Clinical trial • Phase III • Neurology|Rare Disease

REMIBRUTINIB for Generalized myasthenia gravis|Myasthenia gravis

Phase III trial of REMIBRUTINIB for Generalized myasthenia gravis|Myasthenia gravis.

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Generalized myasthenia gravis|Myasthenia gravis
Trial Stage
Phase III
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
09-01-2025
First CTIS Authorization Date
07-05-2025

Trial design

Randomised, open-label, placebo to remibrutinib (lou064) 00 mg film-coated tablet (placebo arm). background standard-of-care (soc) therapies allowed/stable (per inclusion criteria). dose and schedule for remibrutinib and placebo not specified in ctis metadata.-controlled Phase III trial in Belgium, France, Romania and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo to Remibrutinib (LOU064) 00 mg film-coated tablet (placebo arm). Background standard-of-care (SOC) therapies allowed/stable (per inclusion criteria). Dose and schedule for remibrutinib and placebo not specified in CTIS metadata.
Target Sample Size
143
Trial Duration For Participant
180

Eligibility

Recruits 143 No vulnerable population selected. The study enrols adults aged 18-75 years. Informed consent is obtained from adult participants via the adult ICF; optional/extra ICFs are provided for genetic testing, pregnant participants and pregnant partners (country/language-specific ICFs available)..

Pregnancy Exclusion
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week after stopping of study treatment.
Vulnerable Population
No vulnerable population selected. The study enrols adults aged 18-75 years. Informed consent is obtained from adult participants via the adult ICF; optional/extra ICFs are provided for genetic testing, pregnant participants and pregnant partners (country/language-specific ICFs available).

Inclusion criteria

  • {"criterion_text":"- Adult patients with gMG (age 18-75 years)\n- Confirmed diagnosis of MGFA Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator.\n- The confirmation of the diagnosis of gMG should be documented and supported by ≥1 (AChR+ and MuSK+ patients) or by ≥ 2 (double sero-negative patients) of the following 3 tests: -\tHistory of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation -\tHistory of positive test with short-acting acetylcholinesterase inhibitors (e.g., neostigmine or edrophonium chloride) -\tPatient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician\n- Documented evidence of positive serologic testing for AChR+ antibody or MuSK+ antibody at screening, OR seronegative for both AChR and MuSK antibodies at screening\n- Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms\n- Participants receiving at least one of the following treatments for gMG and who have been on a stable dose for the specified time periods prior to baseline: -\tjust one non-steroidal immunosuppressant treatment (NSIST) for ≥ 6 months; and/or -\tacetylcholinesterase inhibitors for ≥ 1 month on a maximum stable dose; and/or -\tsteroids for ≥ 4 months including the inability to taper to an acceptable level\n- Able to safely swallow the study medication according to investigator clinical judgement based on a bedside swallowing test or another formal swallowing test in line with local practice, both at Screening and Baseline"}

Exclusion criteria

  • {"criterion_text":"- Prior to baseline have been treated with intravenous immunoglobulins or plasma exchange (IVIg/PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period\n- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week after stopping of study treatment."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score","definition_or_measurement_approach":"Change from baseline to Month 6 (Day 180) in the MG-ADL total score (Myasthenia Gravis Activity of Daily Living scale)."}

