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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
5/06/2025

Titles & IDs
Public title
A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis
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
Secondary ID [1] 0 0
CLOU064O12301
Universal Trial Number (UTN)
Trial acronym
RELIEVE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Generalized Myasthenia Gravis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Other neurological disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
Timepoint [1] 0 0
Baseline to Month 6
Secondary outcome [1] 0 0
Change from baseline to Month 6 in Quantitative Myasthenia Gravis (QMG) total score
Timepoint [1] 0 0
Baseline to Month 6
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
Timepoint [2] 0 0
Baseline to Month 6
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
Timepoint [3] 0 0
Baseline to Month 6
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
Timepoint [4] 0 0
Month 6
Secondary outcome [5] 0 0
Change from baseline to Month 6 in Myasthenia Gravis Composite score (MGC) total score
Timepoint [5] 0 0
Baseline to Month 6
Secondary outcome [6] 0 0
Change from baseline to Month 6 in revised Myasthenia Gravis Quality of Life Questionnaire (MG-QOL15r) survey score
Timepoint [6] 0 0
Baseline to Month 6
Secondary outcome [7] 0 0
Incidence of adverse events
Timepoint [7] 0 0
Baseline to Month 6
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
Timepoint [8] 0 0
Baseline to Month 6
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)
Timepoint [9] 0 0
Baseline to week 4
Secondary outcome [10] 0 0
Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L)
Timepoint [10] 0 0
Baseline to Month 6
Secondary outcome [11] 0 0
Proportion of participants achieving a reduction from baseline of = 3 points in MGC total score at Month 6
Timepoint [11] 0 0
Baseline to Month 6
Secondary outcome [12] 0 0
Change from baseline in MG-ADL total score
Timepoint [12] 0 0
Baseline to Month 66
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
Timepoint [13] 0 0
Baseline to month 66
Secondary outcome [14] 0 0
Incidence of adverse events
Timepoint [14] 0 0
Month 7 to month 66

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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/02/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
26/02/2033
Actual
Sample size
Target
180
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Novartis Investigative Site - Kogarah
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
United States of America
State/province [6] 0 0
Washington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
[email protected]
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