Study on the Effects of Remibrutinib for Patients with Generalized Myasthenia Gravis

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What is this study about?

This clinical trial is focused on studying a condition called generalized myasthenia gravis, a chronic autoimmune disease that causes muscle weakness. The study will evaluate a treatment using a medication called remibrutinib, which is taken as a film-coated tablet. Remibrutinib works by targeting and inhibiting a specific enzyme in the body known as Bruton’s tyrosine kinase, which plays a role in the immune system.

The purpose of the study is to assess how effective, safe, and tolerable remibrutinib is for patients with generalized myasthenia gravis. Participants in the study will be randomly assigned to receive either remibrutinib or a placebo, which looks like the medication but does not contain the active ingredient. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This phase of the study will last for six months, after which there will be an open-label extension phase where all participants may receive remibrutinib.

Throughout the study, the main focus will be on changes in the participants’ ability to perform daily activities, as measured by a specific scale called the Myasthenia Gravis Activity of Daily Living (MG-ADL) scale. The study will also monitor other health indicators and any side effects that may occur. The goal is to determine if remibrutinib can help reduce the symptoms of generalized myasthenia gravis and improve the quality of life for those affected by this condition.

1 joining the study

Upon joining the study, you will be randomly assigned to receive either the study medication, remibrutinib, or a placebo. A placebo is a tablet that looks like the medication but does not contain the active ingredient.

The study is double-blind, meaning neither you nor the study team will know which treatment you are receiving to ensure unbiased results.

2 taking the medication

You will take the study medication or placebo orally in the form of a film-coated tablet. The exact dosage and frequency will be provided by the study team.

The treatment period lasts for 6 months, during which you will continue taking the medication as instructed.

3 monitoring and assessments

Throughout the study, your health and response to the treatment will be closely monitored. This includes regular visits to the study site for assessments.

You will undergo various tests and evaluations to measure the effectiveness of the treatment, such as the Myasthenia Gravis Activity of Daily Living (MG-ADL) scale, which assesses your ability to perform daily activities.

4 end of treatment phase

At the end of the 6-month treatment phase, your progress will be evaluated to determine any changes in your condition.

You may be eligible to enter an open-label extension phase, where all participants receive the active medication, remibrutinib, regardless of their initial group assignment.

5 open-label extension phase

If you choose to participate in the open-label extension phase, you will receive remibrutinib and continue to be monitored for safety and effectiveness.

This phase allows for further observation of the medication’s long-term effects on your condition.

Who Can Join the Study?

Adults aged 18 to 75 years with Generalized Myasthenia Gravis (gMG).
Confirmed diagnosis of MGFA Class II-IV gMG at screening, and not likely to need a respirator during the study, as judged by the study doctor.
Diagnosis of gMG must be supported by at least one of the following tests for patients with positive antibodies (AChR+ or MuSK+) or at least two tests for patients without these antibodies (double sero-negative):
- History of abnormal muscle response shown by tests like single-fiber electromyography or repetitive nerve stimulation.
- History of a positive response to short-acting drugs that improve muscle strength, such as neostigmine or edrophonium chloride.
- Improvement in MG symptoms when taking oral drugs that enhance muscle strength, as assessed by the treating doctor.
Documented positive test for AChR+ antibody or MuSK+ antibody at screening, or negative for both antibodies at screening.
Baseline score of 6 or higher on the MG-ADL scale, with at least 50% of the score due to symptoms other than eye-related ones.
Currently receiving at least one of the following treatments for gMG and have been on a stable dose for the specified time before the study starts:
- One non-steroidal immunosuppressant treatment for at least 6 months.
- Acetylcholinesterase inhibitors for at least 1 month on a stable dose.
- Steroids for at least 4 months, including inability to reduce the dose to an acceptable level.
Able to safely swallow the study medication, as determined by a swallowing test conducted by the study doctor at both the screening and start of the study.

Who Cannot Join the Study?

