Clinical trial • Phase III • Immunology|Rare Disease

EFGARTIGIMOD ALFA for Ocular myasthenia gravis

Phase III trial of EFGARTIGIMOD ALFA for Ocular myasthenia gravis.

Overview

Trial Therapeutic Area
Immunology|Rare Disease
Trial Disease
Ocular myasthenia gravis
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
29-10-2024
First CTIS Authorization Date
04-03-2025

Trial design

Randomised, open-label, arms: efgartigimod ph20 sc (vyvgart 1 000 mg solution for injection in pre-filled syringe) versus placebo ph20 sc. dose and schedule not specified in the ctis part i/ii entries.-controlled Phase III trial in Finland, Germany, Portugal and others.

Randomised
Yes
Open Label
Yes
Comparator
Arms: efgartigimod PH20 SC (Vyvgart 1 000 mg solution for injection in pre-filled syringe) versus Placebo PH20 SC. Dose and schedule not specified in the CTIS Part I/II entries.
Target Sample Size
98
Trial Duration For Participant
119

Eligibility

Recruits 98 No vulnerable populations selected. Participants must be at least 18 years of age and meet the local legal age for consent. Country-specific caregiver informed consent forms and pregnancy/birth information/ICF documents are provided (multiple country-specific caregiver and pregnancy ICFs are listed in the trial documents)..

Vulnerable Population
No vulnerable populations selected. Participants must be at least 18 years of age and meet the local legal age for consent. Country-specific caregiver informed consent forms and pregnancy/birth information/ICF documents are provided (multiple country-specific caregiver and pregnancy ICFs are listed in the trial documents).

Inclusion criteria

  • {"criterion_text":"- 1. Is at least 18 years of age and the local legal age of consent for clinical studies\n- 2. Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy\n- 3. Is MGFA Class I (any ocular muscle weakness)\n- 4. Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2"}

Exclusion criteria

  • {"criterion_text":"- 1. Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia\n- 2. Known autoimmune disease or any medical condition other than indication under study that would interfere with an accurate assessment of clinical symptoms of ocular myasthenia gravis or puts the participant at undue risk"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- MGII (PRO) ocular score change from baseline to day 29 in part A","definition_or_measurement_approach":"Change from baseline to Day 29 in the MGII (patient-reported outcome) ocular score in Part A"}

Secondary endpoints

  • {"endpoint_text":"- •\tMGII (PRO+PE) ocular score change from baseline to day 29 in part A\n- •\tMG-ADL ocular domain score change from baseline to day 29 in part A\n- •\tMGII total score change from baseline to day 29 in part A\n- •\tMGII (PE) ocular score change from baseline to day 29 in part A\n- •\tMG-ADL total score change from baseline to day 29 in part A\n- •\tMGII ocular scores (PRO, PRO+PE, and PE), generalized score, and total score; actual values and changes from baseline in part A and part A+B\n- •\tIncidence and severity of AEs and SAEs in part A and part A+B\n- •\tClinically relevant changes in laboratory parameters, vital signs, and ECGs in part A and part A+B\n- •\tMG-QoL15r total score actual values and changes from baseline in part A and part A+B\n- •\tNEI VFQ-25 score actual values and changes from baseline in part A and part A+B\n- •\tActual values and percent changes from baseline in total IgG levels over time in part A and part A+B\n- Actual values and percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A and part A+B\n- •\tIncidence and prevalence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A and part A+B\n- •\tIncidence and prevalence of antibodies and Neutralizing Antibodies against rHuPH20 in part A and part A+B","definition_or_measurement_approach":"Endpoints are largely change-from-baseline measures (commonly baseline to Day 29 in Part A) for clinical scales (MGII, MG-ADL), patient-reported outcomes (MG-QoL15r, NEI VFQ-25), laboratory measures (total IgG, AChR-Ab), and safety outcomes (incidence/severity of AEs/SAEs); immunogenicity endpoints measure incidence/prevalence of anti-drug and neutralizing antibodies against efgartigimod and rHuPH20 over time."}

