Clinical trial • Phase III • Immunology|Rare Disease

EFGARTIGIMOD ALFA for Generalized myasthenia gravis (AChR‑Ab seronegative)

Phase III trial of EFGARTIGIMOD ALFA for Generalized myasthenia gravis (AChR‑Ab seronegative).

Overview

Trial Therapeutic Area
Immunology|Rare Disease
Trial Disease
Generalized myasthenia gravis (AChR‑Ab seronegative)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
14-06-2024
First CTIS Authorization Date
01-10-2024

Trial design

Randomised, open-label, argx-113 (efgartigimod alfa) iv solution for infusion (test) vs placebo efgartigimod iv concentrate for solution (matching placebo). max daily dose reported in product data: 1200 mg (per product record).-controlled Phase III trial in Denmark, Greece, Portugal and others.

Randomised
Yes
Open Label
Yes
Comparator
ARGX-113 (efgartigimod alfa) IV solution for infusion (test) vs Placebo efgartigimod IV concentrate for solution (matching placebo). Max daily dose reported in product data: 1200 mg (per product record).
Target Sample Size
105
Trial Duration For Participant
112

Eligibility

Recruits 105 No vulnerable populations are selected. Participants must be adults (at least 18 years and local legal age of consent) and able to provide signed informed consent. Women of child-bearing potential must have negative pregnancy tests (serum at screening and urine at baseline) and agree to use contraception per local regulations. No assent/child consent procedures are required..

Pregnancy Exclusion
7.Pregnant or lactating state or intention to become pregnant during the study
Vulnerable Population
No vulnerable populations are selected. Participants must be adults (at least 18 years and local legal age of consent) and able to provide signed informed consent. Women of child-bearing potential must have negative pregnancy tests (serum at screening and urine at baseline) and agree to use contraception per local regulations. No assent/child consent procedures are required.

Inclusion criteria

  • {"criterion_text":"-1.Is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF."}
  • {"criterion_text":"-2.The participant is capable of providing signed informed and complying with protocol requirements"}
  • {"criterion_text":"-3.The participant agrees to use contraceptive measures consistent with local regulations and the women of child-bearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline before receiving the investigational medicinal product (IMP)."}
  • {"criterion_text":"-4.The participant has no known weakness in infancy and later develop fatigable weakness after aged 16 years and diagnosed with acquired gMG of both of the following: a) History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation (RNS) or is anti-muscle-specific kinase antibodies (MuSK-Ab) seropositive b) Either a history of positive edrophonium chloride test OR a demonstrated improvement in MG signs with treatments such as oral acetylcholinesterase (AChE) inhibitors, plasma exchange (PLEX), immunoabsorption, or intravenous immunoglobulin (IVIg)/ subcutaneous immunoglobulin (SCIg) treatment."}
  • {"criterion_text":"-5.The participant is receiving a stable dose of MG therapy before screening that includes acetylcholinesterase (AChE) inhibitors, steroids, or nonsteroidal immunosuppressive therapies (NSISTs) in combination or alone."}

Exclusion criteria

  • {"criterion_text":"-1.Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or puts the participant at undue risk"}
  • {"criterion_text":"-10. Received a thymectomy less than 3 months before screening or thymectomy is planned during the study"}
  • {"criterion_text":"-11.Use of some medications before screening (more information is captured in the protocol)"}
  • {"criterion_text":"-2.History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥ 3 years before first investigational medicinal product (IMP) administration. Adequately treated participants with the following cancers can be included at any time: basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, carcinoma in situ of the breast, incidental histological findings of prostate cancer."}
  • {"criterion_text":"-3.Clinically significant active infection that is not sufficiently resolved before baseline in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV."}
  • {"criterion_text":"-4.Current participation in another interventional clinical study or previous participation in an efgartigimod clinical study and received at least 1 dose of investigational medicinal product (IMP)"}
  • {"criterion_text":"-5.Known hypersensitivity to investigational medicinal product (IMP) or any of its excipients"}
  • {"criterion_text":"-6.History of or current alcohol, drug, or medication abuse as assessed by the investigator"}
  • {"criterion_text":"-7.Pregnant or lactating state or intention to become pregnant during the study"}
  • {"criterion_text":"-8. Live or live-attenuated vaccine received less than 4 weeks before screening"}
  • {"criterion_text":"-9.Worsening muscle weakness secondary to concurrent infections or medications"}

Endpoints

Primary endpoints

  • {"endpoint_text":"-MG-ADL total score change from baseline to day 29 in part A","definition_or_measurement_approach":"Change from baseline in the MG-ADL total score measured at Day 29 in Part A."}

Secondary endpoints

  • {"endpoint_text":"-• QMG total score change from baseline to day 29 in part A","definition_or_measurement_approach":"Change from baseline in the QMG total score measured at Day 29 in Part A."}
  • {"endpoint_text":"-• Proportion of participants who are both MG-ADL and QMG responders in part A","definition_or_measurement_approach":"Proportion of participants classified as responders on both MG-ADL and QMG scales in Part A (timepoint not further specified in summary; endpoint defined for Part A)."}

Recruitment

Planned Sample Size
105
Recruitment Window Months
33
Consent Approach
Signed informed consent is required from each participant (participants must be ≥18 and meet local legal consent age). Separate pregnancy-specific ICFs are used where applicable. Women of child-bearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline and agree to use contraception per local regulations. ICF materials (main ICF, pregnancy ICF, optional research ICF) are provided in local languages across participating countries.

