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

NCT06744920 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: CLOU064O123012023-510026-32
• Study Type: interventional
• Target: 180
• Locations: 19 countries
• Sites: 100

Brief 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.

Conditions

Generalized Myasthenia Gravis

Keywords

Myasthenia Gravis; gMG; Generalized Myasthenia Gravis; LOU064; Remibrutinib; placebo-controlled; double-blind; phase III

Outcome Measures

Primary Outcomes (1)

• Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score

  The MG-ADL is a categorical scale that assesses MG symptoms and their effects on daily activities. MG-ADL is composed of items related to patient's assessment of functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. Each item is assessed on a 4-points scale where a score 0 represents normal function and a score 3 represents loss of ability to perform that function (total score 0 to 24).

  Baseline to Month 6

Secondary Outcomes (14)

• Change from baseline to Month 6 in Quantitative Myasthenia Gravis (QMG) total score

  Baseline to Month 6

• 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

  Baseline to Month 6

• Proportion of participants with ≥ 3 points reduction from baseline to Month 6 of Myasthenia Gravis Activity of Daily Living (MG-ADL) scale total score without rescue medication and/or strongly confounding prohibited medication

  Baseline to Month 6

• Proportion of participants achieving Minimal Symptom Expression (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

  Month 6

• Change from baseline to Month 6 in Myasthenia Gravis Composite score (MGC) total score

  Baseline to Month 6

Study Arms (2)

Remibrutinib arm

EXPERIMENTAL

Core Part: Remibrutinib tablet taken orally \[Extension Part: Open-label remibrutinib tablet taken orally\]

Drug: Remibrutinib (Blinded); Drug: Remibrutinib (Open Label)

Placebo arm

PLACEBO COMPARATOR

Core Part: Placebo tablet taken orally \[Extension Part: Open-label remibrutinib tablet taken orally\]

Other: Placebo; Drug: Remibrutinib (Open Label)

Interventions

Remibrutinib (Blinded) DRUG

Remibrutinib (Blinded) active treatment

Also known as: LOU64

Remibrutinib arm

Placebo OTHER

Placebo

Placebo arm

Remibrutinib (Open Label) DRUG

Remibrutinib (Open Label) active treatment

Also known as: LOU064

Placebo arm; Remibrutinib arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (100)

Neuromuscular Research Center

Phoenix, Arizona, 85013, United States

RECRUITING

Honor Health Research Institute

Scottsdale, Arizona, 85258, United States

RECRUITING

Fullerton Neuro and Headache Ctr

Fullerton, California, 92835, United States

RECRUITING

University Of Southern California

Los Angeles, California, 90033, United States

RECRUITING

Univ Cali Irvine ALS Neuromuscular

Orange, California, 92868, United States

RECRUITING

SFM Clinical Research LLC

Boca Raton, Florida, 33487, United States

RECRUITING

Homestead Assoc In Research Inc

Homestead, Florida, 33033, United States

RECRUITING

AdventHealth

Orlando, Florida, 32804, United States

RECRUITING

Neurological Services of Orlando PA

Orlando, Florida, 32806, United States

RECRUITING

Mid Atlantic Epilepsy and Sleep Ctr

Bethesda, Maryland, 20817-1807, United States

RECRUITING

Michigan State University-Department of Neurology

East Lansing, Michigan, 48824, United States

RECRUITING

Dent Neurological Institute

Buffalo, New York, 14209, United States

RECRUITING

Univ of Cincinnati Medical Center

Cincinnati, Ohio, 45219, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Houston Methodist Hospital