Recruitment

Registry Or Advocacy Recruitment
True, site-patient-advocacy contact lists are provided (a "Site-Patient-advocacy_Contact-List-for-ICF" document is listed for Austria); specific advocacy organisation names are not specified in the CTIS documents list.
Planned Sample Size
98
Recruitment Window Months
38
Consent Approach
Adult participants (≥18 years) provide written informed consent. Country-specific Main ICFs and caregiver ICFs are provided (documents list Main ICF and Caregiver ICF translations for many Member States). ICFs and related materials are available in multiple languages corresponding to the countries listed (e.g., English, German, Dutch, French, Spanish, Portuguese, Polish, Greek, Swedish, Italian, Czech). Optional/secondary ICFs (e.g., pregnancy/follow-up, optional PK, optional future research, travel reimbursement) are provided as separate documents.

Methods

  • Doctor-to-patient letters (country-specific doctor-to-patient letter documents listed for multiple Member States)
  • Patient recruitment brochures/flyers/posters/site flyers (multilingual materials for sites and public listed under recruitment arrangements for multiple countries)
  • Site-based recruitment through participating neurology/ophthalmology clinics and hospitals (site contact lists and site flyers provided)
  • Vendor-managed recruitment support (PPD/PPD Development indicated duties include vendor management and patient recruitment)
  • Patient travel and reimbursement support (Unisphere Travel / patient travel and reimbursement duties listed)

Geography

Total Number Of Sites
38
Total Number Of Participants
169

Finland

Earliest CTIS Part Ii Submission Date
04-02-2025
Latest Decision Or Authorization Date
17-03-2025
Processing Time Days
41
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Turku University Hospital
Department Name
TE5, Neurokeskus
Principal Investigator Name
Manu Jokela
Principal Investigator Email
manu.jokela@varha.fi
Contact Person Name
Manu Jokela
Contact Person Email
manu.jokela@varha.fi

Germany

Earliest CTIS Part Ii Submission Date
26-02-2025
Latest Decision Or Authorization Date
04-03-2025
Processing Time Days
6
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Neuroscience Clinical Research Center
Principal Investigator Name
Andreas Meisel
Principal Investigator Email
andreas.meisel@charite.de
Contact Person Name
Andreas Meisel
Contact Person Email
andreas.meisel@charite.de
Site Name
Universitaetsmedizin Goettingen
Department Name
Klinik fuer Neurologie
Principal Investigator Name
Jana Zschuentzsch
Principal Investigator Email
j.zschuentzsch@med.uni-goettingen.de
Contact Person Name
Jana Zschuentzsch

Portugal

Earliest CTIS Part Ii Submission Date
23-01-2025
Latest Decision Or Authorization Date
05-03-2025
Processing Time Days
41
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Neurology Department
Principal Investigator Name
Mamede de Carvalho
Principal Investigator Email
mamedealves@medicina.ulisboa.pt
Contact Person Name
Mamede de Carvalho
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Neurology Department
Principal Investigator Name
Ernestina Santos
Principal Investigator Email
ernestina.santos@gmail.com
Contact Person Name
Ernestina Santos
Contact Person Email
ernestina.santos@gmail.com

Belgium

Earliest CTIS Part Ii Submission Date
26-02-2025
Latest Decision Or Authorization Date
04-03-2025
Processing Time Days
6
Number Of Sites
2
Number Of Participants
12

Sites

Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Neurology
Principal Investigator Name
Margaux Poleur
Principal Investigator Email
margaux.poleur@citadelle.be
Contact Person Name
Margaux Poleur
Contact Person Email
margaux.poleur@citadelle.be
Site Name
UZ Leuven
Department Name
Neurology
Principal Investigator Name
Kristl Claeys
Principal Investigator Email
kristl.claeys@uzleuven.be
Contact Person Name
Kristl Claeys
Contact Person Email
kristl.claeys@uzleuven.be

Cyprus

Earliest CTIS Part Ii Submission Date
26-02-2025
Latest Decision Or Authorization Date
06-03-2025
Processing Time Days
8
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
The Cyprus Foundation For Muscular Dystrophy Research
Department Name
Neuroscience Department, Neuromuscular Disorders Center,
Principal Investigator Name
Kleopas Kleopa
Principal Investigator Email
kleopa@cing.ac.cy
Contact Person Name
Kleopas Kleopa
Contact Person Email
kleopa@cing.ac.cy

