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) (ADHERE)

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.

Study Overview

• Status: Completed
• Conditions: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
• Intervention / Treatment: Biological: efgartigimod PH20 SC in stage B; Other: placebo in stage B

• Study Type: Interventional
• Enrollment (Actual): 322
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Sabine Coppieters, MD; Phone Number: +1 857-350-4834; Email: ClinicalTrials@argenx.com

Study Locations

• Austria
  • Graz, Austria, 8036
    • Investigator site 0430009
  • Innsbruck, Austria, 6020
    • Investigator site 0430007
  • Linz, Austria, 4021
    • Investigator site 0430008
  • Salzburg, Austria, 5020
    • Investigator site 0430006
  • Wien, Austria, 1090
    • Investigator site 0430005
• Belgium
  • Brussel, Belgium, 1090
    • Investigator site 0320017
  • Brussel, Belgium, 1090
    • Investigator site 0320019
  • Edegem, Belgium, 2650
    • Investigator site 0320016
  • Leuven, Belgium, 3000
    • Investigator site 0320009
  • Liège, Belgium, 4000
    • Investigator site 0320024
  • Woluwe-Saint-Lambert, Belgium, 1200
    • Investigator site 0320022
• Bulgaria
  • Pleven, Bulgaria, 5800
    • Investigator site 3590007
  • Sofia, Bulgaria, 1113
    • Investigator site 3590008
  • Sofia, Bulgaria, 1431
    • Investigator site 3590009
  • Sofia, Bulgaria, 1680
    • Investigator site 3590005
• China
  • Beijing, China, 100053
    • Investigator site 0860033
  • Changchun, China
    • Investigator site 0860030
  • Changsha, China, 410008
    • Investigator site 0860041
  • Chengdu, China
    • Investigator site 0860036
  • Chifeng, China, 024000
    • Investigator site 0860049
  • Fuzhou, China, 350001
    • Investigator site 0860038
  • Guanzhou, China, 510120
    • Investigator site 0860050
  • Guanzhou, China, 510515
    • Investigator site 0860032
  • Guiyang, China, 550000
    • Investigator site 0860045
  • Hangzhou, China
    • Investigator site 0860046
  • Hanzhou, China, 310003
    • Investigator site 0860035
  • Jinan, China, 2500012
    • Investigator site 0860031
  • Jining, China
    • Investigator site 0860063
  • Nanchang, China, 330088
    • Investigator site 0860044
  • Nanchang, China
    • Investigator Site 0860040
  • Nanchang, China
    • Investigator site 0860051
  • Nanjing, China, 210001
    • Investigator site 0860043
  • Nanjing, China
    • Investigator site 0860043
  • Shangai, China
    • Investigator site 0860028
  • Shanghai, China, 200090
    • Investigator site 0860047
  • Shanghai, China
    • Investigator site 0860052
  • Taiyuan, China, 030001
    • Investigator site 0860042
  • Wuhan, China, 430040
    • Investigator site 0860029
  • Wuhan, China, 430060
    • Investigator site 0860034
  • Xi'an, China, 710038
    • Investigator site 0860048
  • Xi'an, China, 710075
    • Investigator site 0860037
  • Xiangyang, China, 712000
    • Investigator site 0860054
• Czechia
  • Hradec Králové, Czechia, 500-03
    • Investigator site 4200010
• Denmark
  • Aarhus, Denmark, 8200
    • Investigator site 0450002
  • Copenhagen, Denmark, 2100
    • Investigator site 0450001
  • Odense, Denmark, 5000
    • Investigator site 0450003
• France
  • Angers, France, 49033
    • Investigator site 0330034
  • Bordeaux, France, 33076
    • Investigator site 0330013
  • Clermont-Ferrand, France, 63003
    • Investigator site 0330033
  • Garches, France, 92380
    • Investigator site 0330025
  • Le Kremlin-Bicêtre, France, 94275
    • Investigator site 0330023
  • Limoges, France, 87042
    • Investigator site 0330024
  • Nantes, France, 44093
