Clinical trial • Phase III • Neurology

GEFURULIMAB for Generalized myasthenia gravis (acetylcholine receptor antibody positive)

Phase III trial of GEFURULIMAB for Generalized myasthenia gravis (acetylcholine receptor antibody positive).

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Generalized myasthenia gravis (acetylcholine receptor antibody positive)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody

Key dates

Initial CTIS Submission Date
10-04-2024
First CTIS Authorization Date
13-05-2024

Trial design

Randomised, gefurulimab (alxn1720) versus alxn1720 placebo product (placebo-controlled). dose and dosing schedule are not specified in the ctis record provided. Phase III trial across 52 sites in Portugal, France, Italy and others.

Randomised
Yes
Comparator
Gefurulimab (ALXN1720) versus ALXN1720 Placebo Product (placebo-controlled). Dose and dosing schedule are not specified in the CTIS record provided.
Target Sample Size
151
Trial Duration For Participant
182

Eligibility

Recruits 151 Vulnerable population selected. Informed consent is required from adult participants (minimum age ≥ 18). Subject Information Sheets and Informed Consent Forms are provided for Adults, Caregivers and Pregnant Partners. Multiple language translations of consent documents are available (English, French, German, Italian, Portuguese, Spanish, Dutch, Polish). No assent procedures for minors are provided because minimum age is 18..

Pregnancy Exclusion
Pregnancy, breastfeeding, or intention to conceive during the course of the study
Vulnerable Population
Vulnerable population selected. Informed consent is required from adult participants (minimum age ≥ 18). Subject Information Sheets and Informed Consent Forms are provided for Adults, Caregivers and Pregnant Partners. Multiple language translations of consent documents are available (English, French, German, Italian, Portuguese, Spanish, Dutch, Polish). No assent procedures for minors are provided because minimum age is 18.

Inclusion criteria

  • {"criterion_text":"- Must be ≥ 18 years of age at the time of signing the informed consent\n- \"Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV \"\n- Positive serological test for autoantibodies against AChR."}

Exclusion criteria

  • {"criterion_text":"- History of thymectomy or any other thymic surgery within 12 months prior to Screening\n- Untreated thymic malignancy, carcinoma, or thymoma\n- History of Neisseria meningitidis infection\n- Pregnancy, breastfeeding, or intention to conceive during the course of the study"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Week 26","definition_or_measurement_approach":"Change from baseline in MG-ADL total score measured at Week 26."}

Secondary endpoints

  • {"endpoint_text":"- Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Week 26\n- Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26\n- Percentage of Responders based on Reduction of the QMG Total Score at Week 26\n- Change From Baseline in Myasthenia Gravis Composite (MGC) Total Score at Week 26\n- Change from Baseline in the QMG total score at Week 4","definition_or_measurement_approach":"QMG at Week 26: change from baseline in QMG total score measured at Week 26. MG-ADL responder: percentage of patients achieving predefined reduction in MG-ADL total score at Week 26. QMG responder: percentage achieving predefined reduction in QMG total score at Week 26. MGC: change from baseline in MGC total score at Week 26. QMG at Week 4: change from baseline in QMG total score measured at Week 4."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
151
Recruitment Window Months
56
Consent Approach
Informed consent is required from adult participants (minimum age ≥ 18). Subject Information Sheets and Informed Consent Forms (ICFs) are provided for Adults, Caregivers, and Pregnant Partners. ICFs and related subject materials are available in multiple languages including English, French, German, Italian, Portuguese, Spanish, Dutch and Polish as per the document list.

Methods

  • K1_Recruitment arrangements (site-specific recruitment arrangements / recruitment plan documents)
  • K2_Recruitment material_Landing page website (website/landing page listings)
  • K2_Recruitment material_PAG Slides (patient advocacy group presentation slides)
  • K2_Recruitment material_Study Fact Sheet (study fact sheets distributed to sites/patients)
  • K2_Recruitment material_Poster / Study Poster (printed posters at sites and public spaces)
  • K2_Recruitment material_Patient Brochure (patient brochures / information leaflets)
  • K2_Recruitment material_Half Page Ad (advertising materials)
  • K2_Recruitment material_Community Letter (community outreach letters)
  • Website Listing / Digital listings (K2_Recruitment material Website Listing)

Geography

Total Number Of Sites
52
Total Number Of Participants
92

Portugal

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
27-04-2026
Processing Time Days
728
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Unidade Local De Saude De Almada-Seixal E.P.E.
Department Name
Neurologia
Principal Investigator Name
Pedro Pereira
Principal Investigator Email
pedro.m.pereira@hgo.min-saude.pt
Contact Person Name
Pedro Pereira
Site Name
Unidade Local De Saude De Santo Antonio E.P.E.
Department Name
Neurociencias
Principal Investigator Name
Ernestina Santos
Contact Person Name
Ernestina Santos