Austria

Earliest CTIS Part Ii Submission Date
27-02-2025
Latest Decision Or Authorization Date
10-03-2025
Processing Time Days
11
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Medical University Of Vienna
Department Name
Department of Neurology
Principal Investigator Name
Fritz Zimprich
Principal Investigator Email
friedrich.zimprich@meduniwien.ac.at
Contact Person Name
Fritz Zimprich

Sweden

Earliest CTIS Part Ii Submission Date
31-01-2025
Latest Decision Or Authorization Date
14-03-2025
Processing Time Days
42
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
Department Name
Neurosjukvården, Blå Stråket 7, 413 45 Göteborg
Principal Investigator Name
Lenka Nováková Nyrén
Principal Investigator Email
lenka.novakova@vgregion.se
Contact Person Name
Lenka Nováková Nyrén
Contact Person Email
lenka.novakova@vgregion.se

Denmark

Earliest CTIS Part Ii Submission Date
05-02-2025
Latest Decision Or Authorization Date
18-03-2025
Processing Time Days
41
Number Of Sites
2
Number Of Participants
12

Sites

Site Name
Aarhus University Hospital
Department Name
Department of Neurology
Principal Investigator Name
Henning Andersen
Principal Investigator Email
hennande@rm.dk
Contact Person Name
Henning Andersen
Contact Person Email
hennande@rm.dk
Site Name
Rigshospitalet
Department Name
Copenhagen Neuromuscular Center
Principal Investigator Name
Nicolai Rasmus Preisler
Principal Investigator Email
nicolai.rasmus.preisler@regionh.dk
Contact Person Name
Nicolai Rasmus Preisler

Netherlands

Earliest CTIS Part Ii Submission Date
27-02-2025
Latest Decision Or Authorization Date
10-03-2025
Processing Time Days
11
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
Neurology
Principal Investigator Name
Jan Verschuuren
Principal Investigator Email
Myasthenie@Lumc.nl
Contact Person Name
Jan Verschuuren
Contact Person Email
Myasthenie@Lumc.nl

Italy

Earliest CTIS Part Ii Submission Date
06-12-2024
Latest Decision Or Authorization Date
17-03-2025
Processing Time Days
101
Number Of Sites
6
Number Of Participants
16

Sites

Site Name
Azienda Unita Sanitaria Locale Di Bologna
Department Name
UOC Clinica Neurologica
Principal Investigator Name
Maria Pia Giannoccaro
Principal Investigator Email
mariapia.giannoccaro@ausl.bologna.it
Contact Person Name
Maria Pia Giannoccaro
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
UOC Neurology
Principal Investigator Name
Stefania Morino
Principal Investigator Email
stefania.morino@ospedalesantandrea.it
Contact Person Name
Stefania Morino
Site Name
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
Department Name
U.O.C. Neurofisiopatologia
Principal Investigator Name
Francesco Habetswallner
Principal Investigator Email
francesco.habetswallner@aocardarelli.it
Contact Person Name
Francesco Habetswallner
Site Name
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii
Department Name
SC Neurology
Principal Investigator Name
Manlio Sgarzi
Principal Investigator Email
msgarzi@asst-pg23.it
Contact Person Name
Manlio Sgarzi
Contact Person Email
msgarzi@asst-pg23.it
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
UOC Neurology IV- Neuroimmunology and Neuromuscular Diseases
Principal Investigator Name
Carlo Giuseppe Antozzi
Principal Investigator Email
carlo.antozzi@istituto-besta.it
Contact Person Name
Carlo Giuseppe Antozzi
Site Name
Ospedale San Raffaele S.r.l.
Department Name
UOC Neurology
Principal Investigator Name
Stefano Carlo Previtali
Principal Investigator Email
previtali.stefano@hsr.it
Contact Person Name
Stefano Carlo Previtali
Contact Person Email
previtali.stefano@hsr.it

Spain

Earliest CTIS Part Ii Submission Date
25-02-2025
Latest Decision Or Authorization Date
04-03-2025
Processing Time Days
7
Number Of Sites
6
Number Of Participants
25

