Clinical trial • Phase II • Immunology | Neurology

EMPASIPRUBART for AChR-Ab Seropositive Generalized Myasthenia Gravis | Myasthenia gravis

Phase II trial of EMPASIPRUBART for AChR-Ab Seropositive Generalized Myasthenia Gravis | Myasthenia gravis.

Overview

Trial Therapeutic Area
Immunology | Neurology
Trial Disease
AChR-Ab Seropositive Generalized Myasthenia Gravis | Myasthenia gravis
Trial Stage
Phase II
Drug Modality
Monoclonal antibody | Other antibody
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
15-10-2025
First CTIS Authorization Date
18-02-2026

Trial design

open-label, efgartigimod iv (monotherapy) versus efgartigimod iv + empasiprubart (argx-117) iv (add-on); dosing and schedule not specified in the public record-controlled Phase II trial across 19 sites in Italy, Spain, Belgium and others.

Open Label
Yes
Comparator
Efgartigimod IV (monotherapy) versus Efgartigimod IV + Empasiprubart (ARGX-117) IV (add-on); dosing and schedule not specified in the public record
Target Sample Size
18

Eligibility

Recruits 18 Vulnerable population considerations present. The registry shows isVulnerablePopulationSelected=true and subject information / informed consent forms include specific pregnancy and newborn / pregnant participant and partner ICFs (e.g. files titled "Pregnant_Participant_ICF", "Pregnancy-and-Birth-ICF", and country-specific pregnancy ICFs). Consent is obtained from adult participants (minimum age 18 as per inclusion criteria); separate ICFs for pregnancy and partner information are provided..

Vulnerable Population
Vulnerable population considerations present. The registry shows isVulnerablePopulationSelected=true and subject information / informed consent forms include specific pregnancy and newborn / pregnant participant and partner ICFs (e.g. files titled "Pregnant_Participant_ICF", "Pregnancy-and-Birth-ICF", and country-specific pregnancy ICFs). Consent is obtained from adult participants (minimum age 18 as per inclusion criteria); separate ICFs for pregnancy and partner information are provided.

Inclusion criteria

  • {"criterion_text":"- MP Main inclusion criteria:\n- o\tIs at least 18 years of age and the local legal age of consent for clinical studies\n- o\tHas been diagnosed with MG consistent with MG's clinical features and with symptom severity appropriate for currently enrolling ISAs, per the investigator’s clinical judgment\n- o\tIf receiving MG therapy, including nonsteroidal immunosuppressive drugs (NSIDs), corticosteroids, or acetylcholinesterase (AChE) inhibitors either in combination or alone, the participant should receive a stable dosage before master protocol screening\n- ISA 1 Main inclusion criteria:\n- - Is seropositive for anti-acetylcholine receptor antibodies (AChR-Ab).\n- - Has confirmed diagnosis of gMG and is Myasthenia Gravis Foundation of America (MGFA) Class II, III, IVa, or IVb.\n- - Has documented immunization against encapsulated bacterial pathogens (Neisseria meningitidis and Streptococcus pneumoniae) within 5 years of ISA screening or is willing to receive immunization at least 14 days before the first study drug administration."}

Exclusion criteria

  • {"criterion_text":"- MP Main exclusion criteria: - Known autoimmune disease or any medical condition other than the indication under study that would interfere with an accurate assessment of clinical symptoms of MG or puts the participant at undue risk. - Is MGFA (Myasthenia Gravis Foundation of America) Class V\n- ISA 1 Main exclusion criteria: - Clinical diagnosis of systemic lupus erythematosus (SLE). - Any known complement deficiency. - Current administration of a complement inhibitor or received zilucoplan or eculizumab <2 months or ravulizumab <6 months before the first study drug administration. - Patients proven to be refractory to efgartigimod (ie, not achieving a clinically meaningful improvement in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score defined as an improvement of ≥2 points)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- MP - 1.\t Incidence, severity, and relatedness of AEs and SAEs","definition_or_measurement_approach":"Assessment of incidence, severity, and relatedness of adverse events (AEs) and serious adverse events (SAEs) as recorded during the study (safety monitoring)."}
  • {"endpoint_text":"- MP - 2.\t Clinically meaningful changes in vital signs, ECGs, and laboratory parameters","definition_or_measurement_approach":"Assessment of clinically meaningful changes from baseline in vital signs, ECGs and laboratory parameters."}
  • {"endpoint_text":"- ISA 1 - 1. Incidence, severity, and relatedness of adverse events and serious adverse events in parts A and B","definition_or_measurement_approach":"Assessment of incidence, severity, and relatedness of AEs and SAEs in protocol parts A and B."}
  • {"endpoint_text":"- ISA 1 - 2. Clinically meaningful changes in vital signs, electrocardio grams, and laboratory parameters in parts A and B","definition_or_measurement_approach":"Assessment of clinically meaningful changes from baseline in vital signs, electrocardiograms and laboratory parameters in parts A and B."}