Patients with any other serious health condition that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients who have a history of drug or alcohol abuse within the past year.
Patients who have had a recent infection that required treatment with antibiotics.
Patients who have received a live vaccine within the last 4 weeks.
Patients who have a known allergy to the study medication or its ingredients.
Patients who have a history of cancer, except for certain types of skin cancer.
Patients who have uncontrolled high blood pressure.
Patients who have a history of heart attack or stroke within the last 6 months.
Patients who have severe liver or kidney disease.
Patients who have a history of certain autoimmune diseases, other than Generalized Myasthenia Gravis.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Technische Universitaet Dresden	Dresden	Germany
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Miejskie Centrum Medyczne Im. Dr. Karola Jonschera W Lodzi	Lodz	Poland
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Oncopole Claudius Regaud	Toulouse	France
Katholieke Universiteit te Leuven	Leuven	Belgium
Galen Clinic	Lublin	Poland
Neurologia Slaska Centrum Medyczne	Katowice	Poland

Other Sites

Site Name	City	Country
Mtz Clinical Research Powered By Pratia	Warsaw	Poland
Clinical Research Center Sp. z o.o. Medic-R sp.k.	Poznan	Poland
Klinikum Oberberg GmbH	Gummersbach	Germany
Krakowska Akademia Neurologii Sp. z o.o.	Cracow	Poland
Fondation A De Rothschild	Paris	France
Azienda Ospedaliero-Universitaria San Luigi Gonzaga	Orbassano	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Hospital Universitari Arnau De Vilanova De La Gerencia Territorial De Lleida	Lleida	Spain
Centre Hospitalier Universitaire De Nice	Nice	France
Spitalul Clinic Judetean De Urgenta Sibiu	Sibiu	Romania
Neurocentrum Bydgoszcz Sp. z o.o.	Bydgoszcz	Poland
Hopital Beaujon	Clichy	France
Neuroprotect Sp. z o.o.	Warsaw	Poland
Galen Clinic	Warsaw	Poland
Centrum Wsparcia Badań Klinicznych PUM w Szczecinie	Szczecin	Poland
LUX MED Sp. z o.o.	Warsaw	Poland
Centrum Medyczne Hope Clinic Sebastian Szklener	Lublin	Poland
Njrxux Sqv z oray	Rzeszow	Poland
Uuijmgnqmy Mcjgqjg Crobwg Hebtcgvxpkzfdeduh	Hamburg	Germany
Cxsrkwj Myrdvpc Do Djxikzzwfj Se Tikqagdez Artolgmsc Nncaqz Sqzmgx	Brasov	Romania
Nsdozkqhd Siygys	Bucharest	Romania
Mkpx Cunvekq Mlyypgty Bemzucadb	Bydgoszcz	Poland
Azupqpk Sjv z orls	Poznan	Poland
Atvqgihou Usl	Amsterdam	The Netherlands
Uwspjzxnbdwlbbjmnqfal Mxujghgd Auq	Munster	Germany
Grssuz Utognrowrb Feiohhyuq	Frankfurt	Germany
Acjcuai Olrkzcjgort Pvuh Gxiqpkkh Xtnlt	Bergamo	Italy
Hwepthzi Vcza dtteygpz	Barcelona	Spain
Huseuuxu Uzakzwsfghdrnf Sqcpjdkvut &remtpb Hgziknc dt Hsyhqofhhib	STRASBOURG, Alsace	France
Ixcmedlzjeqp Ppqzkyii Ltypjeow Pddtb de hrzc nm mxnv Kqrwrr Rhphxm	Lublin	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Not yet recruiting	23.05.2025
France	Recruiting	23.05.2025
Germany	Recruiting	23.05.2025
Italy	Recruiting	23.05.2025
Poland	Recruiting	23.05.2025
Romania	Recruiting	23.05.2025
Spain	Recruiting	23.05.2025
The Netherlands	Not yet recruiting	23.05.2025

Trial locations

Investigated drugs:

Remibrutinib

Remibrutinib is a medication being studied for its potential to help people with generalized myasthenia gravis, a condition that causes muscle weakness. This medication works by targeting specific parts of the immune system that may be involved in causing the symptoms of this condition. The goal of using remibrutinib in this trial is to see if it can reduce the severity of symptoms and improve the daily living activities of patients over a period of six months. By participating in this study, researchers hope to determine if remibrutinib is effective and safe for people with this condition.

Investigated diseases:

Myasthenia gravis

Generalized Myasthenia Gravis – Generalized Myasthenia Gravis is an autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the body’s own tissues, specifically targeting the acetylcholine receptors at the neuromuscular junction. This disruption impairs the transmission of nerve impulses to muscles, causing symptoms such as drooping eyelids, double vision, and difficulty swallowing. Muscle weakness in this condition typically worsens with activity and improves with rest. Over time, the weakness can spread to other muscle groups, including those involved in breathing and limb movement. The progression of symptoms can vary widely among individuals, with periods of exacerbation and remission.

Trial ID:

2023-510026-32-00

Protocol code:

CLOU064O12301

NCT ID:

NCT06744920

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effects of Remibrutinib for Patients with Generalized Myasthenia Gravis

What is this study about?

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