France

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
27-04-2026
Processing Time Days
728
Number Of Sites
7
Number Of Participants
10

Sites

Site Name
Raymond-Poincare Hospital
Department Name
Service de Réanimation Médicochirurgicale
Principal Investigator Name
Djillali ANNANE
Principal Investigator Email
djillali.annane@aphp.fr
Contact Person Name
Djillali ANNANE
Contact Person Email
djillali.annane@aphp.fr
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Service Explorations Neurologiques
Principal Investigator Name
Elisa DE LA CRUZ
Principal Investigator Email
e-delacruz@chu-montpellier.fr
Contact Person Name
Elisa DE LA CRUZ
Contact Person Email
e-delacruz@chu-montpellier.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Département de Neurologie
Principal Investigator Name
Aleksandra NADAJ-PAKLEZA
Contact Person Name
Aleksandra NADAJ-PAKLEZA
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Centre de référence des Maladies neuromusculaires et de la SLA/ Pôle des Neurosciences Cliniques
Principal Investigator Name
Shahram ATTARIAN
Principal Investigator Email
shahram.attarian@ap-hm.fr
Contact Person Name
Shahram ATTARIAN
Contact Person Email
shahram.attarian@ap-hm.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Département de Neurologie A
Principal Investigator Name
Céline TARD
Principal Investigator Email
celine.tard@chu-lille.fr
Contact Person Name
Céline TARD
Contact Person Email
celine.tard@chu-lille.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Service de Neurologie
Principal Investigator Name
Sophie DEMERET
Principal Investigator Email
sophie.demeret@aphp.fr
Contact Person Name
Sophie DEMERET
Contact Person Email
sophie.demeret@aphp.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
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

Italy

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
28-04-2026
Processing Time Days
729
Number Of Sites
12
Number Of Participants
15

Sites

Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Clinica Neurologica II - Dipartimento di Scienze Neurologica
Principal Investigator Name
Matteo Garibaldi
Principal Investigator Email
matteo.garibaldi@uniroma1.it
Contact Person Name
Matteo Garibaldi
Contact Person Email
matteo.garibaldi@uniroma1.it
Site Name
Universita Degli Studi Di Napoli Federico II
Department Name
Dipartimento Di Neuroscienze E Scienze Riproduttive Ed Odontostomatologiche
Principal Investigator Name
Francesco Saccà
Principal Investigator Email
francesco.sacca@unina.it
Contact Person Name
Francesco Saccà
Contact Person Email
francesco.sacca@unina.it
Site Name
Azienda Unita Locale Socio Sanitaria N. 2 Marca Trevigiana
Department Name
Bonifati
Principal Investigator Name
Domenico Marco Bonifati
Principal Investigator Email
domenicomarco.bonifati@aulss2.veneto.it
Contact Person Name
Domenico Marco Bonifati
Site Name
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Department Name
UOSD di Neurofisiopatologia
Principal Investigator Name
Filippo Brighina
Principal Investigator Email
filippobrighina@gmail.com
Contact Person Name
Filippo Brighina
Contact Person Email
filippobrighina@gmail.com
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
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
UOC Neurologia
Principal Investigator Name
Raffaele Iorio
Principal Investigator Email
raffaele.iorio@policlinicogemelli.it
Contact Person Name
Raffaele Iorio
Site Name
Azienda Sanitaria Universitaria Friuli Centrale
Department Name
Clinica Neurologica e di Neuroriabilitazione
Principal Investigator Name
Mariarosaria Valente
Principal Investigator Email
mariarosaria.valente@uniud.it
Contact Person Name
Mariarosaria Valente
Contact Person Email
mariarosaria.valente@uniud.it
Site Name
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Department Name
UO Neurologia II
Principal Investigator Name
Alessandro Padovani
Principal Investigator Email
alessandro.padovani@unibs.it
Contact Person Name
Alessandro Padovani
Contact Person Email
alessandro.padovani@unibs.it
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
UOC Neurologia IV
Principal Investigator Name
Rita Frangiamore
Principal Investigator Email
rita.frangiamore@istituto-besta.it
Contact Person Name
Rita Frangiamore
Site Name
Azienda Ospedaliero Universitaria Pisana
Department Name
Medicina Clinica e Sperimentale – UO Neurologia
Principal Investigator Name
Michelangelo Maestri Tassoni
Principal Investigator Email
m.maestri@ao-pisa.toscana.it
Contact Person Name
Michelangelo Maestri Tassoni
Contact Person Email
m.maestri@ao-pisa.toscana.it
Site Name
Azienda Ospedaliera Papa Giovanni XXIII
Department Name
UO Neurologia
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
Careggi University Hospital
Department Name
Dip di Neurofarba – Neurologia 2
Principal Investigator Name
Valentina Damato
Principal Investigator Email
valentina.damato@unifi.it
Contact Person Name
Valentina Damato
Contact Person Email
valentina.damato@unifi.it