Secondary endpoints

  • {"endpoint_text":"- Change from baseline to Month 6 in Quantitative MG (QMG) total score","definition_or_measurement_approach":"Change from baseline to Month 6 in QMG total score."}
  • {"endpoint_text":"- Proportion of participants with ≥ 5 points reduction from baseline to Month 6 of QMG total score without rescue medication and/or strongly confounding prohibited medication","definition_or_measurement_approach":"Proportion of participants achieving ≥5-point reduction in QMG total score from baseline to Month 6, assessed excluding participants who used rescue medication and/or strongly confounding prohibited medication."}
  • {"endpoint_text":"- Proportion of participants with ≥ 3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and/or strongly confounding prohibited medication","definition_or_measurement_approach":"Proportion of participants achieving ≥3-point reduction in MG-ADL total score from baseline to Month 6, assessed without rescue medication and/or strongly confounding prohibited medication."}
  • {"endpoint_text":"- Proportion of participants achieving MSE at Month 6, defined as MG-ADL score of 0 or 1 at Month 6 without rescue therapy and/or strongly confounding prohibited medication","definition_or_measurement_approach":"Proportion achieving Minimal Symptom Expression (MSE) at Month 6 defined as MG-ADL 0 or 1 at Month 6 without rescue therapy and/or strongly confounding prohibited medication."}
  • {"endpoint_text":"- Change from baseline to Month 6 in MGC total score","definition_or_measurement_approach":"Change from baseline to Month 6 in Myasthenia Gravis Composite (MGC) total score."}
  • {"endpoint_text":"- Change from baseline to Month 6 in revised MG Quality of Life Questionnaire (MG-QOL15r) survey score","definition_or_measurement_approach":"Change from baseline to Month 6 in MG-QOL15r survey score."}
  • {"endpoint_text":"- Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating","definition_or_measurement_approach":"Safety endpoints including incidence of adverse events and changes in lab values, vital signs, ECGs, and Columbia Suicide Severity Rating assessments."}
  • {"endpoint_text":"- Proportion of time during which participants showed a reduction from baseline of ≥ 2 points in MG-ADL score that was maintained up to Month 6","definition_or_measurement_approach":"Proportion of time participants maintained ≥2-point reduction in MG-ADL from baseline up to Month 6."}
  • {"endpoint_text":"- Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of ≥ 2 points occurring by week 4）","definition_or_measurement_approach":"Proportion of participants with first MG-ADL improvement ≥2 points by Week 4 (early responders)."}
  • {"endpoint_text":"- Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L)","definition_or_measurement_approach":"Change from baseline to Month 6 in EQ-5D-5L score."}
  • {"endpoint_text":"- Proportion of participants achieving a reduction of ≥ 3 points from baseline to Month 6 in MGC total score.","definition_or_measurement_approach":"Proportion of participants with ≥3-point reduction in MGC total score from baseline to Month 6."}

Recruitment

Planned Sample Size
143
Recruitment Window Months
86
Consent Approach
Informed consent is obtained from adult participants via country/language-specific Main ICFs; optional genetic consent is covered by an Optional Genetic ICF. Pregnancy-specific ICFs and Pregnant Partner ICFs are provided where applicable. ICFs and recruitment materials are available in multiple languages (English, French, Dutch, Spanish, Italian, German, Polish, Romanian and others as per country-specific documents).

Methods

  • K1 country-specific Recruitment arrangements documents (e.g., K1_CLOU064O12301_Recruitment-Arrangements for BE, FR, ES, IT, NL, DE, PL, RO) — country-targeted recruitment materials provided.
  • K2 materials: Fact Sheet and Welcome Booklet (country/language variants) — information for potential participants and local clinicians.
  • Doctor-to-Doctor letters and Family-Doctor-letter (Italy) — local physician outreach.
  • Patient-facing ICFs and informational materials in local languages (see country-specific ICFs) to support site-level recruitment.

Geography

Total Number Of Sites
44
Total Number Of Participants
127

Belgium

Earliest CTIS Part Ii Submission Date
04-04-2025
Latest Decision Or Authorization Date
11-06-2025
Processing Time Days
68
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
UZ Leuven
Department Name
Neurologie
Contact Person Name
Kristl Claeys
Contact Person Email
kristl.claeys@uzleuven.be

France

Earliest CTIS Part Ii Submission Date
21-02-2025
Latest Decision Or Authorization Date
31-07-2025
Processing Time Days
160
Number Of Sites
7
Number Of Participants
6

Sites

Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Centre d’Investigation Clinique
Contact Person Name
Pascal CINTAS
Contact Person Email
cintas.p@chu-toulouse.fr
Site Name
Bicetre Hospital
Department Name
CRMR Neurologie
Contact Person Name
Andoni ECHANIZ-LAGUNA
Contact Person Email
andoni.echaniz-laguna@aphp.fr
Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Centre de référence des maladies neuromusculaires AOC
Contact Person Name
Guilhem SOLE
Contact Person Email
Guilhem.sole@chu-bordeaux.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Institut de Myologie Bâtiment Babinski
Contact Person Name
Anthony BEHIN
Contact Person Email
Anthony.behin@aphp.fr
Site Name
Les Hopitaux Universitaires De Strasbourg
Department Name
Centre de Référence des Maladies
Contact Person Name
Aleksandra NADAJ-PAKLEZA
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Service Système Nerveux Périphérique
Contact Person Name
Sabrina SACCONI
Contact Person Email
sacconi.s@chu-nice.fr
Site Name
Fondation A De Rothschild
Contact Person Name
Antoine Gueguen
Contact Person Email
agueguen@for.paris