Houston, Texas, 77030, United States

RECRUITING

UT Health Science Center

Houston, Texas, 77030, United States

RECRUITING

University of WA Division of Cardio

Seattle, Washington, 98195, United States

RECRUITING

Center for Neurological Disorders G

Greenfield, Wisconsin, 53228-1321, United States

RECRUITING

Novartis Investigative Site

Rosario, Santa Fe Province, 2000, Argentina

RECRUITING

Novartis Investigative Site

Caba, C1055AAF, Argentina

RECRUITING

Novartis Investigative Site

Capital Federal, C1023AAB, Argentina

RECRUITING

Novartis Investigative Site

San Miguel de Tucumán, 4000, Argentina

RECRUITING

Novartis Investigative Site

Kogarah, New South Wales, 2217, Australia

RECRUITING

Novartis Investigative Site

Fitzroy, Victoria, 3065, Australia

RECRUITING

Novartis Investigative Site

Southport, 4215, Australia

RECRUITING

Novartis Investigative Site

Leuven, Vlaams Brabant, 3000, Belgium

RECRUITING

Novartis Investigative Site

Salvador, Estado de Bahia, 40050-410, Brazil

RECRUITING

Novartis Investigative Site

London, Ontario, N6A 5W9, Canada

RECRUITING

Novartis Investigative Site

Ottawa, Ontario, K1H 8L6, Canada

RECRUITING

Novartis Investigative Site

Hefei, Anhui, 230001, China

RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, 510030, China

RECRUITING

Novartis Investigative Site

Shijiazhuang, Hebei, 50030, China

RECRUITING

Novartis Investigative Site

Wuhan, Hubei, 430030, China

RECRUITING

Novartis Investigative Site

Wuxi, Jiangsu, 214023, China

RECRUITING

Novartis Investigative Site

Changchun, Jilin, 130021, China

RECRUITING

Novartis Investigative Site

Shanghai, 200040, China

RECRUITING

Novartis Investigative Site

Shanghai, 200080, China

RECRUITING

Novartis Investigative Site

Bordeaux, 33076, France

RECRUITING

Novartis Investigative Site

Nice, 06000, France

RECRUITING

Novartis Investigative Site

Paris, 75013, France

RECRUITING

Novartis Investigative Site

Strasbourg, 67081, France

RECRUITING

Novartis Investigative Site

Toulouse, 31059, France

RECRUITING

Novartis Investigative Site

Kutaisi, 4600, Georgia

RECRUITING

Novartis Investigative Site

Tbilisi, 0144, Georgia

RECRUITING

Novartis Investigative Site

Tbilisi, 114, Georgia

RECRUITING

Novartis Investigative Site

Hamburg, 20246, Germany

RECRUITING

Novartis Investigative Site

Gurugram, Haryana, 122011, India

RECRUITING

Novartis Investigative Site

Mangalore, Karnataka, 575002, India

RECRUITING

Novartis Investigative Site

Nashik, Maharashtra, 422005, India

RECRUITING

Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, 110029, India

RECRUITING

Novartis Investigative Site

Ludhiana, Punjab, 141001, India

RECRUITING

Novartis Investigative Site

Vellore, Tamil Nadu, 632 004, India

RECRUITING

Novartis Investigative Site

Hyderabad, Telangana, 500082, India

RECRUITING

Novartis Investigative Site

Lucknow, Uttar Pradesh, 226007, India

RECRUITING

Novartis Investigative Site

Bergamo, BG, 24127, Italy

RECRUITING

Novartis Investigative Site

Orbassano, TO, 10043, Italy

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608543, Japan

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0630005, Japan

RECRUITING

Novartis Investigative Site

Nishinomiya, Hyōgo, 6638501, Japan

RECRUITING

Novartis Investigative Site

Hanamaki, Iwate, 0250082, Japan

RECRUITING

Novartis Investigative Site

Sendai, Miyagi, 9838520, Japan

RECRUITING

Novartis Investigative Site

Suita, Osaka, 565-0871, Japan

RECRUITING

Novartis Investigative Site

Higashi-Matsuyama, Saitama, 355-0005, Japan

RECRUITING

Novartis Investigative Site

Koshigaya, Saitama, 343-8555, Japan

RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, 160-0023, Japan

RECRUITING

Novartis Investigative Site

Chiba, 2608677, Japan

RECRUITING

Novartis Investigative Site

Fukushima, 9601295, Japan

RECRUITING

Novartis Investigative Site

Hiroshima, 7348551, Japan

RECRUITING

Novartis Investigative Site

Lublin, Lublin Voivodeship, 20-064, Poland

RECRUITING

Novartis Investigative Site

Lublin, Lublin Voivodeship, 20-080, Poland

RECRUITING

Novartis Investigative Site

Krakow, POL, 31-505, Poland

RECRUITING

Novartis Investigative Site

Bydgoszcz, Woj Kujawsko Pomorskie, 85-796, Poland

RECRUITING

Novartis Investigative Site

Bydgoszcz, 85-065, Poland

RECRUITING

Novartis Investigative Site

Katowice, 40-689, Poland

RECRUITING

Novartis Investigative Site

Lodz, 93-113, Poland

RECRUITING

Novartis Investigative Site

Lublin, 20-410, Poland

RECRUITING

Novartis Investigative Site

Lublin, 20-701, Poland

RECRUITING

Novartis Investigative Site

Poznan, 61-731, Poland

RECRUITING

Novartis Investigative Site

Skorzewo, 60-185, Poland

RECRUITING

Novartis Investigative Site

Warsaw, 01-684, Poland

RECRUITING

Novartis Investigative Site

Warsaw, 02-172, Poland

RECRUITING

Novartis Investigative Site

Brasov, 500283, Romania

RECRUITING

Novartis Investigative Site

Bucharest, 040215, Romania

RECRUITING

Novartis Investigative Site

Sibiu, 550245, Romania

RECRUITING

Novartis Investigative Site

Belgrade, 11000, Serbia

RECRUITING

Novartis Investigative Site

Niš, 18108, Serbia

RECRUITING

Novartis Investigative Site

Busan, 49241, South Korea

RECRUITING

Novartis Investigative Site

Seoul, 03080, South Korea

RECRUITING

Novartis Investigative Site

Seoul, 03722, South Korea

RECRUITING

Novartis Investigative Site

Seoul, 134 727, South Korea

RECRUITING

Novartis Investigative Site

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

RECRUITING

Novartis Investigative Site

Lleida, 25198, Spain

RECRUITING

Novartis Investigative Site

Madrid, 28034, Spain

RECRUITING

Novartis Investigative Site

Málaga, 29010, Spain

RECRUITING

Novartis Investigative Site

Valencia, 46026, Spain

RECRUITING

Novartis Investigative Site

Kaohsiung City, 83301, Taiwan

RECRUITING

Novartis Investigative Site

Tainan, 704302, Taiwan

RECRUITING

Novartis Investigative Site

Taipei, 10002, Taiwan

RECRUITING

Novartis Investigative Site

Taipei, 111045, Taiwan

RECRUITING

Novartis Investigative Site

Taoyuan, 33305, Taiwan

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

remibrutinib

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2024
• First Posted: December 20, 2024
• Study Start: February 7, 2025
• Primary Completion (Estimated): February 25, 2028
• Study Completion (Estimated): February 26, 2033
• Last Updated: May 5, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

US (18); IN (8); RO (3); AR (4); IT (2); TW (5); CA (2); FR (5); JP (12); KR (4); CN (8); AU (3); ES (5); BE (1); PL (13); GE (3); SE (2); BR (1); DE (1)