Secondary endpoints

  • {"endpoint_text":"- MP - 1. Change in MG disease severity as measured by change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score or Myasthenia Gravis Impairment Index (MGII) total score","definition_or_measurement_approach":"Change from baseline in MG-ADL total score or MGII total score."}
  • {"endpoint_text":"- MP - 2. Proportion of participants reaching minimal symptom expression (MSE) and other percentage improvements at variable time points.","definition_or_measurement_approach":"Proportion achieving minimal symptom expression (MSE) and other percentage improvements at specified time points."}
  • {"endpoint_text":"- ISA1 - 1. Myasthenia Gravis Activities of Daily Living (MG-ADL) total score change from baseline at week 18 in part B (cycle 2 day 29) compared with MG-ADL total score change from baseline at week 4 in part A","definition_or_measurement_approach":"Comparison of MG-ADL change from baseline at week 18 in part B vs change at week 4 in part A (timepoint-specified change from baseline)."}
  • {"endpoint_text":"- ISA 1 - 2. Proportion of participants reaching minimal symptom expression (MSE) at any point in part B cycles 1 and 2, and part B cycles 1 or 2","definition_or_measurement_approach":"Proportion of participants who reach MSE at any point during part B cycles 1 and 2 or in cycles 1 or 2."}
  • {"endpoint_text":"- ISA 1 - 3. MG-ADL total score actual values and changes from baseline over time in part B compared with part A","definition_or_measurement_approach":"Longitudinal MG-ADL actual values and change-from-baseline comparisons between part B and part A."}
  • {"endpoint_text":"- ISA 1 - 4. Quantitative Myasthenia Gravis (QMG) total score change from baseline at week 18 in part B (cycle 2 day 29) compared with QMG total score change from baseline at week 4 in part A","definition_or_measurement_approach":"Comparison of QMG total score change from baseline at specified timepoints (week 18 part B vs week 4 part A)."}
  • {"endpoint_text":"- ISA 1 - 5. QMG total score actual values and changes from baseline over time in part B compared with part A","definition_or_measurement_approach":"Longitudinal QMG actual values and change-from-baseline comparisons between part B and part A."}
  • {"endpoint_text":"- ISA 1 - 6. Proportion of participants who have a 50% MG-ADL total score improvement in part B cycles 1 and 2","definition_or_measurement_approach":"Proportion of participants achieving a 50% improvement in MG-ADL total score during part B cycles 1 and 2."}
  • {"endpoint_text":"- ISA 1 - 7. Proportion of participants who have positive patient acceptable symptom state (PASS) in part B cycles 1 and 2, and part B cycles 1 or 2","definition_or_measurement_approach":"Proportion of participants achieving a positive patient acceptable symptom state (PASS) in specified part B cycles."}

Recruitment

Planned Sample Size
52
Recruitment Window Months
23
Consent Approach
Informed consent is obtained from adult participants (minimum age 18). Subject information and ICF documents are provided per country and language (multiple country-specific ICFs listed: Italian, Spanish, English, French, Dutch, German, Greek, Polish, etc.). There are specific ICFs for pregnancy/partner and for optional future research where applicable.