Methods

  • Doctor-to-patient letters (country-specific doctor-to-patient letters are listed, e.g., DE, BE, CY, ES, NL, PL, NO).
  • Patient recruitment brochures / patient recruitment brochures in local languages (documents exist for multiple countries including CY, DE, FR, BE, ES, PL, NL, FIN, NOR, RO).
  • GP / primary care letters (e.g., GP letter DE) to facilitate recruitment via referring physicians.
  • Recruitment and informed consent procedure documents (K1/K2) describing site-level recruitment processes in each participating country.
  • Site-level materials (patient letters, brochures) targeted to patients with generalized myasthenia gravis (AChR‑Ab seronegative) and their treating clinicians; materials are provided in country/local languages as per the document list.

Geography

Total Number Of Sites
49
Total Number Of Participants
167

Denmark

Earliest CTIS Part Ii Submission Date
18-09-2024
Latest Decision Or Authorization Date
05-08-2025
Processing Time Days
321
Number Of Sites
3
Number Of Participants
10

Sites

Site Name
Aarhus Universitetshospital
Department Name
Department of Neurology
Contact Person Name
Henning Andersen
Contact Person Email
heenande@rm.dk
Site Name
Odense University Hospital
Department Name
Department of Neurology
Contact Person Name
Jacob Vormstrup Holbech
Contact Person Email
jacob.holbech@rsyd.dk
Site Name
Rigshospitalet
Department Name
Copenhagen Neuromuscular Center
Contact Person Name
Nicolai Rasmus Preisler

Greece

Earliest CTIS Part Ii Submission Date
25-09-2024
Latest Decision Or Authorization Date
05-08-2025
Processing Time Days
314
Number Of Sites
4
Number Of Participants
11

Sites

Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
Department of Clinical Νeurophysiology
Contact Person Name
Marianthi Arnaoutoglou
Contact Person Email
marnaoutoglou@yahoo.com
Site Name
General University Hospital Of Patras
Department Name
Neurology Clinic
Contact Person Name
Elisabeth Chroni
Contact Person Email
dbakos@pgnp.gr
Site Name
University General Hospital Attikon
Department Name
Second Department of Neurology Clinic
Contact Person Name
Sotirios Giannopoulos
Contact Person Email
sgiannop@uoi.gr
Site Name
Eginitio Hospital
Department Name
1st Neurology Department
Contact Person Name
Vasiliki Zouvelou
Contact Person Email
vzouvelu@med.uoa.gr

Portugal

Earliest CTIS Part Ii Submission Date
13-09-2024
Latest Decision Or Authorization Date
04-08-2025
Processing Time Days
325
Number Of Sites
3
Number Of Participants
7

Sites

Site Name
Unidade Local De Saude De Lisboa Ocidental E.P.E.
Department Name
Neurology Department
Contact Person Name
Marco Fernandes
Contact Person Email
marcoa.fernandes91@gmail.com
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Neurology Department
Contact Person Name
Mamede de Carvalho
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Neurology Department
Contact Person Name
Ernestina Santos
Contact Person Email
ernestina.santos@gmail.com

Hungary

Earliest CTIS Part Ii Submission Date
12-09-2024
Latest Decision Or Authorization Date
05-08-2025
Processing Time Days
327
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Semmelweis University
Department Name
Genomikai Medicina és Ritka Betegségek Intézete
Contact Person Name
Mária Judit Molnár
Contact Person Email
molnarmj@gmail.com

Czechia

Earliest CTIS Part Ii Submission Date
05-09-2024
Latest Decision Or Authorization Date
04-08-2025
Processing Time Days
333
Number Of Sites
3
Number Of Participants
11

Sites

Site Name
Nemocnice Pardubickeho kraje a.s.
Department Name
Neurologická klinika
Contact Person Name
Edvard Ehler
Contact Person Email
Edvard.ehler@nempk.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Neurologická klinika
Contact Person Name
Stanislav Voháňka
Contact Person Email
vohanka.stanislav@fnbrno.cz
Site Name
Neurologie and Rehabilitace
Department Name
N-A
Contact Person Name
Eduard Minks
Contact Person Email
eduardminks@seznam.cz