    • Investigator site 0330022
  • Nice, France, 06202
    • Investigator site 0330021
  • Paris, France, 75013
    • Investigator site 0330035
  • Strasbourg, France, 67098
    • Investigator site 0330020
• Georgia
  • Kutaisi, Georgia, 4600
    • Investigator site 9950020
  • Tbilisi, Georgia, 0112
    • Investigator site 9950005
  • Tbilisi, Georgia
    • Investigator Site 9950002
  • Tbilisi, Georgia
    • Investigator Site 9950003
  • Tbilisi, Georgia
    • Investigator Site 9950004
• Germany
  • Berlin, Germany, 10117
    • Investigator site 0490018
  • Berlin, Germany
    • Investigator site 0490017
  • Bochum, Germany, 37075
    • Investigator site 0490044
  • Essen, Germany, 45147
    • Investigator site 0490045
  • Göttingen, Germany
    • Investigator site 0490021
  • Hannover, Germany
    • Investigator site 0490014
  • Kiel, Germany, 24105
    • Investigator site 0490016
  • Köln, Germany, 50937
    • Investigator site 0490013
  • Leipzig, Germany
    • Investigator site 0490020
  • Potsdam, Germany, 14471
    • Investigator site 0490019
  • Regensburg, Germany, 93053
    • Investigator site 0490015
• Hungary
  • Budapest, Hungary, 1121
    • Investigator site 0360017
  • Kistarcsa, Hungary, 1121
    • Investigator site 0360018
• Israel
  • H̱olon, Israel, 58100
    • Investigator site 9720006
  • Ramat Gan, Israel, 52621
    • Investigator site 9720005
  • Tel Aviv, Israel, 6423906
    • Investigator site 9720004
• Italy
  • Brescia, Italy
    • Investigator site 0390022
  • Firenze, Italy
    • Investigator site 0390029
  • Genova, Italy
    • Investigator site 0390024
  • Messina, Italy, 98125
    • Investigator site 0390027
  • Milano, Italy
    • Investigator site 0390003
  • Milano, Italy
    • Investigator site 0390026
  • Napoli, Italy, 80131
    • Investigator site 0390007
  • Pisa, Italy
    • Investigator site 0390023
  • Roma, Italy
    • Investigator site 0390008
  • Siena, Italy
    • Investigator site 0390028
  • Torino, Italy, 10126
    • Investigator site 0390042
• Japan
  • Bunkyō-Ku, Japan, 113-8582
    • Investigator site 0810035
  • Chiba, Japan
    • Investigator site 0810002
  • Chuo Ku, Japan
    • Investigator site 0810034
  • Fuchū, Japan, 183-0042
    • Investigator site 0810030
  • Fukuoka, Japan, 812-8582
    • Investigator site 0810031
  • Ginowan, Japan, 901-214
    • Investigator site 0810065
  • Hakodate, Japan, 041-0821
    • Investigator site 0810066
  • Hiroshima, Japan, 730-0011
    • Investigator site 0810058
  • Itabashi, Japan, 173-8606
    • Investigator site 0810036
  • Kawaguchi, Japan
    • Investigator site 0810029
  • Kawasaki, Japan, 2016-0015
    • Investigator site 0810062
  • Kodaira, Japan
    • Investigator site 0810026
  • Kyoto, Japan, 616-8255
    • Investigator site 0810061
  • Mibu, Japan
    • Investigator site 0810027
  • Nagoya, Japan
    • Investigator site 0810032
  • Osaka, Japan, 565-0871
    • Investigator site 0810003
  • Osaka, Japan
    • Investigator site 0810007
  • Sagamihara, Japan
    • Investigator site 0810028
  • Sapporo, Japan, 0608638
    • Investigator site 0810033
  • Shinjuku-Ku, Japan, 160-8582
    • Investigator site 0810037
  • Suita, Japan, 565-0871
    • Investigator site 0810063
  • Tokushima, Japan, 770-0042
    • Investigator site 0810064
  • Yokohama, Japan, 236-0004
    • Investigator site 0810060
• Latvia
  • Riga, Latvia, 1038
    • Investigator site 3710001
• Netherlands
  • Amsterdam, Netherlands, 1105
    • Investigator site 0310010
  • Rotterdam, Netherlands
    • Investigator site 0310011
• Poland
  • Białystok, Poland, 15-402
    • Investigator site 0480019
  • Katowice, Poland, 40-650