Methods

  • Doctor-to-patient letters (K2 / Doctor Letter documents) — channel: letter via treating physicians; target audience: patients with Myasthenia Gravis; country-specific versions available (e.g. ITA, ESP, BE, DEU, GRC, PL, NLD).
  • HCP cards / HCP-Card documents — channel: healthcare professional outreach; target audience: neurologists and treating clinicians; country-specific versions available (e.g. ITA, BE, DEU, GRC, PL, NLD).
  • Patient brochures, flyers and patient letters (K2 / Patient-Brochure, Patient-Flyer, Patient-Letter documents) — channel: printed materials for patients; target audience: potential participant patients; country/language-specific materials available (e.g. ITA, ESP, BE English/French/Dutch, DEU, GRC Greek, PL Polish, NLD).
  • Recruitment arrangements (K1 documents) — channel: formal recruitment arrangement documents submitted per country; country-specific K1 documents present for Italy, Spain, Belgium, Germany, Greece, Poland, Netherlands.

Geography

Total Number Of Sites
19
Total Number Of Participants
52

Italy

Earliest CTIS Part Ii Submission Date
13-01-2026
Latest Decision Or Authorization Date
23-02-2026
Processing Time Days
41
Number Of Sites
5
Number Of Participants
6

Sites

Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Neurology
Principal Investigator Name
Raffaele Iorio
Principal Investigator Email
Raffaele.iorio@policlinicogemelli.it
Contact Person Name
Raffaele Iorio
Site Name
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii
Department Name
SC Neurology
Principal Investigator Name
Giorgia Camera
Principal Investigator Email
gcamera@asst-pg23.it
Contact Person Name
Giorgia Camera
Contact Person Email
gcamera@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
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
Department Name
UOC Neurofisiopatologia
Principal Investigator Name
Francesco Habetswallner
Principal Investigator Email
francesco.habetswallner@aocardarelli.it
Contact Person Name
Francesco Habetswallner
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

Spain

Earliest CTIS Part Ii Submission Date
13-01-2026
Latest Decision Or Authorization Date
20-02-2026
Processing Time Days
38
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Hospital De La Santa Creu I Sant Pau
Department Name
Neurology
Principal Investigator Name
Elena Cortés Vicente
Principal Investigator Email
ecortes@santpau.cat
Contact Person Name
Elena Cortés Vicente
Contact Person Email
ecortes@santpau.cat
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurology
Principal Investigator Name
Guillermina Garcia Martin
Principal Investigator Email
guille.garcia.eecc@gmail.com
Contact Person Name
Guillermina Garcia Martin
Contact Person Email
guille.garcia.eecc@gmail.com

Belgium

Earliest CTIS Part Ii Submission Date
12-01-2026
Latest Decision Or Authorization Date
18-02-2026
Processing Time Days
37
Number Of Sites
2
Number Of Participants
3

Sites

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
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

Germany

Earliest CTIS Part Ii Submission Date
15-01-2026
Latest Decision Or Authorization Date
24-02-2026
Processing Time Days
40
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Department of Neurology, Neuroscience Clinical Research Center
Principal Investigator Name
Sarah Hoffmann
Principal Investigator Email
sarah.hoffmann@charite.de
Contact Person Name
Sarah Hoffmann
Contact Person Email
sarah.hoffmann@charite.de
Site Name
Universitaetsmedizin Goettingen
Department Name
Department of Neurology
Principal Investigator Name
Jana Zschüntzsch
Principal Investigator Email
j.zschuentzsch@med.uni-goettingen.de
Contact Person Name
Jana Zschüntzsch

Greece

Earliest CTIS Part Ii Submission Date
29-10-2025
Latest Decision Or Authorization Date
24-02-2026
Processing Time Days
118
Number Of Sites
2
Number Of Participants
8

Sites

Site Name
Eginitio Hospital
Department Name
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
Site Name
University General Hospital Of Thessaloniki Ahepa
Department Name
Clinical Νeurophysiology
Principal Investigator Name
Marianthi Arnaoutoglou
Principal Investigator Email
marnaout@auth.gr
Contact Person Name
Marianthi Arnaoutoglou
Contact Person Email
marnaout@auth.gr

Poland

Earliest CTIS Part Ii Submission Date
13-01-2026
Latest Decision Or Authorization Date
22-02-2026
Processing Time Days
40
Number Of Sites
5
Number Of Participants
25

