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 (CLOU064O12301)
- Conditions
- generalized Myasthenia Gravis
- Registration Number
- jRCT2021250004
- Lead Sponsor
- Novartis Pharma. K.K.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 14
-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
-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
Study & Design
- Study Type
- Interventional
- Study Design
- parallel assignment
- Primary Outcome Measures
Name Time Method - Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
- Secondary Outcome Measures
Name Time Method