Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06744920
Registration number
NCT06744920
Ethics application status
Date submitted
17/12/2024
Date registered
20/12/2024
Date last updated
16/07/2025
Titles & IDs
Public title
A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis
Query!
Scientific 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
Query!
Secondary ID [1]
0
0
CLOU064O12301
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
RELIEVE
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Generalized Myasthenia Gravis
0
0
Query!
Condition category
Condition code
Inflammatory and Immune System
0
0
0
0
Query!
Autoimmune diseases
Query!
Neurological
0
0
0
0
Query!
Other neurological disorders
Query!
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Remibrutinib (Blinded)
Other interventions - Placebo
Treatment: Drugs - Remibrutinib (Open Label)
Experimental: Remibrutinib arm - Core Part: Remibrutinib tablet taken orally
\[Extension Part: Open-label remibrutinib tablet taken orally\]
Placebo comparator: Placebo arm - Core Part: Placebo tablet taken orally
\[Extension Part: Open-label remibrutinib tablet taken orally\]
Treatment: Drugs: Remibrutinib (Blinded)
Remibrutinib (Blinded) active treatment
Other interventions: Placebo
Placebo
Treatment: Drugs: Remibrutinib (Open Label)
Remibrutinib (Open Label) active treatment
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Intervention code [2]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
Query!
Assessment method [1]
0
0
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).
Query!
Timepoint [1]
0
0
Baseline to Month 6
Query!
Secondary outcome [1]
0
0
Change from baseline to Month 6 in Quantitative Myasthenia Gravis (QMG) total score
Query!
Assessment method [1]
0
0
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).
Query!
Timepoint [1]
0
0
Baseline to Month 6
Query!
Secondary outcome [2]
0
0
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
Query!
Assessment method [2]
0
0
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).
Query!
Timepoint [2]
0
0
Baseline to Month 6
Query!
Secondary outcome [3]
0
0
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
Query!
Assessment method [3]
0
0
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).
Query!
Timepoint [3]
0
0
Baseline to Month 6
Query!
Secondary outcome [4]
0
0
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
Query!
Assessment method [4]
0
0
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).
Query!
Timepoint [4]
0
0
Month 6
Query!
Secondary outcome [5]
0
0
Change from baseline to Month 6 in Myasthenia Gravis Composite score (MGC) total score
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline to Month 6
Query!
Secondary outcome [6]
0
0
Change from baseline to Month 6 in revised Myasthenia Gravis Quality of Life Questionnaire (MG-QOL15r) survey score
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
Baseline to Month 6
Query!
Secondary outcome [7]
0
0
Incidence of adverse events
Query!
Assessment method [7]
0
0
Incidence of adverse events including changes in clinical laboratory values, vital signs, electrocardiograms and suicidality results qualifying and reported as AEs.
Query!
Timepoint [7]
0
0
Baseline to Month 6
Query!
Secondary outcome [8]
0
0
Proportion of time during which participants showed a reduction of = 2 points in MG-ADL total score, that was maintained up to Month 6
Query!
Assessment method [8]
0
0
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).
Query!
Timepoint [8]
0
0
Baseline to Month 6
Query!
Secondary outcome [9]
0
0
Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of = 2 points occurring by week 4)
Query!
Assessment method [9]
0
0
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.
Query!
Timepoint [9]
0
0
Baseline to week 4
Query!
Secondary outcome [10]
0
0
Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L)
Query!
Assessment method [10]
0
0
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.
Query!
Timepoint [10]
0
0
Baseline to Month 6
Query!
Secondary outcome [11]
0
0
Proportion of participants achieving a reduction from baseline of = 3 points in MGC total score at Month 6
Query!
Assessment method [11]
0
0
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.
Query!
Timepoint [11]
0
0
Baseline to Month 6
Query!
Secondary outcome [12]
0
0
Change from baseline in MG-ADL total score
Query!
Assessment method [12]
0
0
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).
Query!
Timepoint [12]
0
0
Baseline to Month 66
Query!
Secondary outcome [13]
0
0
Proportion of participants achieving a reduction from core part in oral corticosteroids (OCS) dose till the end of extension part
Query!
Assessment method [13]
0
0
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.
Query!
Timepoint [13]
0
0
Baseline to month 66
Query!
Secondary outcome [14]
0
0
Incidence of adverse events
Query!
Assessment method [14]
0
0
Incidence of adverse events including changes in clinical laboratory values, vital signs, electrocardiograms and suicidality results qualifying and reported as AEs.
Query!
Timepoint [14]
0
0
Month 7 to month 66
Query!
Eligibility
Key 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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
75
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
7/02/2025
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
26/02/2033
Query!
Actual
Query!
Sample size
Target
180
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Query!
Recruitment hospital [1]
0
0
Novartis Investigative Site - Kogarah
Query!
Recruitment hospital [2]
0
0
Novartis Investigative Site - Fitzroy
Query!
Recruitment postcode(s) [1]
0
0
2217 - Kogarah
Query!
Recruitment postcode(s) [2]
0
0
3065 - Fitzroy
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arizona
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Florida
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
New York
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Texas
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Washington
Query!
Country [7]
0
0
Japan
Query!
State/province [7]
0
0
Miyagi
Query!
Country [8]
0
0
Korea, Republic of
Query!
State/province [8]
0
0
Busan
Query!
Country [9]
0
0
Poland
Query!
State/province [9]
0
0
Katowice
Query!
Country [10]
0
0
Poland
Query!
State/province [10]
0
0
Warszawa
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Novartis Pharmaceuticals
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT06744920
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Novartis Pharmaceuticals
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
1-888-669-6682
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06744920
Download to PDF