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Recruiting NCT06744920

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

Phase III Interventional Generalized Myasthenia Gravis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Remibrutinib (Blinded), Placebo, Remibrutinib (Open Label).
Who it may be relevant to
Registry conditions: Generalized Myasthenia Gravis. Basic parameters: 18 years — 85 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Argentina, Australia, Belgium, Brazil +15
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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).

Interventions

  • Drug Remibrutinib (Blinded)
    Remibrutinib (Blinded) active treatment
  • Other Placebo
    Placebo
  • Drug Remibrutinib (Open Label)
    Remibrutinib (Open Label) active treatment

Primary outcome measures

  • Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score [Time frame: Baseline to Month 6]
Secondary outcome measures (12)
  • Change from baseline to Month 6 in Quantitative Myasthenia Gravis (QMG) total score [Time frame: 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]
  • 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]
  • 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]
  • Change from baseline to Month 6 in Myasthenia Gravis Composite score (MGC) total score [Time frame: 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]
  • Incidence of adverse events [Time frame: 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]
  • 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]
  • Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L) [Time frame: 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]
  • Change from baseline in MG-ADL total score [Time frame: Baseline to Month 66]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Double blind
Primary purpose
Treatment

Study locations

United States · 24 centers
  • Neuromuscular Research Center — Phoenix
  • Honor Health Research Institute — Scottsdale
  • Fullerton Neuro and Headache Ctr — Fullerton
  • University Of Southern California — Los Angeles
  • Univ Cali Irvine ALS Neuromuscular — Orange
  • California Pacific Medical Center — Sacramento
  • Medstar Washington Hospital Center — Washington D.C.
  • SFM Clinical Research LLC — Boca Raton
  • … and 16 more centers
Poland · 15 centers

Center list to be confirmed — check the primary protocol.

Japan · 13 centers
  • Novartis Investigative Site — Chiba
  • Novartis Investigative Site — Sapporo
  • … and 11 more centers
China · 12 centers
  • Novartis Investigative Site — Hefei
  • Novartis Investigative Site — Guangzhou
  • Novartis Investigative Site — Shijiazhuang
  • Novartis Investigative Site — Zhengzhou
  • Novartis Investigative Site — Wuhan
  • Novartis Investigative Site — Wuxi
  • Novartis Investigative Site — Changchun
  • Novartis Investigative Site — Beijing
  • … and 4 more centers
India · 8 centers
  • Novartis Investigative Site — Gurugram
  • Novartis Investigative Site — Mangalore
  • Novartis Investigative Site — Nashik
  • Novartis Investigative Site — New Delhi
  • Novartis Investigative Site — Ludhiana
  • Novartis Investigative Site — Vellore
  • Novartis Investigative Site — Hyderabad
  • Novartis Investigative Site — Lucknow
Spain · 7 centers

Center list to be confirmed — check the primary protocol.

Brazil · 6 centers
  • Novartis Investigative Site — Salvador
  • Novartis Investigative Site — Brasília
  • Novartis Investigative Site — Caxias do Sul
  • Novartis Investigative Site — Porto Alegre
  • Novartis Investigative Site — Porto Alegre
  • Novartis Investigative Site — São Paulo
France · 6 centers
  • Novartis Investigative Site — Bordeaux
  • Novartis Investigative Site — Nice
  • Novartis Investigative Site — Paris
  • Novartis Investigative Site — Paris
  • Novartis Investigative Site — Strasbourg
  • Novartis Investigative Site — Toulouse
Taiwan · 5 centers

Center list to be confirmed — check the primary protocol.

Argentina · 4 centers
  • Novartis Investigative Site — Rosario
  • Novartis Investigative Site — Caba
  • Novartis Investigative Site — Capital Federal
  • Novartis Investigative Site — San Miguel de Tucumán
Canada · 4 centers
  • Novartis Investigative Site — Edmonton
  • Novartis Investigative Site — London
  • Novartis Investigative Site — Ottawa
  • Novartis Investigative Site — Québec
Germany · 4 centers
  • Novartis Investigative Site — Frankfurt am Main
  • Novartis Investigative Site — Gummersbach
  • Novartis Investigative Site — Hamburg
  • Novartis Investigative Site — Münster
South Korea · 4 centers

Center list to be confirmed — check the primary protocol.

Australia · 3 centers
  • Novartis Investigative Site — Kogarah
  • Novartis Investigative Site — Fitzroy
  • Novartis Investigative Site — Southport
Georgia · 3 centers
  • Novartis Investigative Site — Kutaisi
  • Novartis Investigative Site — Tbilisi
  • Novartis Investigative Site — Tbilisi
Italy · 3 centers
  • Novartis Investigative Site — Bergamo
  • Novartis Investigative Site — Orbassano
  • Novartis Investigative Site — Naples
Romania · 3 centers

Center list to be confirmed — check the primary protocol.

Serbia · 3 centers

Center list to be confirmed — check the primary protocol.

United Kingdom · 2 centers

Center list to be confirmed — check the primary protocol.

Belgium · 1 center
  • Novartis Investigative Site — Leuven

Identifiers

NCT: NCT06744920 · CLOU064O12301 · 2023-510026-32

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