Romania

Earliest CTIS Part Ii Submission Date
29-01-2025
Latest Decision Or Authorization Date
10-06-2025
Processing Time Days
132
Number Of Sites
3
Number Of Participants
6

Sites

Site Name
Neurocity S.R.L.
Department Name
Neurology
Contact Person Name
Dan-Andrei Mitrea
Contact Person Email
dan.mitrea@neuroaxis.ro
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
Centrul Medical De Diagnostic Si Tratament Ambulator Neomed S.R.L.
Department Name
Neurology
Contact Person Name
Marilena Cioropariu
Contact Person Email
marilena.cioropariu@neomed.org

Spain

Earliest CTIS Part Ii Submission Date
07-04-2025
Latest Decision Or Authorization Date
27-06-2025
Processing Time Days
81
Number Of Sites
7
Number Of Participants
10

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
Complexo Hospitalario Universitario De Santiago
Department Name
Neurology
Contact Person Name
Tania García Sobrino
Contact Person Email
ainat.tania@hotmail.com
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Contact Person Name
Teresa Sevilla Mantecón
Contact Person Email
sevilla_ter@gva.es
Site Name
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida
Department Name
Neurology
Contact Person Name
Cristina Gonzalez Mingot
Site Name
Hospital Universitario Regional De Malaga
Department Name
Neurology
Contact Person Name
Virginia Reyes Garrido
Contact Person Email
v.reyes.eecc@gmail.com
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Neurology
Contact Person Name
Rodrigo Álvarez Velasco
Site Name
Bellvitge University Hospital
Department Name
Neurology
Contact Person Name
Carlos Casasnovas Pons

Germany

Earliest CTIS Part Ii Submission Date
11-08-2025
Latest Decision Or Authorization Date
15-10-2025
Processing Time Days
65
Number Of Sites
6
Number Of Participants
6

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Department of Neurology with Experimental Neurology, Integrated Myasthenia Center, NCRC
Contact Person Name
Andreas Meisel
Contact Person Email
andreas.meisel@charite.de
Site Name
Goethe University Frankfurt
Department Name
Klinik für Neurologie
Contact Person Name
Yavor Yalachkov
Contact Person Email
Yalachkov@med.uni-frankfurt.de
Site Name
Klinikum Oberberg GmbH
Department Name
Kreiskrankenhaus Gummersbach, Neurologische Klinik
Contact Person Name
Franz Blaes
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Institut für Neuroimmunologie und Multiple Sklerose, Klinik und Poliklinik für Neurologie
Contact Person Name
Christoph Heesen
Contact Person Email
heesen@uke.de
Site Name
Technische Universitaet Dresden
Department Name
Klinik und Poliklinik für Neurologie
Contact Person Name
Jochen Schäfer
Site Name
Universitaet Muenster
Department Name
Klinik für Neurologie mit Institut für Translationale Neurologie
Contact Person Name
Jan Lünemann
Contact Person Email
Jan.Luenemann@ukmuenster.de

Italy

Earliest CTIS Part Ii Submission Date
12-08-2025
Latest Decision Or Authorization Date
12-09-2025
Processing Time Days
31
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii
Department Name
Neurologia
Contact Person Name
Sgarzi Manlio
Contact Person Email
msgarzi@asst-pg23.it
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
Clinica Neurologica II - Dipartimento di Scienze Neurologiche
Contact Person Name
Saccà Francesco
Contact Person Email
francesco.sacca@unina.it
Site Name
Azienda Ospedaliero-Universitaria San Luigi Gonzaga
Department Name
SSD Patologie Neurologiche e Specialistiche
Contact Person Name
Clerico Marinella
Contact Person Email
marinella.clerico@unito.it