Denmark

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
28-04-2026
Processing Time Days
729
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Aalborg University Hospital
Department Name
Department of Neurology
Principal Investigator Name
Izabella Obal
Principal Investigator Email
i.obal@rn.dk
Contact Person Name
Izabella Obal
Contact Person Email
i.obal@rn.dk
Site Name
Rigshospitalet
Department Name
Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center
Principal Investigator Name
Nicolai Rasmus Preisler
Principal Investigator Email
nicolai.rasmus.preisler@regionh.dk
Contact Person Name
Nicolai Rasmus Preisler

Poland

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
30-04-2026
Processing Time Days
731
Number Of Sites
6
Number Of Participants
27

Sites

Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Department Name
MICS Centrum Medyczne Bydgoszcz
Principal Investigator Name
Lukasz Rzepinski
Principal Investigator Email
luk.rzepinski@gmail.com
Contact Person Name
Lukasz Rzepinski
Contact Person Email
luk.rzepinski@gmail.com
Site Name
Clinical Research Center Sp. z o.o. Medic-R sp.k.
Department Name
Clinical Research Center Sp. z o.o. Medic-R sp.k.
Principal Investigator Name
Agnieszka Adamczak-Ratajczak
Principal Investigator Email
agnieszka.adamczak-ratajczak@cr-center.pl
Contact Person Name
Agnieszka Adamczak-Ratajczak
Site Name
Krakowska Akademia Neurologii Sp. z o.o.
Department Name
Centrum Neurologii Klinicznej
Principal Investigator Name
Andrzej Szczudlik
Principal Investigator Email
andrzej.szczudlik@neurologia.org.pl
Contact Person Name
Andrzej Szczudlik
Site Name
Neurologia Slaska Centrum Medyczne
Department Name
Neurologia Slaska Centrum Medyczne
Principal Investigator Name
Marek Smilowski
Principal Investigator Email
kontakt@neurologiaslaska.pl
Contact Person Name
Marek Smilowski
Contact Person Email
kontakt@neurologiaslaska.pl
Site Name
Niepubliczny Zaklad Opieki Zdrowotnej Neuromed M. I M. Nastaj. sp.p.
Department Name
NZOZ Neuromed M. I M. Nastaj. sp.p.
Principal Investigator Name
Marcin Nastaj
Principal Investigator Email
marcinnastaj@gmail.com
Contact Person Name
Marcin Nastaj
Contact Person Email
marcinnastaj@gmail.com
Site Name
Krakowski Szpital Specjalistyczny Im. Sw. Jana Pawla II
Department Name
Odd Neurologiczny z Pododdzialem Udarowym i z Pododdzialem Rehabilitacji Neurologicznej
Principal Investigator Name
Agnieszka Kulaga
Principal Investigator Email
kulaga_a@poczta.onet.pl
Contact Person Name
Agnieszka Kulaga
Contact Person Email
kulaga_a@poczta.onet.pl

Spain

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
30-04-2026
Processing Time Days
731
Number Of Sites
14
Number Of Participants
14