Sites

Site Name
Hospital Universitario Donostia
Department Name
Neurology
Principal Investigator Name
Adolfo Jose Lopez de Munain Arregui
Contact Person Name
Adolfo Jose Lopez de Munain Arregui
Site Name
Hospital Del Mar
Department Name
Neurology
Principal Investigator Name
Miguel Angel Rubio Perez
Principal Investigator Email
Marubio@psmar.cat
Contact Person Name
Miguel Angel Rubio Perez
Contact Person Email
Marubio@psmar.cat
Site Name
Hospital Clinico Universitario De Valencia
Department Name
Neurology
Principal Investigator Name
Ana Isabel Pareja Martinez
Principal Investigator Email
pareja_anamar@gva.es
Contact Person Name
Ana Isabel Pareja Martinez
Contact Person Email
pareja_anamar@gva.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Principal Investigator Name
Raul Juntas Morales
Principal Investigator Email
raul.juntas@vallhebron.cat
Contact Person Name
Raul Juntas Morales
Contact Person Email
raul.juntas@vallhebron.cat
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Neurology
Principal Investigator Name
Rodrigo Álvarez Velasco
Principal Investigator Email
rodrigo.alvarez@salud.madrid.org
Contact Person Name
Rodrigo Álvarez Velasco
Site Name
Hospital Clinico San Carlos
Department Name
Neurology
Principal Investigator Name
Lucia Galan Davila
Principal Investigator Email
lgalan@salud.madrid.org
Contact Person Name
Lucia Galan Davila
Contact Person Email
lgalan@salud.madrid.org

Czechia

Earliest CTIS Part Ii Submission Date
25-02-2025
Latest Decision Or Authorization Date
05-03-2025
Processing Time Days
8
Number Of Sites
2
Number Of Participants
19

Sites

Site Name
Fakultni Nemocnice Brno
Department Name
Neurologická klinika
Principal Investigator Name
Stanislav Voháňka
Principal Investigator Email
vohanka.stanisla@fnbrno.cz
Contact Person Name
Stanislav Voháňka
Contact Person Email
vohanka.stanisla@fnbrno.cz
Site Name
Nemocnice Pardubickeho kraje a.s.
Department Name
Neurologie
Principal Investigator Name
Edvard Ehler
Principal Investigator Email
edvard.ehler@nempk.cz
Contact Person Name
Edvard Ehler
Contact Person Email
edvard.ehler@nempk.cz

Greece

Earliest CTIS Part Ii Submission Date
19-11-2024
Latest Decision Or Authorization Date
06-03-2025
Processing Time Days
107
Number Of Sites
2
Number Of Participants
10

Sites

Site Name
General University Hospital Of Patras
Department Name
Neurology Clinic
Principal Investigator Name
Elisabeth Chroni
Principal Investigator Email
echroni@yahoo.com
Contact Person Name
Elisabeth Chroni
Contact Person Email
echroni@yahoo.com
Site Name
Eginitio Hospital
Department Name
1st Neurology Department
Principal Investigator Name
Vasiliki Zouvelou
Principal Investigator Email
vzouvelu@med.uoa.gr
Contact Person Name
Vasiliki Zouvelou
Contact Person Email
vzouvelu@med.uoa.gr

France

Earliest CTIS Part Ii Submission Date
21-02-2025
Latest Decision Or Authorization Date
10-03-2025
Processing Time Days
17
Number Of Sites
4
Number Of Participants
16

Sites

Site Name
Fondation A De Rothschild
Department Name
Service de Neurologie
Principal Investigator Name
Antoine GUEGUEN
Principal Investigator Email
agueguen@for.paris
Contact Person Name
Antoine GUEGUEN
Contact Person Email
agueguen@for.paris
Site Name
Centre Hospitalier Universitaire De Dijon
Department Name
Service Neurologie
Principal Investigator Name
Christelle BLANC-LABARRE
Principal Investigator Email
christelle.blanc@chu-dijon.fr
Contact Person Name
Christelle BLANC-LABARRE
Contact Person Email
christelle.blanc@chu-dijon.fr
Site Name
Quinze-Vingts National Ophthalmology Hospital
Department Name
Service de Médecine Interne
Principal Investigator Name
Jennifer ABOAB
Principal Investigator Email
jaboab@15-20.fr
Contact Person Name
Jennifer ABOAB
Contact Person Email
jaboab@15-20.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Service Neurologie
Principal Investigator Name
Saskia BRESCH
Principal Investigator Email
bresch.s@chu-nice.fr
Contact Person Name
Saskia BRESCH
Contact Person Email
bresch.s@chu-nice.fr