    • Investigator site 0480023
  • Kraków, Poland, 30-539
    • Investigator site 0480017
  • Kraków, Poland, 31-202
    • Investigator site 0480024
  • Lublin, Poland, 20-090
    • Investigator site 0480018
  • Warsaw, Poland
    • Investigator site 0480022
  • Łódź, Poland, 90-324
    • Investigator site 0480020
• Romania
  • Braşov, Romania, 500299
    • Investigation site 0400002
  • Bucharest, Romania, 011302
    • Investigator site 0400001
  • Constanţa, Romania, 900591
    • Investigator site 0400004
  • Timişoara, Romania, 300723
    • Investigator site 0400003
• Russian Federation
  • Kazan, Russian Federation, 420021
    • Investigator site 0070017
  • Kazan, Russian Federation, 420097
    • Investigator site 0070023
  • Moscow, Russian Federation, 117186
    • Investigator site 0070016
  • Moscow, Russian Federation, 117186
    • Investigator site 0070020
  • Perm, Russian Federation
    • Investigator site 0070018
  • Rostov-on-Don, Russian Federation, 344022
    • Investigator site 0070019
  • Saint Petersburg, Russian Federation, 194354
    • Investigator site 0070014
  • Saransk, Russian Federation, 430032
    • Investigator site 0070021
• Serbia
  • Belgrad, Serbia, 11000
    • Investigator site 3810001
  • Belgrad, Serbia, 11000
    • Investigator site 3810003
  • Kragujevac, Serbia
    • Investigator site 3810004
• Spain
  • Alicante, Spain, 03010
    • Investigator site 0340020
  • Badalona, Spain, 08041
    • Investigator site 0340021
  • Barcelona, Spain
    • Investigator site 0340038
  • Córdoba, Spain, 14011
    • Investigator site 0340019
  • Madrid, Spain, 28007
    • Investigator site 0340017
  • Madrid, Spain, 28040
    • Investigator site 0340018
  • Sevilla, Spain, 41013
    • Investigator site 0340016
• Taiwan
  • Kaohsiung, Taiwan
    • Investigator site 8860014
  • Taichung, Taiwan
    • Investigator site 8860015
  • Tainan, Taiwan
    • Investigator site 8860013
  • Taipei, Taiwan
    • Investigator site 8860011
  • Taipei, Taiwan
    • Investigator site 8860012
  • Taipei, Taiwan
    • Investigator site 8860016
  • Taoyuan, Taiwan
    • Investigator site 8860017
• Turkey
  • Bursa, Turkey
    • Investigator site 0900025
  • Istanbul, Turkey
    • Investigator site 0900023
  • Samsun, Turkey
    • Investigator site 0900022
  • Sarıçam, Turkey
    • Investigator site 0900024
  • İzmir, Turkey
    • Investigator site 0900021
• Ukraine
  • Dnipro, Ukraine, 49069
    • Investigator site 3800012
  • Dnipropetrovs'k, Ukraine, 79044
    • Investigator site 3800014
  • Ivano-Frankivs'k, Ukraine, 76008
    • Investigator site 3800010
  • Kharkiv, Ukraine, 61058
    • Investigator site 3800015
  • Kyiv, Ukraine, 21000
    • Investigator site 3800013
  • Luts'k, Ukraine, 43024
    • Investigator site 380008
  • Vinnytsia, Ukraine, 21009
    • Investigator site 380009
  • Vinnytsia, Ukraine
    • Investigator site 3800015
  • Zaporizhzhya, Ukraine, 69068
    • Investigator site 3800011
• United Kingdom
  • Glasgow, United Kingdom
    • Investigator site 0440017
  • Inverness, United Kingdom
    • Investigator site 0440015
  • London, United Kingdom
    • Investigator site 0440026
  • Oxford, United Kingdom
    • Investigator site 0440016
  • Sheffield, United Kingdom
    • Investigator site 0440018
  • Stoke-on-Trent, United Kingdom
    • Investigator site 0440019
  • Tooting, United Kingdom
    • Investigator site 0440028
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233-2110
      • Investigator site 0010065
  • Arizona
    • Phoenix, Arizona, United States, 85018
      • Investigator site 0010013
    • Scottsdale, Arizona, United States, 85028
      • Investigator site 0010055
