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

July 28, 2026 updated by: Novartis Pharmaceuticals

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / 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).

Study Type

Interventional

Enrollment (Estimated)

180

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Novartis Pharmaceuticals
  • Phone Number: +41613241111

Study Locations

  • Argentina
      • Caba, Argentina, C1055AAF
        • Recruiting
        • Novartis Investigative Site
      • Capital Federal, Argentina, C1023AAB
        • Recruiting
        • Novartis Investigative Site
      • San Miguel de Tucumán, Argentina, T4000AXL
        • Recruiting
        • Novartis Investigative Site
    • Santa Fe Province
      • Rosario, Santa Fe Province, Argentina, 2000
        • Recruiting
        • Novartis Investigative Site
  • Australia
      • Southport, Australia, 4215
        • Recruiting
        • Novartis Investigative Site
    • New South Wales
      • Kogarah, New South Wales, Australia, 2217
        • Recruiting
        • Novartis Investigative Site
    • Victoria
      • Fitzroy, Victoria, Australia, 3065
        • Recruiting
        • Novartis Investigative Site
  • Belgium
    • Vlaams Brabant
      • Leuven, Vlaams Brabant, Belgium, 3000
        • Recruiting
        • Novartis Investigative Site
  • Brazil
    • Estado de Bahia
      • Salvador, Estado de Bahia, Brazil, 40050-410
        • Recruiting
        • Novartis Investigative Site
    • Federal District
      • Brasília, Federal District, Brazil, 70200-730
        • Recruiting
        • Novartis Investigative Site
    • Rio Grande do Sul
      • Caxias do Sul, Rio Grande do Sul, Brazil, 95070-560
        • Recruiting
        • Novartis Investigative Site
      • Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
        • Recruiting
        • Novartis Investigative Site
      • Porto Alegre, Rio Grande do Sul, Brazil, 90480-110
        • Recruiting
        • Novartis Investigative Site
    • São Paulo
      • São Paulo, São Paulo, Brazil, 05403-000
        • Recruiting
        • Novartis Investigative Site
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • Recruiting
        • Novartis Investigative Site
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • Recruiting
        • Novartis Investigative Site
      • Ottawa, Ontario, Canada, K1H 8L6
        • Recruiting
        • Novartis Investigative Site
    • Quebec
      • Québec, Quebec, Canada, G1J 1Z4
        • Recruiting
        • Novartis Investigative Site
  • China
      • Beijing, China, 100730
        • Recruiting
        • Novartis Investigative Site
      • Beijing, China, 100034
        • Recruiting
        • Novartis Investigative Site
      • Qingdao, China, 266000
        • Recruiting
        • Novartis Investigative Site
      • Shanghai, China, 200080
        • Recruiting
        • Novartis Investigative Site
      • Shanghai, China, 200040
        • Recruiting
        • Novartis Investigative Site
    • Anhui
      • Hefei, Anhui, China, 230001
        • Recruiting
        • Novartis Investigative Site
    • Guangdong
      • Guangzhou, Guangdong, China, 510080
        • Recruiting
        • Novartis Investigative Site
    • Hebei
      • Shijiazhuang, Hebei, China, 50030
        • Recruiting
        • Novartis Investigative Site
    • Henan
      • Zhengzhou, Henan, China, 450003
        • Recruiting
        • Novartis Investigative Site
    • Hubei
      • Wuhan, Hubei, China, 430030
        • Recruiting
        • Novartis Investigative Site
    • Jiangsu
      • Wuxi, Jiangsu, China, 214023
        • Recruiting
        • Novartis Investigative Site
    • Jilin
      • Changchun, Jilin, China, 130021
        • Recruiting
        • Novartis Investigative Site
  • France
      • Bordeaux, France, 33076
        • Recruiting
        • Novartis Investigative Site
      • Nice, France, 06000