Poland

Earliest CTIS Part Ii Submission Date
26-02-2025
Latest Decision Or Authorization Date
09-03-2025
Processing Time Days
11
Number Of Sites
5
Number Of Participants
32

Sites

Site Name
Clinirem Sp. z o.o.
Principal Investigator Name
Urszula Chyrchel-Paszkiewicz
Principal Investigator Email
pgluchyrchel@gmail.com
Contact Person Name
Urszula Chyrchel-Paszkiewicz
Contact Person Email
pgluchyrchel@gmail.com
Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Department Name
MICS Centrum Medyczne Bydgoszcz
Principal Investigator Name
Łukasz Rzepiński
Principal Investigator Email
luk.rzepinski@gmail.com
Contact Person Name
Łukasz Rzepiński
Contact Person Email
luk.rzepinski@gmail.com
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Zespół Poradni Specjalistycznych - Botaniczna 3, Poradnia Neurologiczna
Principal Investigator Name
Agnieszka Słowik
Principal Investigator Email
neurologiabk@su.krakow.pl
Contact Person Name
Agnieszka Słowik
Contact Person Email
neurologiabk@su.krakow.pl
Site Name
Neurologia Śląska Centrum Medyczne
Principal Investigator Name
Marek Śmiłowski
Principal Investigator Email
marek.smilowski@neurologiaslaska.pl
Contact Person Name
Marek Śmiłowski
Site Name
Krakowska Akademia Neurologii Sp. z o.o.
Principal Investigator Name
Andrzej Szczudlik
Principal Investigator Email
Andrzej.Szczudlik@neurologia.org.pl
Contact Person Name
Andrzej Szczudlik

Sponsor

Primary sponsor

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

Contract research organisations

Name
PPD Development LP / PPD International / PPD Global Ltd. / PPD Denmark Filial Af PPD Scandinavia AB Sverige
Responsibilities
Vendor management, patient recruitment, monitoring/regulatory, project management (roles listed under sponsor third parties)
Name
IQVIA Limited
Responsibilities
ICSR processing and other clinical trial support duties
Name
WCG Clinical Inc.
Responsibilities
Online training portal and organisation of Virtual Investigator meeting
Name
Fisher Clinical Services GmbH
Responsibilities
IMP storage and distribution
Name
Eresearchtechnology Inc.
Responsibilities
Central ECG and eCOA

Third parties

  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central ECG and eCOA","organisation_type":"Pharmaceutical company"}
  • {"country":"Denmark","full_name":"PPD Denmark Filial Af PPD Scandinavia AB Sverige","duties_or_roles":"Code '1' (role code present in CTIS; specific text not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"ICSR processing; additional duties coded in sponsor duties","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Role coded '4' (specific text not provided in listing)","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":"PPD Development LP","duties_or_roles":"Vendor management, Patient recruitment; and other coded duties","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"Fisher Clinical Services GmbH","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":"Role coded '4' (specific text not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Role coded '4' (specific text not provided)","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"Medical Data Review, and DSMB; other roles coded","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"Project management duties and monitoring/regulatory","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Vyvgart 1 000 mg solution for injection in pre-filled syringe
Active Substance
EFGARTIGIMOD ALFA
Modality
Monoclonal antibody
Routes Of Administration
Subcutaneous injection
Route
SUBCUTANEOUS INJECTION
Authorisation Status
Authorised (marketing authorisation EU/1/22/1674/003)
Orphan Designation
Yes
Maximum Dose
1000 mg (maxDailyDoseAmount)
Investigational Product Name
Placebo PH20 SC
Modality
Other

Related trials

Other published trials that may interest you.