  • California
    • Carlsbad, California, United States, 92011
      • Investigator Site 0010032
    • Orange, California, United States, 92868
      • Investigator site 0010004
    • Pomona, California, United States, 91767-2009
      • Investigator site 0010190
    • Rancho Mirage, California, United States, 92270
      • Investigator site 0010160
    • San Francisco, California, United States, 94109
      • Investigator site 0010071
  • Colorado
    • Centennial, Colorado, United States, 80112
      • Investigator site 0010057
  • Connecticut
    • New Haven, Connecticut, United States, 06520
      • Investigator site 0010026
  • Florida
    • Boca Raton, Florida, United States, 33487
      • Investigator site 0010072
    • Coral Springs, Florida, United States, 33067-4640
      • Investigator site 0010144
    • Jacksonville, Florida, United States, 32209
      • Investigator site 0010023
    • Maitland, Florida, United States, 32751
      • Investigator Site 0010068
    • Miami, Florida, United States, 33136
      • Investigator site 0010059
    • Orlando, Florida, United States, 32806
      • Investigator site 0010050
    • Ormond Beach, Florida, United States, 32174-3102
      • Investigator site 0010172
    • Tampa, Florida, United States, 33612
      • Investigator site 0010006
  • Georgia
    • Augusta, Georgia, United States, 30912-3125
      • Investigator site 0010125
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Investigator site 0010011
  • Kansas
    • Fairway, Kansas, United States, 66205
      • Investigator site 0010015
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • Investigator site 0010147
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Investigator site 0010014
    • East Lansing, Michigan, United States, 48824
      • Investigator site 0010063
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455-4800
      • Investigator site 0010052
  • Missouri
    • Columbia, Missouri, United States, 65212
      • Investigator site 0010028
  • New Jersey
    • New Brunswick, New Jersey, United States, 08550
      • Investigator site 0010070
  • New York
    • New York, New York, United States, 06511
      • Investigator site 0010069
    • New York, New York, United States, 10001
      • Investigator site 0010168
    • New York, New York, United States, 10021
      • Investigator site 0010191
    • New York, New York, United States, 10032
      • Investigator site 0010074
    • Patchogue, New York, United States, 11772
      • Investigator site 0010075
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27517
      • Investigator site 0010003
    • Durham, North Carolina, United States, 27710
      • Investigator site 0010077
  • Ohio
    • Cincinnati, Ohio, United States, 45267-0525
      • Investigator site 0010051
    • Columbus, Ohio, United States, 43210
      • Investigator site 0010064
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 15213
      • Investigator site 0010047
    • Philadelphia, Pennsylvania, United States, 19104
      • Investigator site 0010007
    • Pittsburgh, Pennsylvania, United States, 15123
      • Investigator site 0010067
  • Texas
    • Austin, Texas, United States, 78756
      • Investigator site 0010066
    • Houston, Texas, United States, 77055-7421
      • Investigator site 0010026
    • San Antonio, Texas, United States, 78229
      • Investigator site 0010009
  • Vermont
    • Burlington, Vermont, United States, 05401
      • Investigator site 0010076
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • Investigator site 0010007
    • Richmond, Virginia, United States, 23298
      • Investigator site 0010061

