860040

    Nanchang

  • Investigator site 0860051

    Nanchang

  • Investigator site 0860043

    Nanjing, 210001

  • Investigator site 0860043

    Nanjing

  • Investigator site 0860047

    Shanghai, 200090

  • Investigator site 0860028

    Shanghai

  • Investigator site 0860052

    Shanghai

  • Investigator site 0860042

    Taiyuan, 030001

  • Investigator site 0860029

    Wuhan, 430040

  • Investigator site 0860034

    Wuhan, 430060

  • Investigator site 0860048

    Xi'an, 710038

  • Investigator site 0860037

    Xi'an, 710075

  • Investigator site 0860054

    Xiangyang, 712000

Other trials in China

Japan23 sites

  • Investigator site 0810035

    Bunkyō City, 113-8582

  • Investigator site 0810002

    Chiba

  • Investigator site 0810034

    Chūōku

  • Investigator site 0810030

    Fuchū, 183-0042

  • Investigator site 0810031

    Fukuoka, 812-8582

  • Investigator site 0810065

    Ginowan, 901-214

  • Investigator site 0810066

    Hakodate, 041-0821

  • Investigator site 0810058

    Hiroshima, 730-0011

  • Investigator site 0810029

    Kawaguchi

  • Investigator site 0810062

    Kawasaki, 2016-0015

  • Investigator site 0810026

    Kodaira

  • Investigator site 0810061

    Kyoto, 616-8255

  • Investigator site 0810027

    Mibu

  • Investigator site 0810032

    Nagoya

  • Investigator site 0810003

    Osaka, 565-0871

  • Investigator site 0810007

    Osaka

  • Investigator site 0810028

    Sagamihara

  • Investigator site 0810033

    Sapporo, 0608638

  • Investigator site 0810037

    Shinjuku-Ku, 160-8582

  • Investigator site 0810063

    Suita, 565-0871

  • Investigator site 0810036

    tabashi City, 173-8606

  • Investigator site 0810064

    Tokushima, 770-0042

  • Investigator site 0810060

    Yokohama, 236-0004

Other trials in Japan

Germany11 sites

  • Investigator site 0490018

    Berlin, 10117

  • Investigator site 0490017

    Berlin

  • Investigator site 0490044

    Bochum, 37075

  • Investigator site 0490013

    Cologne, 50937

  • Investigator site 0490045

    Essen, 45147

  • Investigator site 0490021

    Göttingen

  • Investigator site 0490014

    Hanover

  • Investigator site 0490016

    Kiel, 24105

  • Investigator site 0490020

    Leipzig

  • Investigator site 0490019

    Potsdam, 14471

  • Investigator site 0490015

    Regensburg, 93053

Other trials in Germany

Italy11 sites

  • Investigator site 0390022

    Brescia

  • Investigator site 0390029

    Florence

  • Investigator site 0390024

    Genova

  • Investigator site 0390027

    Messina, 98125

  • Investigator site 0390003

    Milan

  • Investigator site 0390026

    Milan

  • Investigator site 0390007

    Naples, 80131

  • Investigator site 0390023

    Pisa

  • Investigator site 0390008

    Roma

  • Investigator site 0390028

    Siena

  • Investigator site 0390042

    Torino, 10126

Other trials in Italy

France10 sites

  • Investigator site 0330034

    Angers, 49033

  • Investigator site 0330013

    Bordeaux, 33076

  • Investigator site 0330033

    Clermont-Ferrand, 63003

  • Investigator site 0330025

    Garches, 92380

  • Investigator site 0330023

    Le Kremlin-Bicêtre, 94275

  • Investigator site 0330024

    Limoges, 87042

  • Investigator site 0330022

    Nantes, 44093

  • Investigator site 0330021

    Nice, 06202

  • Investigator site 0330035

    Paris, 75013

  • Investigator site 0330020

    Strasbourg, 67098

Other trials in France

Ukraine9 sites

  • Investigator site 3800012

    Dnipro, 49069

  • Investigator site 3800014

    Dnipro, 79044

  • Investigator site 3800010

    Ivano-Frankivsk, 76008

  • Investigator site 3800015

    Kharkiv, 61058

  • Investigator site 3800013

    Kyiv, 21000

  • Investigator site 380008

    Lutsk, 43024

  • Investigator site 380009

    Vinnytsia, 21009

  • Investigator site 3800015

    Vinnytsia

  • Investigator site 3800011

    Zaporizhzhya, 69068

Other trials in Ukraine

Russia8 sites

  • Investigator site 0070017

    Kazan', 420021

  • Investigator site 0070023

    Kazan', 420097

  • Investigator site 0070016

    Moscow, 117186

  • Investigator site 0070020

    Moscow, 117186

  • Investigator site 0070018

    Perm

  • Investigator site 0070019

    Rostov-on-Don, 344022

  • Investigator site 0070014

    Saint Petersburg, 194354

  • Investigator site 0070021

    Saransk, 430032

Other trials in Russia

Poland7 sites

  • Investigator site 0480019

