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
Neuromuscular Research Center
Phoenix 5308655, Arizona 5551752 85013
Phoenix 5308655, Arizona 5551752 85013
Honor Health Research Institute
Scottsdale 5313457, Arizona 5551752 85258
Scottsdale 5313457, Arizona 5551752 85258
Fullerton Neuro and Headache Ctr
Fullerton 5351247, California 5332921 92835
Fullerton 5351247, California 5332921 92835
University Of Southern California
Los Angeles 5368361, California 5332921 90033
Los Angeles 5368361, California 5332921 90033
Univ Cali Irvine ALS Neuromuscular
Orange 5379513, California 5332921 92868
Orange 5379513, California 5332921 92868
SFM Clinical Research LLC
Boca Raton 4148411, Florida 4155751 33487
Boca Raton 4148411, Florida 4155751 33487
Homestead Assoc In Research Inc
Homestead 4159050, Florida 4155751 33033
Homestead 4159050, Florida 4155751 33033
AdventHealth
Orlando 4167147, Florida 4155751 32804
Orlando 4167147, Florida 4155751 32804
Neurological Services of Orlando PA
Orlando 4167147, Florida 4155751 32806
Orlando 4167147, Florida 4155751 32806
Mid Atlantic Epilepsy and Sleep Ctr
Bethesda 4348599, Maryland 4361885 20817-1807
Bethesda 4348599, Maryland 4361885 20817-1807
Michigan State University-Department of Neurology
East Lansing 4991640, Michigan 5001836 48824
East Lansing 4991640, Michigan 5001836 48824
Dent Neurological Institute
Buffalo 5110629, New York 5128638 14209
Buffalo 5110629, New York 5128638 14209
Univ of Cincinnati Medical Center
Cincinnati 4508722, Ohio 5165418 45219
Cincinnati 4508722, Ohio 5165418 45219
University of Pittsburgh Medical Center
Pittsburgh 5206379, Pennsylvania 6254927 15213
Pittsburgh 5206379, Pennsylvania 6254927 15213
Houston Methodist Hospital
Houston 4699066, Texas 4736286 77030
Houston 4699066, Texas 4736286 77030
UT Health Science Center
Houston 4699066, Texas 4736286 77030
Houston 4699066, Texas 4736286 77030
University of WA Division of Cardio
Seattle 5809844, Washington 5815135 98195
Seattle 5809844, Washington 5815135 98195
Center for Neurological Disorders G
Greenfield 5255068, Wisconsin 5279468 53228-1321
Greenfield 5255068, Wisconsin 5279468 53228-1321
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).