Last Update: Oct 02, 2025
A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Remibrutinib in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLOU064O12301
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

A study to evaluate the efficacy, safety and tolerability of Remibrutinib versus placebo in adult patients with Generalized Myasthenia Gravis who are on stable, standard-of-care (SOC) treatment. This study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate the efficacy, safety and tolerability of remibrutinib in gMG patients who are on stable SOC treatment. Approximately 180 eligible participants will be randomized in a ratio of 1:1, to receive either remibrutinib or matching placebo.

The study consists of a Core Part (6-months double-blind treatment) and an Extension Part (up to 60-month open-label treatment).

Generalized Myasthenia Gravis
Phase3
Recruiting
180
Feb 07, 2025
Feb 26, 2033
All
18 Years - 75 Years (Adult, Older Adult)

Interventions

Other

Placebo

Placebo
Drug

Remibrutinib (Blinded)

Remibrutinib (Blinded) active treatment
Drug

Remibrutinib (Open Label)

Remibrutinib (Open Label) active treatment

Eligibility Criteria

Inclusion Criteria:

* Adult patients with gMG (age 18-75 years)
* Confirmed diagnosis of Myasthenia Gravis Foundation of America (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
* Documented evidence of positive serologic testing for AChR+ antibody or MuSK+ antibody at screening, OR seronegative for both AChR and MuSK antibodies at screening
* Baseline MG-ADL score ≥ 6 with ≥ 50% of the total score due to non ocular symptoms
* Participants who have been on a stable dose of standard-of-care treatment as specified in the protocol
* 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:

* 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
* 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

Other protocol-defined inclusion/exclusion criteria may apply.

Novartis Investigative Site

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Southport,4215,Australia

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Fitzroy,Victoria,3065,Australia

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Kogarah,New South Wales,2217,Australia

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Leuven,3000,Belgium

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London,Ontario,N6a 5w9,Canada

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Ottawa,Ontario,K1h 8l6,Canada

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Wuxi,Jiangsu,214023,China

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Guangzhou,Guangdong,510080,China

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Shanghai,200040,China

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Shanghai,200080,China

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Paris,75013,France

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Toulouse,31059,France

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Nice,06001,France

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Nashik,Maharashtra,422005,India

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Ludhiana,Punjab,141001,India

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Vellore,Tamil Nadu,632 004,India

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Suita,Osaka,565 0871,Japan

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Fukushima,Fukushima,960 1295,Japan

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Sapporo,Hokkaido,060-8543,Japan

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Sapporo,Hokkaido,063-0005,Japan

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Nishinomiya,Hyōgo,663 8501,Japan

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Hanamaki,Iwate,025-0082,Japan

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Higashi-Matsuyama,Saitama,355-0005,Japan

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Koshigaya,Saitama,343-8555,Japan

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Shinjuku Ku,Tokyo,160-0023,Japan

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Chiba,2608677,Japan

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Hiroshima,730-8518,Japan

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Sendai-shi,Miyagi,983 8520,Japan

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Busan,49241,Korea, Republic of

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Warsaw,01-684,Poland

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Warsaw,02 106,Poland

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Katowice,40-689,Poland

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Bydgoszcz,Woj Kujawsko Pomorskie,85-796,Poland

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Brasov,500283,Romania

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Bucharest,040215,Romania

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Sibiu,550245,Romania

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Busan,49241,South Korea

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Seoul,03080,South Korea

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Seoul,134 727,South Korea

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Seoul,03722,South Korea

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L'Hospitalet de Llobregat,Barcelona,08907,Spain

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Lleida,25198,Spain

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Madrid,28034,Spain

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Málaga,29009,Spain

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Taoyuan District,33305,Taiwan

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Taipei,103616,Taiwan

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Kaohsiung City,83301,Taiwan

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Tainan City,70403,Taiwan

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Taipei,10002,Taiwan

Fullerton Neuro and Headache Ctr

Recruiting

Fullerton,California,92835,United States

Jack H Florin
Alexandra Vasquez

Center for Neurological Disorders G

Recruiting

Greenfield,Wisconsin,53228-1321,United States

Tayo Olapo
Bhupendra Khatri

Univ of Cincinnati Medical Center

Recruiting

Cincinnati,Ohio,45219,United States

Sharon Briley
Hani Kushlaf

Univ Cali Irvine ALS Neuromuscular

Recruiting

Orange,California,92868,United States

Karina Bjazevic
Karina Bjazevic
Ali Habib

Neurological Services of Orlando PA

Recruiting

Orlando,Florida,32806,United States

Tanya Palafox
Tanya Palafox
Daniel H Jacobs

Homestead Assoc In Research Inc

Recruiting

Homestead,Florida,33033,United States

Osmany Morales Leon
Osmany Morales Leon
Christopher Jimenez
Ana Moncada

SFM Clinical Research LLC

Recruiting

Boca Raton,Florida,33487,United States

Melany Zafra
Marc Feinberg
Alyssa Gosling

Houston Methodist Hospital

Recruiting

Houston,Texas,77030,United States

Ericka Greene
Kadeesia Brown
Kadeesia Brown

Honor Health Research Institute

Recruiting

Scottsdale,Arizona,85258,United States

Claudia Crooks
Claudia Crooks
Anne Hatch

Neuromuscular Research Center

Recruiting

Phoenix,Arizona,85013,United States

Lucia Rodriguez
Kumaraswamy Sivakumar

Dent Neurological Institute

Recruiting

Buffalo,New York,14209,United States

Jacara Washington
Heather Macnamara
Bennett Myers

Mid Atlantic Epilepsy and Sleep Ctr

Recruiting

Bethesda,Maryland,20817,United States

Jonathan Ross
Salman Hashmi
Salman Hashmi

Michigan State University-Department of Neurology

Recruiting

East Lansing,Michigan,48824,United States

Amit Sachdev

University Of Southern California

Recruiting

Los Angeles,California,90033,United States

Salma Akhter
Said R Beydoun

Advent Health Cancer Institute

Recruiting

Orlando,Florida,32804,United States

Yvonne Burrell
Carrie Bednarz
Annalyn Dallas
Anita Fletcher

University of WA Division of Cardio

Recruiting

Seattle,Washington,98195,United States

Kaycie Opiyo
Kaycie Opiyo
Barbara Jane Distad

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Novartis Pharmaceuticals

Novartis Pharmaceuticals