    Bialystok, 15-402

  • Investigator site 0480023

    Katowice, 40-650

  • Investigator site 0480017

    Krakow, 30-539

  • Investigator site 0480024

    Krakow, 31-202

  • Investigator site 0480020

    Lodz, 90-324

  • Investigator site 0480018

    Lublin, 20-090

  • Investigator site 0480022

    Warsaw

Other trials in Poland

Spain7 sites

  • Investigator site 0340020

    Alicante, 03010

  • Investigator site 0340021

    Badalona, 08041

  • Investigator site 0340038

    Barcelona

  • Investigator site 0340019

    Córdoba, 14011

  • Investigator site 0340017

    Madrid, 28007

  • Investigator site 0340018

    Madrid, 28040

  • Investigator site 0340016

    Seville, 41013

Other trials in Spain

Taiwan7 sites

  • Investigator site 8860014

    Kaohsiung City

  • Investigator site 8860015

    Taichung

  • Investigator site 8860013

    Tainan

  • Investigator site 8860011

    Taipei

  • Investigator site 8860012

    Taipei

  • Investigator site 8860016

    Taipei

  • Investigator site 8860017

    Taoyuan

Other trials in Taiwan

United Kingdom7 sites

  • Investigator site 0440017

    Glasgow

  • Investigator site 0440015

    Inverness

  • Investigator site 0440026

    London

  • Investigator site 0440016

    Oxford

  • Investigator site 0440018

    Sheffield

  • Investigator site 0440019

    Stoke-on-Trent

  • Investigator site 0440028

    Tooting

Other trials in United Kingdom

Belgium6 sites

  • Investigator site 0320017

    Brussels, 1090

  • Investigator site 0320019

    Brussels, 1090

  • Investigator site 0320016

    Edegem, 2650

  • Investigator site 0320009

    Leuven, 3000

  • Investigator site 0320024

    Liège, 4000

  • Investigator site 0320022

    Woluwe-Saint-Lambert, 1200

Other trials in Belgium

Austria5 sites

  • Investigator site 0430009

    Graz, 8036

  • Investigator site 0430007

    Innsbruck, 6020

  • Investigator site 0430008

    Linz, 4021

  • Investigator site 0430006

    Salzburg, 5020

  • Investigator site 0430005

    Vienna, 1090

Other trials in Austria

Georgia5 sites

  • Investigator site 9950020

    Kutaisi, 4600

  • Investigator site 9950005

    Tbilisi, 0112

  • Investigator Site 9950002

    Tbilisi

  • Investigator Site 9950003

    Tbilisi

  • Investigator Site 9950004

    Tbilisi

Other trials in Georgia

Turkey (Türkiye)5 sites

  • Investigator site 0900025

    Bursa

  • Investigator site 0900023

    Istanbul

  • Investigator site 0900021

    Izmir

  • Investigator site 0900022

    Samsun

  • Investigator site 0900024

    Sarıçam

Other trials in Turkey (Türkiye)

Bulgaria4 sites

  • Investigator site 3590007

    Pleven, 5800

  • Investigator site 3590008

    Sofia, 1113

  • Investigator site 3590009

    Sofia, 1431

  • Investigator site 3590005

    Sofia, 1680

Other trials in Bulgaria

Romania4 sites

  • Investigation site 0400002

    Brasov, 500299

  • Investigator site 0400001

    Bucharest, 011302

  • Investigator site 0400004

    Constanța, 900591

  • Investigator site 0400003

    Timișoara, 300723

Other trials in Romania

Denmark3 sites

  • Investigator site 0450002

    Aarhus, 8200

  • Investigator site 0450001

    Copenhagen, 2100

  • Investigator site 0450003

    Odense, 5000

Other trials in Denmark

Israel3 sites

  • Investigator site 9720006

    Holon, 58100

  • Investigator site 9720005

    Ramat Gan, 52621

  • Investigator site 9720004

    Tel Aviv, 6423906

Other trials in Israel

Serbia3 sites

  • Investigator site 3810001

    Belgrade, 11000

  • Investigator site 3810003

    Belgrade, 11000

  • Investigator site 3810004

    Kragujevac

Other trials in Serbia

Hungary2 sites

  • Investigator site 0360017

    Budapest, 1121

  • Investigator site 0360018

    Kistarcsa, 1121

Other trials in Hungary

Netherlands2 sites

  • Investigator site 0310010

    Amsterdam, 1105

  • Investigator site 0310011

    Rotterdam

Other trials in Netherlands

Czechia1 site

  • Investigator site 4200010

    Hradec Králové, 500-03

Other trials in Czechia

Latvia1 site

  • Investigator site 3710001

    Riga, 1038

Other trials in Latvia

About this study

Official title: A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

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

Being tested
efgartigimod PH20 SC in stage B, placebo in stage B
Medical subject headings
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • efgartigimod PH20 SC in stage BBiological