Sites

Site Name
Hospital Germans Trias I Pujol
Department Name
Neurología
Principal Investigator Name
Alicia Martínez Piñeiro
Principal Investigator Email
amartinezp.germanstrias@gencat.cat
Contact Person Name
Alicia Martínez Piñeiro
Site Name
Hospital Clinico San Carlos
Department Name
Neurología
Principal Investigator Name
Alejandro Horga Hernández
Principal Investigator Email
ahorga@gmail.com
Contact Person Name
Alejandro Horga Hernández
Contact Person Email
ahorga@gmail.com
Site Name
Bellvitge University Hospital
Department Name
Neurología
Principal Investigator Name
Carlos Casasnovas Pons
Principal Investigator Email
carloscasasnovas@bellvitgehospital.cat
Contact Person Name
Carlos Casasnovas Pons
Site Name
Hospital Universitario Virgen De La Macarena
Department Name
Neurología
Principal Investigator Name
Eva María Martínez Fernández
Principal Investigator Email
evam.martinez.sspa@juntadeandalucia.es
Contact Person Name
Eva María Martínez Fernández
Site Name
Clinica Universidad De Navarra
Department Name
Neurología
Principal Investigator Name
Jaime Gállego Pérez de Larraya
Principal Investigator Email
jgallego@unav.es
Contact Person Name
Jaime Gállego Pérez de Larraya
Contact Person Email
jgallego@unav.es
Site Name
Hospital Universitario Reina Sofia
Department Name
Neurología
Principal Investigator Name
Eduardo Agüera Morales
Principal Investigator Email
doctoredu@gmail.com
Contact Person Name
Eduardo Agüera Morales
Contact Person Email
doctoredu@gmail.com
Site Name
Hospital Universitario Infanta Sofía
Department Name
Neurología
Principal Investigator Name
Gerardo Gutierrez Gutierrez
Principal Investigator Email
g3.neuro@gmail.com
Contact Person Name
Gerardo Gutierrez Gutierrez
Contact Person Email
g3.neuro@gmail.com
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Neurología
Principal Investigator Name
Rodrigo Álvarez Velasco
Principal Investigator Email
rodrigo.alvarez@salud.madrid.org
Contact Person Name
Rodrigo Álvarez Velasco
Site Name
Hospital Universitario La Paz
Department Name
Neurología
Principal Investigator Name
Francisco Javier Rodríguez de Rivera Garrido
Principal Investigator Email
frriveragarrido@salud.madrid.org
Contact Person Name
Francisco Javier Rodríguez de Rivera Garrido
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurología
Principal Investigator Name
Virginia Reyes Garrido
Principal Investigator Email
virginiareyesgarrido@gmail.com
Contact Person Name
Virginia Reyes Garrido
Contact Person Email
virginiareyesgarrido@gmail.com
Site Name
Hospital Universitario La Paz (duplicate entry not provided separately)
Department Name
Neurología
Site Name
Hospital Germans Trias I Pujol (additional entry)
Site Name
Bellvitge University Hospital (additional entry)

Germany

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
30-04-2026
Processing Time Days
731
Number Of Sites
5
Number Of Participants
14

Sites

Site Name
Ludwig Maximilian University Of Munich
Department Name
Friedrich-Bauer-Institut Neurologische Klinik und Poliklinik
Principal Investigator Name
Stephan Wenninger
Principal Investigator Email
stephan.wenninger@med.uni-muenchen.de
Contact Person Name
Stephan Wenninger
Site Name
Klinikum Wuerzburg Mitte gGmbH
Department Name
Klinikum für Neurologie
Principal Investigator Name
Mathias Mäurer
Principal Investigator Email
mathias.maeurer@kwm-klinikum.de
Contact Person Name
Mathias Mäurer
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Neurologie
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
Klinikum rechts der Isar der TU Muenchen AöR
Department Name
Klinik und Poliklinik für Neurologie
Principal Investigator Name
Achim Berthele
Principal Investigator Email
achim.berthele@tum.de
Contact Person Name
Achim Berthele
Contact Person Email
achim.berthele@tum.de
Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
Klinik für Neurologie
Principal Investigator Name
Michael Gliem
Principal Investigator Email
michael.gliem@med.uni-duesseldorf.de
Contact Person Name
Michael Gliem

Austria

Earliest CTIS Part Ii Submission Date
29-04-2024
Latest Decision Or Authorization Date
28-04-2026
Processing Time Days
729
Number Of Sites
3
Number Of Participants
2

Sites

Site Name
Klinikum Wels-Grieskirchen GmbH
Department Name
Neurologie
Principal Investigator Name
Raffi Topakian
Principal Investigator Email
Raffi.Topakian@klinikum-wegr.at
Contact Person Name
Raffi Topakian
Site Name
Allgemeines Krankenhaus Der Stadt Wien Universitatskliniken
Department Name
Universitätsklinik für Neurologie
Principal Investigator Name
Fritz Zimprich
Principal Investigator Email
friedrich.zimprich@meduniwien.ac.at
Contact Person Name
Fritz Zimprich
Site Name
Medizinische Universitaet Innsbruck
Department Name
Universitätsklinik für Neurologie
Principal Investigator Name
Wolfang Löscher
Principal Investigator Email
wolfgang.loescher@i-med.ac.at
Contact Person Name
Wolfang Löscher
Contact Person Email
wolfgang.loescher@i-med.ac.at

Sponsor

Primary sponsor

Full Name
Alexion Pharmaceuticals Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
IQVIA Limited
Responsibilities
Medical Monitoring; site payments; Regulatory; Product Complaint Call Center

Third parties

  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Medical Monitoring; site payments; Regulatory; Product Complaint Call Center","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Gefurulimab
Active Substance
GEFURULIMAB
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS INJECTION
Route
SUBCUTANEOUS INJECTION
Authorisation Status
Investigational (clinical trial product)
Investigational Product Name
ALXN1720 Placebo Product
Modality
Other
Authorisation Status
Placebo (for trial use)

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