        • Recruiting
        • Novartis Investigative Site
      • Paris, France, 75013
        • Recruiting
        • Novartis Investigative Site
      • Paris, France, 75940
        • Recruiting
        • Novartis Investigative Site
      • Strasbourg, France, 67081
        • Recruiting
        • Novartis Investigative Site
      • Toulouse, France, 31059
        • Recruiting
        • Novartis Investigative Site
  • Georgia
      • Kutaisi, Georgia, 4600
        • Recruiting
        • Novartis Investigative Site
      • Tbilisi, Georgia, 0144
        • Recruiting
        • Novartis Investigative Site
      • Tbilisi, Georgia, 0114
        • Recruiting
        • Novartis Investigative Site
  • Germany
      • Gummersbach, Germany, 51643
        • Recruiting
        • Novartis Investigative Site
      • Hamburg, Germany, 20246
        • Recruiting
        • Novartis Investigative Site
      • Münster, Germany, 48149
        • Recruiting
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main, Hesse, Germany, 60590
        • Recruiting
        • Novartis Investigative Site
  • India
    • Haryana
      • Gurugram, Haryana, India, 122011
        • Recruiting
        • Novartis Investigative Site
    • Karnataka
      • Mangalore, Karnataka, India, 575002
        • Recruiting
        • Novartis Investigative Site
    • Maharashtra
      • Nashik, Maharashtra, India, 422005
        • Recruiting
        • Novartis Investigative Site
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, India, 110029
        • Recruiting
        • Novartis Investigative Site
    • Punjab
      • Ludhiana, Punjab, India, 141001
        • Recruiting
        • Novartis Investigative Site
    • Tamil Nadu
      • Vellore, Tamil Nadu, India, 632 004
        • Recruiting
        • Novartis Investigative Site
    • Telangana
      • Hyderabad, Telangana, India, 500082
        • Recruiting
        • Novartis Investigative Site
    • Uttar Pradesh
      • Lucknow, Uttar Pradesh, India, 226007
        • Recruiting
        • Novartis Investigative Site
  • Italy
      • Naples, Italy, 80131
        • Recruiting
        • Novartis Investigative Site
    • BG
      • Bergamo, BG, Italy, 24127
        • Recruiting
        • Novartis Investigative Site
    • TO
      • Orbassano, TO, Italy, 10043
        • Recruiting
        • Novartis Investigative Site
  • Japan
      • Fukuoka, Japan, 8128582
        • Recruiting
        • Novartis Investigative Site
      • Fukushima, Japan, 9601295
        • Recruiting
        • Novartis Investigative Site
      • Hiroshima, Japan, 7348551
        • Recruiting
        • Novartis Investigative Site
    • Chiba
      • Chiba, Chiba, Japan, 2608677
        • Recruiting
        • Novartis Investigative Site
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 0608543
        • Recruiting
        • Novartis Investigative Site
      • Sapporo, Hokkaido, Japan, 0630005
        • Recruiting
        • Novartis Investigative Site
    • Hyōgo
      • Nishinomiya, Hyōgo, Japan, 6638501
        • Recruiting
        • Novartis Investigative Site
    • Iwate
      • Hanamaki, Iwate, Japan, 0250082
        • Recruiting
        • Novartis Investigative Site
    • Miyagi
      • Sendai, Miyagi, Japan, 9838520
        • Recruiting
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 5650871
        • Recruiting
        • Novartis Investigative Site
    • Saitama
      • Higashi-Matsuyama, Saitama, Japan, 355-0005
        • Recruiting
        • Novartis Investigative Site
      • Koshigaya, Saitama, Japan, 343-8555
        • Recruiting
        • Novartis Investigative Site
    • Tokyo
      • Shinjuku Ku, Tokyo, Japan, 160-0023
        • Recruiting
        • Novartis Investigative Site
  • Poland
      • Bydgoszcz, Poland, 85-065
        • Recruiting
        • Novartis Investigative Site
      • Katowice, Poland, 40-689
        • Recruiting