Poland

Earliest CTIS Part Ii Submission Date
04-04-2025
Latest Decision Or Authorization Date
10-12-2025
Processing Time Days
250
Number Of Sites
16
Number Of Participants
90

Sites

Site Name
Galen Clinic
Contact Person Name
Urszula Chyrchel-Paszkiewicz
Contact Person Email
pgluchyrchel@gmail.com
Site Name
Aidport Sp. z o.o.
Contact Person Name
Karolina Piasecka-Stryczyńska
Contact Person Email
kpe@poczta.onet.pl
Site Name
Krakowska Akademia Neurologii Sp. z o.o.
Contact Person Name
Andrzej Szczudlik
Site Name
Clinical Research Center Sp. z o.o. Medic-R sp.k.
Contact Person Name
Artur Drużdż
Contact Person Email
artur.druzdz@crcenter.pl
Site Name
Centrum Medyczne HOPE Clinic Sebastian Szklener
Contact Person Name
Sebastian Szklener
Contact Person Email
sebastianszklener@gmail.com
Site Name
Mtz Clinical Research Powered By Pratia
Contact Person Name
Małgorzata Zajda
Contact Person Email
badacz@pratia.com
Site Name
Miejskie Centrum Medyczne Im. Dr. Karola Jonschera W Lodzi
Department Name
Oddział Neurologiczny i Udarowy
Contact Person Name
Tomasz Berkowicz
Contact Person Email
berkowt@gmail.com
Site Name
LUX MED Sp. z o.o.
Contact Person Name
Anna Kostera-Pruszczyk
Contact Person Email
akosterapruszczyk@gmail.com
Site Name
MICS Centrum Medyczne Bydgoszcz
Contact Person Name
Łukasz Rzepiński
Contact Person Email
luk.rzepinski@gmail.com
Site Name
Nmedis Sp. z o.o.
Contact Person Name
Iwona Rościszewska-Żukowska
Contact Person Email
iwona.rosciszewska@op.pl
Site Name
Centrum Wsparcia Badań Klinicznych PUM w Szczecinie
Contact Person Name
Marta Masztalewicz
Contact Person Email
marta.masztalewicz@pum.edu.pl
Site Name
Neurologia Śląska Centrum Medyczne
Contact Person Name
Marek Śmiłowski
Contact Person Email
marek.smilowski2@gmail.com
Site Name
Neurocentrum Bydgoszcz Sp. z o.o.
Contact Person Name
Robert Bonek
Contact Person Email
r.bonek@ncbydgoszcz.pl
Site Name
Indywidualna Praktyka Lekarska Prof. dr hab. n. med. Konrad Rejdak
Contact Person Name
Konrad Rejdak
Contact Person Email
krejdak@europe.com
Site Name
Galen Clinic (Warszawa)
Contact Person Name
Anna DomżałStryga
Contact Person Email
adstryga@gmail.com
Site Name
Centrum Medyczne Neuroprotect
Contact Person Name
Mariusz Grudniak

Sponsor

Primary sponsor

Full Name
Novartis Pharma AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
Parexel International (IRL) Limited
Name
PPD Global Limited
Responsibilities
Site management, develop & distribute Newsletters for PI, perform close-out visits
Name
IQVIA Limited
Responsibilities
Randomization of Patients and Management of drug supply logistics, dispensing and unblinding

Third parties

  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"PRO management, PRO licensing and translations, data collection via tablet","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"Safety tests per protocol","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac Assessment","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"SGS France","duties_or_roles":"CLOU064 PK analysis (ROW)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Meeting Protocol Worldwide LP","duties_or_roles":"Investigator meetings planner","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"PRO formatting and translations","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"Independent statistical group for Data Monitoring Committee (DMC)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"Site management, develop & distribute Newsletters for PI, perform close-out visits","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Travel patient reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Randomization of Patients and Management of drug supply logistics, dispensing and unblinding","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Somalogic Operating Co. Inc.","duties_or_roles":"Exploratory biomarkers (protein profiling on SomaScan Platform)","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Safety tests per protocol","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
LOU064
Active Substance
REMIBRUTINIB
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
oral
Investigational Product Name
Placebo to Remibrutinib (LOU064) 00 mg film-coated tablet
Modality
Other
Routes Of Administration
oral
Route
oral

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