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NCT06744920PHASE3Recruiting

A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis

Novartis Pharmaceuticals

Start Date

2/7/2025

Completion Date

2/26/2033

Summary

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.

Detailed Description

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).

Eligibility Criteria

Age Range: 18 years to 85 years

Inclusion Criteria: * Adult patients with gMG (age 18-85 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 receiving a 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, from menarche until becoming post-menopausal, 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.

Interventions

DRUG

Remibrutinib (Blinded)

OTHER

Placebo

DRUG

Remibrutinib (Open Label)

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Conditions

Generalized Myasthenia Gravis

Locations

Neuromuscular Research Center

Phoenix, Arizona 85013

United States

Honor Health Research Institute

Scottsdale, Arizona 85258

United States

Fullerton Neuro and Headache Ctr

Fullerton, California 92835

United States

University Of Southern California

Los Angeles, California 90033

United States

Univ Cali Irvine ALS Neuromuscular

Orange, California 92868

United States

California Pacific Medical Center

Sacramento, California 94115

United States

Medstar Washington Hospital Center

Washington D.C., District of Columbia 20010-2975

United States

SFM Clinical Research LLC

Boca Raton, Florida 33487

United States

Homestead Assoc In Research Inc

Homestead, Florida 33033

United States

Neurology Associates PA

Maitland, Florida 32751

United States

AdventHealth

Orlando, Florida 32804

United States

Neurological Services of Orlando PA

Orlando, Florida 32806

United States

University of Chicago Medical Centr

Chicago, Illinois 60637

United States

Mid Atlantic Epilepsy and Sleep Ctr

Bethesda, Maryland 20817-1807

United States

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

Michigan State University-Department of Neurology

East Lansing, Michigan 48824

United States

Dent Neurological Institute

Buffalo, New York 14209

United States

Univ of Cincinnati Medical Center

Cincinnati, Ohio 45219

United States

Cleveland Clinic Foundation

Cleveland, Ohio 44195

United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania 15213

United States

Houston Methodist Hospital

Houston, Texas 77030

United States

UT Health Science Center

Houston, Texas 77030

United States

University of WA Division of Cardio

Seattle, Washington 98195

United States

Center for Neurological Disorders G

Greenfield, Wisconsin 53228-1321

United States

Novartis Investigative Site

Rosario, Santa Fe Province 2000

Argentina

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Caba, C1055AAF

Argentina

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Capital Federal, C1023AAB

Argentina

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San Miguel de Tucumán, T4000AXL

Argentina

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Kogarah, New South Wales 2217

Australia

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Fitzroy, Victoria 3065

Australia

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Southport, 4215

Australia

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Leuven, Vlaams Brabant 3000

Belgium

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Salvador, Estado de Bahia 40050-410

Brazil

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Brasília, Federal District 70200-730

Brazil

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Caxias do Sul, Rio Grande do Sul 95070-560

Brazil

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Porto Alegre, Rio Grande do Sul 90035-903

Brazil

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Porto Alegre, Rio Grande do Sul 90480-110

Brazil

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São Paulo, São Paulo 05403-000

Brazil

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Edmonton, Alberta T6G 2B7

Canada

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London, Ontario N6A 5W9

Canada

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Ottawa, Ontario K1H 8L6

Canada

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Québec, Quebec G1J 1Z4

Canada

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Hefei, Anhui 230001

China

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Guangzhou, Guangdong 510080

China

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Shijiazhuang, Hebei 50030

China

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Zhengzhou, Henan 450003

China

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Wuhan, Hubei 430030

China

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Wuxi, Jiangsu 214023

China

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Changchun, Jilin 130021

China

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Beijing, 100034

China

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Beijing, 100730

China

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Qingdao, 266000

China

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Shanghai, 200040

China

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Shanghai, 200080

China

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Bordeaux, 33076

France

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Nice, 06000

France

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Paris, 75013

France

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Paris, 75940

France

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Strasbourg, 67081

France

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Toulouse, 31059

France

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Kutaisi, 4600

Georgia

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Tbilisi, 0114

Georgia

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Tbilisi, 0144

Georgia

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Frankfurt am Main, Hesse 60590

Germany

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Gummersbach, 51643

Germany

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Hamburg, 20246

Germany

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Münster, 48149

Germany

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Gurugram, Haryana 122011

India

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Mangalore, Karnataka 575002

India

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Nashik, Maharashtra 422005

India

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New Delhi, National Capital Territory of Delhi 110029

India

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Ludhiana, Punjab 141001

India

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Vellore, Tamil Nadu 632 004

India

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Hyderabad, Telangana 500082

India

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Lucknow, Uttar Pradesh 226007

India

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Bergamo, BG 24127

Italy

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Orbassano, TO 10043

Italy

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Naples, 80131

Italy

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Chiba, Chiba 2608677

Japan

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Sapporo, Hokkaido 0608543

Japan

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Sapporo, Hokkaido 0630005

Japan

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Nishinomiya, Hyōgo 6638501

Japan

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Hanamaki, Iwate 0250082

Japan

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Sendai, Miyagi 9838520

Japan

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Suita, Osaka 5650871

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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Fukuoka, 8128582

Japan

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Fukushima, 9601295

Japan

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Hiroshima, 7348551

Japan

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Lublin, Lublin Voivodeship 20-064

Poland

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Lublin, Lublin Voivodeship 20-080

Poland

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Krakow, POL 31-505

Poland

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Bydgoszcz, Woj Kujawsko Pomorskie 85-796

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Bydgoszcz, 85-065

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Katowice, 40-689

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Lodz, 93-113

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Lublin, 20-410

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Lublin, 20-701

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Poznan, 61-731

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Rzeszów, 35-323

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Skorzewo, 60-185

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Szczecin, 71-252

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Warsaw, 01-684

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Warsaw, 02-172

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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Belgrade, 11000

Serbia

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Niš, 18108

Serbia

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Novi Sad, 21000

Serbia

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Seoul, Seoul 03080

South Korea

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Busan, 49241

South Korea

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Seoul, 03722

South Korea

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Seoul, 134 727

South Korea

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Santiago Compostela, A Coruna 15706

Spain

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L'Hospitalet de Llobregat, Barcelona 08907

Spain

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Barcelona, 08035

Spain

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Lleida, 25198

Spain

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Madrid, 28034

Spain

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Málaga, 29010

Spain

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Valencia, 46026

Spain

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Kaohsiung City, 833

Taiwan

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Tainan, 704302

Taiwan

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Taipei, 10002

Taiwan

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Taipei, 111045

Taiwan

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Taoyuan, 33305

Taiwan

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London, NW1 2BU

United Kingdom

Novartis Investigative Site

London, SW17 0QT

United Kingdom