        • Novartis Investigative Site
      • Lodz, Poland, 93-113
        • Recruiting
        • Novartis Investigative Site
      • Lublin, Poland, 20-410
        • Recruiting
        • Novartis Investigative Site
      • Lublin, Poland, 20-701
        • Recruiting
        • Novartis Investigative Site
      • Poznan, Poland, 61-731
        • Recruiting
        • Novartis Investigative Site
      • Rzeszów, Poland, 35-323
        • Recruiting
        • Novartis Investigative Site
      • Skorzewo, Poland, 60-185
        • Recruiting
        • Novartis Investigative Site
      • Szczecin, Poland, 71-252
        • Recruiting
        • Novartis Investigative Site
      • Warsaw, Poland, 01-684
        • Recruiting
        • Novartis Investigative Site
      • Warsaw, Poland, 02-172
        • Recruiting
        • Novartis Investigative Site
    • Lublin Voivodeship
      • Lublin, Lublin Voivodeship, Poland, 20-064
        • Recruiting
        • Novartis Investigative Site
      • Lublin, Lublin Voivodeship, Poland, 20-080
        • Recruiting
        • Novartis Investigative Site
    • POL
      • Krakow, POL, Poland, 31-505
        • Recruiting
        • Novartis Investigative Site
    • Woj Kujawsko Pomorskie
      • Bydgoszcz, Woj Kujawsko Pomorskie, Poland, 85-796
        • Recruiting
        • Novartis Investigative Site
  • Romania
      • Brasov, Romania, 500283
        • Recruiting
        • Novartis Investigative Site
      • Bucharest, Romania, 040215
        • Recruiting
        • Novartis Investigative Site
      • Sibiu, Romania, 550245
        • Recruiting
        • Novartis Investigative Site
  • Serbia
      • Belgrade, Serbia, 11000
        • Recruiting
        • Novartis Investigative Site
      • Niš, Serbia, 18108
        • Recruiting
        • Novartis Investigative Site
      • Novi Sad, Serbia, 21000
        • Recruiting
        • Novartis Investigative Site
  • South Korea
      • Busan, South Korea, 49241
        • Recruiting
        • Novartis Investigative Site
      • Seoul, South Korea, 03722
        • Recruiting
        • Novartis Investigative Site
      • Seoul, South Korea, 134 727
        • Recruiting
        • Novartis Investigative Site
    • Seoul
      • Seoul, Seoul, South Korea, 03080
        • Recruiting
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain, 08035
        • Recruiting
        • Novartis Investigative Site
      • Lleida, Spain, 25198
        • Recruiting
        • Novartis Investigative Site
      • Madrid, Spain, 28034
        • Recruiting
        • Novartis Investigative Site
      • Málaga, Spain, 29010
        • Recruiting
        • Novartis Investigative Site
      • Valencia, Spain, 46026
        • Recruiting
        • Novartis Investigative Site
    • A Coruna
      • Santiago Compostela, A Coruna, Spain, 15706
        • Recruiting
        • Novartis Investigative Site
    • Barcelona
      • L'Hospitalet de Llobregat, Barcelona, Spain, 08907
        • Recruiting
        • Novartis Investigative Site
  • Taiwan
      • Kaohsiung City, Taiwan, 833
        • Recruiting
        • Novartis Investigative Site
      • Tainan, Taiwan, 704302
        • Recruiting
        • Novartis Investigative Site
      • Taipei, Taiwan, 10002
        • Recruiting
        • Novartis Investigative Site
      • Taipei, Taiwan, 111045
        • Recruiting
        • Novartis Investigative Site
      • Taoyuan, Taiwan, 33305
        • Recruiting
        • Novartis Investigative Site
  • United Kingdom
      • London, United Kingdom, SW17 0QT
        • Recruiting
        • Novartis Investigative Site
      • London, United Kingdom, NW1 2BU
        • Withdrawn
        • Novartis Investigative Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Recruiting
        • Neuromuscular Research Center
        • Principal Investigator:
          • Kumaraswamy Sivakumar
        • Contact:
      • Scottsdale, Arizona, United States, 85258