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 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)
2. Male or female patient aged 18 years or older, at the time of signing the informed consent.
3. 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.
4. CIDP Disease Activity Status (CDAS) score ≥2 at screening.
5. 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.

6. 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.
7. Women of childbearing potential who have a negative pregnancy test at screening and a negative urine pregnancy test up to Stage A baseline.
8. Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last dose of IMP

Exclusion Criteria:

1. Pure sensory atypical CIDP (EFNS/PNS definition).
2. 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.
3. Any other disease that could better explain the patient's signs and symptoms.
4. Any history of myelopathy or evidence of central demyelination.
5. Current or past history (within 12 months of screening) of alcohol, drug or medication abuse.
6. 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.
7. 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.
8. Total IgG level <6 g/L at screening.

9. 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.

10. Pregnant and lactating women and those intending to become pregnant during the trial or within 90 days after last IMP administration.
11. 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.
12. 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.
13. 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).
14. Patients who previously participated in a trial with efgartigimod and have received at least one administration of IMP.
15. Patients with known medical history of hypersensitivity to any of the ingredients of IMP.
16. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: efgartigimod PH20 SC; patients receiving efgartigimod PH20 SC in both stage A as stage B	Biological: efgartigimod PH20 SC in stage B; Stage A: efgartigimod PH20 SC, Stage B: efgartigimod PH20 SC
Placebo Comparator: Placebo; patients receiving efgartigimod PH20 SC during stage A and receiving placebo in stage B	Biological: efgartigimod PH20 SC in stage B; Stage A: efgartigimod PH20 SC, Stage B: efgartigimod PH20 SC; Other: placebo in stage B; Stage A: N/A, stage B: placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Stage A: Percentage of patients 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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Stage A: Time to initial confirmed ECI		Up to 12 weeks during the open-label stage A
Stage A: Change from Stage A baseline over time in adjusted INCAT score		Up to 12 weeks during the open-label stage A
Stage A: Change from Stage A baseline over time in Medical Research Council (MRC) Sum score		Up to 12 weeks during the open-label stage A
Stage A: Change from Stage A baseline over time in I-RODS disability scores		Up to 12 weeks during the open-label stage A
Stage A: Change from Stage A baseline over time in TUG score		Up to 12 weeks during the open-label stage A
Stage A: Change from Stage A baseline over time in mean grip strength		Up to 12 weeks during the open-label stage A
Stage A: Exposure adjusted occurrence of treatment-emergent (serious) adverse events		Up to 12 weeks during the open-label stage A
Stage A: Incidence of clinically significant laboratory abnormalities		Up to 12 weeks during the open-label stage A
Stage A: Pre-dosing efgartigimod serum concentrations over time		Up to 12 weeks during the open-label stage A
Stage A: Changes of serum IgG levels over time		Up to 12 weeks during the open-label stage A
Stage A: Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and/or rHuPH20		Up to 12 weeks during the open-label stage A
Stage A: Changes from D1A in EQ-5D-5L over time		Up to 12 weeks during the open-label stage A
Stage B: Time to CIDP disease progression	Time to 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.	Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Percentage of patients with improved functional level compared to Stage B baseline		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change from Stage B baseline over time in adjusted INCAT score		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change from Stage B baseline over time in MRC Sum score		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change from Stage B baseline over time in 24-item I-RODS disability score		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change from Stage B baseline over time in TUG score		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Change from Stage B baseline over time in mean grip strength		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Time to 10% decrease in the 24-item I-RODS		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Exposure adjusted occurrence of treatment-emergent adverse events and serious adverse events		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Incidence of clinically significant laboratory abnormalities		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Pre-dosing efgartigimod serum concentrations over time		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Changes of serum IgG levels over time		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and/or rHuPH20		Up to 48 weeks during the randomized placebo-controlled stage B
Stage B: Changes from D1A in EQ-5D-5L over time		Up to 48 weeks during the randomized placebo-controlled stage B

Collaborators and Investigators

• Sponsor: argenx

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): April 15, 2020
• Primary Completion (Actual): May 11, 2023
• Study Completion (Actual): May 11, 2023

Study Registration Dates

• First Submitted: February 20, 2020
• First Submitted That Met QC Criteria: February 21, 2020
• First Posted (Actual): February 24, 2020

Study Record Updates

• Last Update Posted (Actual): August 4, 2023
• Last Update Submitted That Met QC Criteria: August 3, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease Attributes; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyradiculoneuropathy; Chronic Disease; Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
• Other Study ID Numbers: ARGX-113-1802; 2019-003076-39 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No