Sites

Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Department Name
MICS Centrum Medyczne Bydgoszcz
Principal Investigator Name
Łukasz Rzepiński
Principal Investigator Email
lukasz.rzepinski@mics.medicover.com
Contact Person Name
Łukasz Rzepiński
Site Name
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Department Name
Centralny Szpital Kliniczny, Klinika Neurologii
Principal Investigator Name
Anna Kostera-Pruszczyk
Principal Investigator Email
anna.kostera-pruszczyk@wum.edu.pl
Contact Person Name
Anna Kostera-Pruszczyk
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

Netherlands

Earliest CTIS Part Ii Submission Date
20-01-2026
Latest Decision Or Authorization Date
20-02-2026
Processing Time Days
31
Number Of Sites
1
Number Of Participants
2

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

Sponsor

Primary sponsor

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

Contract research organisations

Name
Pharmaceutical Product Development LLC (PPD)
Responsibilities
sponsorDuties codes include 4; vendor management and patient recruitment duties referenced (e.g. PPD Development LP duties include vendor management, patient recruitment)
Name
PPD Development LP
Responsibilities
sponsorDuties: 1, 11, 12, 13, 15 (Vendor management, Patient recruitment), 2, 5
Name
PPD International Holdings LLC / PPD Global Ltd.
Responsibilities
project management, monitoring/regulatory, site services
Name
Almac Clinical Services LLC / Almac Clinical Services Limited / Almac Clinical Services (Ireland) / Almac Pharmaceutical Services Pte. Ltd.
Responsibilities
logistics and shipment; sample handling (sponsorDuties codes: 14; 15 (Shipment))
Name
IQVIA Limited
Responsibilities
Pharmacovigilance; other study management responsibilities
Name
SGS Belgium / SGS France
Responsibilities
data safety monitoring, eCRF / IxRS, laboratory/service provisioning
Name
Endpoint Clinical Inc.
Responsibilities
audit and site oversight (sponsorDuties code: 3)
Name
Drug Development Solutions Limited
Responsibilities
laboratory/service provisioning (sponsorDuties code: 4)

Third parties

  • {"country":"Poland","full_name":"Synexus Polska Sp. z o.o.","duties_or_roles":"PPD Home care Service (sponsorDuties code 15 / value: PPD Home care Service)","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Services LLC","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"SAFIRA Clinical Research Limited","duties_or_roles":"sponsorDuties codes: 15 (eCOA consulting)","organisation_type":"Health care"}
  • {"country":"France","full_name":"SGS France","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Services LLC (additional address)","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"Singapore","full_name":"Almac Pharmaceutical Services Pte. Ltd.","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Services LLC (other addresses)","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 1, 11, 12, 13, 15 (Vendor management, Patient recruitment), 2, 5","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"sponsorDuties codes: 10; 15 (Data Safety monitoring Board, eCRF / IxRS); 6; 7","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Almac Clinical Services LLC (25 Fretz Road)","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Drug Development Solutions Limited","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"sponsorDuties codes: 15 (Project management duties and monitoring/regulatory)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Advarra Inc.","duties_or_roles":"sponsorDuties codes: 15 (Investigator Site portal)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Almac Clinical Services (Ireland) Limited","duties_or_roles":"sponsorDuties codes: 14; 15 (Shipment)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties codes: 15 (Respiratory, Cardiac safety; ECG Machine, Ancillaries & eCOA Services)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"sponsorDuties codes: 15 (Participant travel reimbursment)","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"sponsorDuties codes: 15 (Long Term sample Storage)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties codes: 15 (Pharmacovigilance); 8","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Celerion Switzerland AG","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Finland","full_name":"SYRINX Bioanalytics Oy","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
ARGX-117
Active Substance
EMPASIPRUBART
Modality
Monoclonal antibody
Routes Of Administration
IV INFUSION
Route
IV INFUSION
Authorisation Status
Authorised
Investigational Product Name
ARGX-113
Active Substance
EFGARTIGIMOD ALFA
Modality
Other antibody
Routes Of Administration
IV INFUSION
Route
IV INFUSION
Authorisation Status
Authorised
Orphan Designation
Yes
Maximum Dose
Max daily dose 1200 mg; max total dose 33600 mg
Combination Treatment
Yes

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