        • Recruiting
        • Honor Health Research Institute
        • Principal Investigator:
          • Anne Hatch
        • Contact:
    • California
      • Fullerton, California, United States, 92835
        • Recruiting
        • Fullerton Neuro and Headache Ctr
        • Principal Investigator:
          • Jack H Florin
        • Contact:
      • Los Angeles, California, United States, 90033
        • Recruiting
        • University of Southern California
        • Principal Investigator:
          • Said R Beydoun
        • Contact:
      • Orange, California, United States, 92868
        • Recruiting
        • Univ Cali Irvine ALS Neuromuscular
        • Principal Investigator:
          • Ali Habib
        • Contact:
      • Sacramento, California, United States, 94115
        • Recruiting
        • California Pacific Medical Center
        • Principal Investigator:
          • Liberty Jenkins
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010-2975
        • Recruiting
        • Medstar Washington Hospital Center
        • Principal Investigator:
          • Nicholas Streicher
        • Contact:
    • Florida
      • Boca Raton, Florida, United States, 33487
        • Recruiting
        • SFM Clinical Research LLC
        • Principal Investigator:
          • Marc Feinberg
        • Contact:
      • Homestead, Florida, United States, 33033
        • Recruiting
        • Homestead Assoc In Research Inc
        • Principal Investigator:
          • Christopher Jimenez
        • Contact:
      • Maitland, Florida, United States, 32751
        • Recruiting
        • Neurology Associates PA
        • Principal Investigator:
          • Arnaldo Isa
        • Contact:
      • Orlando, Florida, United States, 32804
      • Orlando, Florida, United States, 32806
        • Recruiting
        • Neurological Services of Orlando PA
        • Principal Investigator:
          • Daniel H Jacobs
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60637
    • Maryland
      • Bethesda, Maryland, United States, 20817-1807
        • Recruiting
        • Mid Atlantic Epilepsy and Sleep Ctr
        • Contact:
        • Principal Investigator:
          • Jonathan Ross
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
        • Principal Investigator:
          • Amanda Guidon
        • Contact:
    • Michigan
      • East Lansing, Michigan, United States, 48824
        • Recruiting
        • Michigan State University-Department of Neurology
        • Contact:
        • Principal Investigator:
          • Amit Sachdev
    • New York
      • Buffalo, New York, United States, 14209
        • Recruiting
        • Dent Neurological Institute
        • Principal Investigator:
          • Bennett Myers
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Recruiting
        • Univ of Cincinnati Medical Center
        • Principal Investigator:
          • Hani Kushlaf
        • Contact:
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic Foundation
        • Contact:
        • Principal Investigator:
          • Yuebing Li
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • University of Pittsburgh Medical Center
        • Principal Investigator:
          • Fang Sun
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Houston Methodist Hospital
        • Principal Investigator:
          • Ericka Greene
        • Contact:
      • Houston, Texas, United States, 77030
    • Washington
      • Seattle, Washington, United States, 98195
        • Recruiting
        • University of WA Division of Cardio
        • Principal Investigator:
          • Barbara Jane Distad
        • Contact:
    • Wisconsin
      • Greenfield, Wisconsin, United States, 53228-1321
        • Recruiting
        • Center for Neurological Disorders G
        • Principal Investigator:
          • Bhupendra Khatri
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Remibrutinib arm