Cyprus

Earliest CTIS Part Ii Submission Date
05-09-2024
Latest Decision Or Authorization Date
05-08-2025
Processing Time Days
334
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
The Cyprus Foundation For Muscular Dystrophy Research
Department Name
Neuroscience Department
Contact Person Name
Kleopas Kleopa
Contact Person Email
kleopa@cing.ac.cy

France

Earliest CTIS Part Ii Submission Date
20-09-2024
Latest Decision Or Authorization Date
07-08-2025
Processing Time Days
321
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Service de Médecine intensive et réanimation à orientation neurologique (MIR neuro)
Contact Person Name
Sophie Demeret
Contact Person Email
Sophie.demeret@aphp.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Service Neurologie
Contact Person Name
Saskia Bresch
Contact Person Email
Bresch.s@chu-nice.fr
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Centre de Référence des Maladies Neuromusculaire et de la SLA
Contact Person Name
Shahram Attarian
Contact Person Email
Shahram.attarian@ap-hm.fr

Germany

Earliest CTIS Part Ii Submission Date
23-09-2024
Latest Decision Or Authorization Date
07-08-2025
Processing Time Days
318
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Charité – Universitätsmedizin Berlin Neuroscience Clinical Research Center
Contact Person Name
Sarah Hoffmann
Contact Person Email
sarah.hoffmann@charite.de
Site Name
Katholisches Klinikum Bochum gGmbH
Department Name
Neurologische Studienambulanz
Contact Person Name
Christiane Schneider-Gold
Site Name
Universitaetsmedizin Goettingen
Department Name
Klinik für Neurologie
Contact Person Name
Jana Zschüntzsch
Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Neurologische Klinik I
Contact Person Name
Axel Haarmann
Contact Person Email
haarmann_a@ukw.de

Romania

Earliest CTIS Part Ii Submission Date
11-10-2024
Latest Decision Or Authorization Date
11-08-2025
Processing Time Days
304
Number Of Sites
4
Number Of Participants
25

Sites

Site Name
Spitalul Clinic Judetean De Urgenta Sibiu
Department Name
Sectia Clinica de Neurologie
Contact Person Name
Carmen Corina Roman-Filip
Contact Person Email
corina.roman@ulbsibiu.ro
Site Name
Spitalul Clinic Judetean De Urgenta Targu Mures
Department Name
Sectia Clinica de Neurologie I
Contact Person Name
Rodica Ioana Balasa
Contact Person Email
rodica.balasa@umfst.ro
Site Name
Spitalul Clinic Judetean De Urgenta Cluj
Department Name
Sectia Clinica Neurologie II
Contact Person Name
Vitalie Vacaras
Contact Person Email
vvacaras@umfcluj.ro
Site Name
Spitalul Clinic Judetean De Urgenta Sfantul Apostol Andrei Constanta
Department Name
Sectia Clinica Neurologie
Contact Person Name
Any Axelerad
Contact Person Email
docuaxi@yahoo.com

Belgium

Earliest CTIS Part Ii Submission Date
03-09-2024
Latest Decision Or Authorization Date
04-08-2025
Processing Time Days
335
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
AZ Sint-Lucas & Volkskliniek
Department Name
Neurology
Contact Person Name
Jan De Bleecker
Contact Person Email
Jan.DeBleecker@AZSTLUCAS.BE
Site Name
UZ Leuven
Department Name
Neurology
Contact Person Name
Kristl Claeys
Contact Person Email
kristl.claeys@uzleuven.be
Site Name
Hopital Erasme
Department Name
Neurology
Contact Person Name
Gauthier Remiche
Site Name
Cliniques Universitaires Saint-Luc
Department Name
Neurology
Contact Person Name
Vinciane Van Parys

Finland

Earliest CTIS Part Ii Submission Date
04-09-2024
Latest Decision Or Authorization Date
04-08-2025
Processing Time Days
334
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Turku University Hospital
Department Name
T-hospital, Poliklinikka D2
Contact Person Name
Manu Jokela
Contact Person Email
Manu.Jokela@tyks.fi

Norway

Earliest CTIS Part Ii Submission Date
06-09-2024
Latest Decision Or Authorization Date
05-08-2025
Processing Time Days
333
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
Oslo University Hospital HF
Department Name
Forskning og Utvikling
Contact Person Name
Angelina Hatlø Maniaol
Contact Person Email
angman@ous-hf.no
Site Name
Helse Bergen HF
Department Name
Nevrologisk avdeling
Contact Person Name
Nils Erik Gilhus

Spain

Earliest CTIS Part Ii Submission Date
05-09-2024
Latest Decision Or Authorization Date
06-08-2025
Processing Time Days
335
Number Of Sites
7
Number Of Participants
20