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

  • placebo in stage BOther

    Stage A: N/A, stage B: placebo

Study groups in NCT04281472
Study groupRoleWhat they receive
efgartigimod PH20 SCExperimentalpatients receiving efgartigimod PH20 SC in both stage A as stage B
Receives: Biological: efgartigimod PH20 SC in stage B
PlaceboPlacebo comparatorpatients receiving efgartigimod PH20 SC during stage A and receiving placebo in stage B
Receives: Biological: efgartigimod PH20 SC in stage B; Other: placebo in stage B

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Up to 12 weeks during the open-label stage A

  2. Stage B: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline

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

Secondary outcomes (24)

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

    Measured: Up to 12 weeks during the open-label stage A

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

    Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability.

    Measured: Up to 12 weeks during the open-label stage A

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

    The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness.

    Measured: Up to 12 weeks during the open-label stage A

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

    The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.

    Measured: Up to 12 weeks during the open-label stage A

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

    The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.

    Measured: Up to 12 weeks during the open-label stage A

  6. Stage A: Change From Stage A Baseline to Last Assessment in Stage A, in Mean Grip Strength

    This is measured with a handheld device called a vigometer

    Measured: Up to 12 weeks during the open-label stage A

  7. Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events

    Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)

    Measured: Up to 12 weeks during the open-label stage A

  8. Stage A: Pre-dosing Efgartigimod Serum Concentrations Over Time

    Measured: Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI))

  9. Stage A: Percent Changes From Stage A Baseline of Serum IgG Levels Over Time

    Measured: Up to 13 weeks during the open-label stage A (12 weeks + optional 1 additional week to confirm evidence of clinical improvement (ECI))

  10. Stage A: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20

    Measured: Up to 12 weeks during the open-label stage A

  11. Stage A: Changes From Stage A Baseline to Last Assessment in Stage A, in EQ-5D-5L Visual Analog Scale (VAS) Over Time

    Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.

    Measured: Up to 12 weeks during the open-label stage A

  12. Stage B: Time to CIDP Disease Progression

    Time to chronic inflammatory demyelinating polyneuropathy (CIDP) disease progression is defined by the time from first dose of double-blind IMP to the first I-RODS score decrease ≥4 points compared to Stage B baseline using the centile metric.

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

  13. Stage B: Number of Participants With Improved Functional Level Compared to Stage B Baseline

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

  14. Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Adjusted INCAT Score

    Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability.

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

  15. Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in MRC Sum Score

    The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness.

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

  16. Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in 24-item I-RODS Disability Score

    The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.

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

  17. Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in TUG Score

    The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.

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

  18. Stage B: Change From Stage B Baseline to Last Assessment in Stage B, in Mean Grip Strength

    This is measured with a handheld device called a vigometer

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

  19. Stage B: Time to 10% Decrease in the 24-item I-RODS

    The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability.

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

  20. Stage B: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events

    Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)

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

  21. Stage B: Pre-dosing Efgartigimod Serum Concentrations Over Time

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

  22. Stage B: Percent Changes of Serum IgG Levels Over Time

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

  23. Stage B: Number of Participants With Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20

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

  24. Stage B: Changes From Stage B Baseline to Last Assessment in Stage B, in EQ-5D-5L Visual Analog Scale (VAS) Over Time

    Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Feb 2020
  2. Study start15 Apr 2020
  3. Primary completion11 May 2023
  4. Study completion11 May 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
322 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04281472
Sponsor's protocol ID
ARGX-113-1802
Other IDs
2019-003076-39, 2019
Acronym
ADHERE
Status last verified
Jul 2024
First submitted
20 Feb 2020
Last updated
20 Aug 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • 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.

    PubMed 39304241

  • Fisse AL, Schafer E, Hieke A, Schroder M, Klimas R, Brunger J, Huckemann S, Gruter T, Sgodzai M, Schneider-Gold C, Gold R, Nguyen HP, Pitarokoili K, Motte J, Arning L. Association of the neonatal Fc receptor promoter variable number of tandem repeat polymorphism with immunoglobulin response in patients with chronic inflammatory demyelinating polyneuropathy. Eur J Neurol. 2024 Apr;31(4):e16205. doi: 10.1111/ene.16205. Epub 2024 Jan 11.

    PubMed 38205888

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing efgartigimod PH20 SC in stage B in people with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), run by argenx at 216 sites in 25 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 216 sites in United States, China, Japan, Germany, Italy and 20 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Stage A: Percentage of Participants With Confirmed Evidence of Clinical Improvement(ECI) (time frame: Up to 12 weeks during the open-label stage A). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04281472, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Aug 2024.

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