Core Part: Remibrutinib tablet taken orally

[Extension Part: Open-label remibrutinib tablet taken orally]
Drug: Remibrutinib (Blinded)
Remibrutinib (Blinded) active treatment
Other Names:
  • LOU64
Drug: Remibrutinib (Open Label)
Remibrutinib (Open Label) active treatment
Other Names:
  • LOU064
Placebo Comparator: Placebo arm

Core Part: Placebo tablet taken orally

[Extension Part: Open-label remibrutinib tablet taken orally]
Other: Placebo
Placebo
Drug: Remibrutinib (Open Label)
Remibrutinib (Open Label) active treatment
Other Names:
  • LOU064

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
Time Frame: Baseline to Month 6

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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline to Month 6 in Quantitative Myasthenia Gravis (QMG) total score
Time Frame: Baseline to Month 6
The Quantitative Myasthenia Gravis (QMG) Score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicated greater disease severity. The QMG score is composed of the following items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item) and respiratory (1 item).
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
Time Frame: Baseline to Month 6
The Quantitative Myasthenia Gravis (QMG) Score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicated greater disease severity. The QMG score is composed of the following items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item) and respiratory (1 item).
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
Time Frame: Baseline to Month 6

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
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
Time Frame: Month 6

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).
Month 6
Change from baseline to Month 6 in Myasthenia Gravis Composite score (MGC) total score
Time Frame: Baseline to Month 6
The MGC is a 10-item instrument that measures the symptoms and signs of MG based on physician examination and patient history. Items relate to ptosis, double vision, eye closure, talking, chewing, swallowing, breathing, neck flexion, shoulder abduction, and hip flexion. Each item is scored on an ordinal scale with 4 possible categories and weighted. The total score ranges from 0 to 50, where higher scores indicating more severe impairments.
Baseline to Month 6
Change from baseline to Month 6 in revised Myasthenia Gravis Quality of Life Questionnaire (MG-QOL15r) survey score
Time Frame: Baseline to Month 6
The revised MG-QoL15 is a 15-item health related quality of life questionnaire completed by participants, designed to measure quality of life in gMG. Items on the MG-QoL15 relate to physical, social, and psychological components and are scored from 0 (not at all) to 2 (very much). The cumulative scores range from 0 to 30, with higher scores representing worse quality of life and dissatisfaction with MG-related dysfunction.
Baseline to Month 6
Incidence of adverse events
Time Frame: Baseline to Month 6
Incidence of adverse events including changes in clinical laboratory values, vital signs, electrocardiograms and suicidality results qualifying and reported as AEs.
Baseline to Month 6
Proportion of time during which participants showed a reduction of ≥ 2 points in MG-ADL total score, that was maintained up to Month 6
Time Frame: Baseline to Month 6
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
Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of ≥ 2 points occurring by week 4）
Time Frame: Baseline to week 4
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). Participants with a first MG-ADL improvement from baseline of ≥ 2 points occurring by week 4 are considered early responders.
Baseline to week 4
Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L)
Time Frame: Baseline to Month 6
The EQ-5D-5L is a widely used questionnaire designed to assess health status in adults. The measure is divided into two distinct sections, the descriptive system and the EQ visual analogue scale (EQ VAS). The first section includes one item addressing each of five dimensions (mobility, self-care, usual activity, pain/discomfort, and anxiety/depression). Participants rate each of these items from 1 of the 5 levels: no problems, slight problems, moderate problems, severe problems, or unable to/extreme. A composite health state is then defined by combining the levels for each dimension into a 5-digit number. The second section includes the EQ visual analogue scale (EQ VAS) that measures self-rated (global) health status utilizing a vertically oriented visual analogue scale where 100 represents the "best imaginable health state" and 0 represents the "worst imaginable health state." Respondents are asked to rate their current health by placing a mark along this continuum.
Baseline to Month 6
Proportion of participants achieving a reduction from baseline of ≥ 3 points in MGC total score at Month 6
Time Frame: Baseline to Month 6
The MGC is a 10-item instrument that measures the symptoms and signs of MG based on physician examination and patient history. Items relate to ptosis, double vision, eye closure, talking, chewing, swallowing, breathing, neck flexion, shoulder abduction, and hip flexion. Each item is scored on an ordinal scale with 4 possible categories and weighted. The total score ranges from 0 to 50, where higher scores indicating more severe impairments.
Baseline to Month 6
Change from baseline in MG-ADL total score
Time Frame: Baseline to Month 66
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 66
Proportion of participants achieving a reduction from core part in oral corticosteroids (OCS) dose till the end of extension part
Time Frame: Baseline to month 66
Participants receiving oral corticosteroids are required to be on a stable dose for at least 4 weeks prior to baseline. The dose can be reduced or discontinued during the open label extension period at investigator discretion. Oral corticosteroids use will be recorded.
Baseline to month 66
Incidence of adverse events
Time Frame: Month 7 to month 66
Incidence of adverse events including changes in clinical laboratory values, vital signs, electrocardiograms and suicidality results qualifying and reported as AEs.
Month 7 to month 66
Change from baseline in QMG total score
Time Frame: Baseline to Month 66
The Quantitative Myasthenia Gravis (QMG) Score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicated greater disease severity. The QMG score is composed of the following items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item) and respiratory (1 item).
Baseline to Month 66
Change from baseline in MGC total score
Time Frame: Baseline to Month 66
The MGC is a 10-item instrument that measures the symptoms and signs of MG based on physician examination and patient history. Items relate to ptosis, double vision, eye closure, talking, chewing, swallowing, breathing, neck flexion, shoulder abduction, and hip flexion. Each item is scored on an ordinal scale with 4 possible categories and weighted. The total score ranges from 0 to 50, where higher scores indicating more severe impairments.
Baseline to Month 66

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 7, 2025

Primary Completion (Estimated)

February 25, 2028

Study Completion (Estimated)

February 26, 2033

Study Registration Dates

First Submitted

December 17, 2024

First Submitted That Met QC Criteria

December 17, 2024

First Posted (Actual)

December 20, 2024

Study Record Updates

Last Update Posted (Actual)

July 29, 2026

Last Update Submitted That Met QC Criteria

July 28, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLOU064O12301
  • 2023-510026-32 (Other Identifier: EU CTIS)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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