Sites

Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Contact Person Name
Raúl Juntas Morales
Contact Person Email
raul.juntas@vallhebron.cat
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Neurology
Contact Person Name
Rodrigo Álvarez Velasco
Site Name
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida
Department Name
Neurology
Contact Person Name
Cristina González Mingot
Site Name
Complexo Hospitalario Universitario De Santiago
Department Name
Neuromuscular Disorder
Contact Person Name
Beatrice Canneti Heredia
Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Neurology
Contact Person Name
Elena Cortés Vicente
Contact Person Email
ecortes@santpau.cat
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Contact Person Name
María Teresa Sevilla Mantecón
Contact Person Email
sevilla_ter@gva.es
Site Name
Hospital General Universitario Dr. Balmis
Department Name
Neurology
Contact Person Name
Raquel Hernández Lorido
Contact Person Email
ensayosneralicante@gmail.com

Netherlands

Earliest CTIS Part Ii Submission Date
05-09-2024
Latest Decision Or Authorization Date
11-08-2025
Processing Time Days
340
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
Neurology
Contact Person Name
Jan Verschuuren
Contact Person Email
j.j.g.m.verschuuren@lumc.nl
Site Name
Universitair Medisch Centrum Utrecht
Department Name
Neuromuscular diseases
Contact Person Name
Michael van Es
Contact Person Email
cipn@umcutrecht.nl

Poland

Earliest CTIS Part Ii Submission Date
06-09-2024
Latest Decision Or Authorization Date
11-08-2025
Processing Time Days
339
Number Of Sites
7
Number Of Participants
50

Sites

Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Contact Person Name
Łukasz Rzepiński
Contact Person Email
luk.rzepinski@gmail.com
Site Name
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Contact Person Name
Edyta Dziadkowiak
Contact Person Email
edyta.dziadkowiak@umw.edu.pl
Site Name
Clinirem Sp. z o.o.
Contact Person Name
Urszula Chyrchel-Paszkiewicz
Contact Person Email
pgluchyrchel@gmail.com
Site Name
Neurologia Śląska Centrum Medyczne
Contact Person Name
Marek Śmiłowski
Site Name
Krakowska Akademia Neurologii Sp. z o.o.
Contact Person Name
Andrzej Szczudlik
Contact Person Email
olga.fraczek@neurologia.org.pl
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Contact Person Name
Agnieszka Słowik
Contact Person Email
neurologiabk@su.krakow.pl
Site Name
Other listed Polish site

Sponsor

Primary sponsor

Full Name
Argenx
Organisation Type
Pharmaceutical company
Country Of Registered Address
Belgium

Contract research organisations

Name
PPD Development LP
Responsibilities
Vendor Management; Patient recruitment; other sponsor duties (codes present)
Name
PPD Global Ltd.
Responsibilities
Clinical site/regulatory support (contact listed); duties codes present
Name
IQVIA Limited
Responsibilities
ICSR processing and pharmacovigilance support
Name
Fisher Clinical Services GmbH
Responsibilities
IMP storage and distribution
Name
WCG Clinical Inc.
Responsibilities
Online training portal and organising of Virtual Investigator meeting
Name
SGS Belgium / SGS France
Responsibilities
Medical data review, DSMB activities
Name
Suvoda LLC
Responsibilities
Electronic data capture / site services (code indicated)

Third parties

  • {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"IMP storage and distribution","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Codes: 1;12;13;15 (15: Vendor Management; Patient recruitment);2;5","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Online training portal and organising of Virtual Investigator meeting","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central ECG and eCOA","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"Medical Data Review, and DSMB; code 10;6","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"Fisher Clinical Services GmbH (additional address)","duties_or_roles":"IMP storage and distribution","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"Patient Travel and Reimbursement","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"France","full_name":"SGS France","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd. (Greece address)","duties_or_roles":"1;12;5","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"ICSR processing; code 8","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
ARGX-113
Active Substance
EFGARTIGIMOD ALFA
Modality
Monoclonal antibody
Routes Of Administration
Intravenous infusion
Route
Intravenous infusion
Authorisation Status
Investigational (no marketing authorisation listed)
Orphan Designation
Yes
Maximum Dose
1200 mg
Investigational Product Name
Vyvgart 20 mg/mL concentrate for solution for infusion
Active Substance
EFGARTIGIMOD ALFA
Modality
Monoclonal antibody
Routes Of Administration
Intravenous infusion
Route
Intravenous infusion
Authorisation Status
Authorised (marketing authorisation EU/1/22/1674/001)
Orphan Designation
Yes
Maximum Dose
1200 mg
Investigational Product Name
Placebo efgartigimod IV concentrate for solution
Modality
Other
Routes Of Administration
Intravenous infusion (matching placebo)
Route
Intravenous infusion

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