A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis
Purpose
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.
Condition
- Generalized Myasthenia Gravis
Eligibility
- Eligible Ages
- Between 18 Years and 75 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
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
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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] |
|
Recruiting Locations
Honor Health Research Institute
Scottsdale, Arizona 85258
Scottsdale, Arizona 85258
Univ Cali Irvine ALS Neuromuscular
Orange, California 92868
Orange, California 92868
SFM Clinical Research LLC
Boca Raton, Florida 33487
Boca Raton, Florida 33487
Homestead Assoc In Research Inc
Homestead, Florida 33033
Homestead, Florida 33033
Advent Health Cancer Institute
Orlando, Florida 32804
Orlando, Florida 32804
Neurological Services of Orlando PA
Orlando, Florida 32806
Orlando, Florida 32806
Dent Neurological Institute
Buffalo, New York 14209
Buffalo, New York 14209
Houston Methodist Hospital
Houston, Texas 77030
Houston, Texas 77030
University of WA Division of Cardio
Seattle, Washington 98195
Seattle, Washington 98195
More Details
- NCT ID
- NCT06744920
- Status
- Recruiting
- Sponsor
- Novartis Pharmaceuticals
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).