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
Novartis Pharmaceuticals
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.
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).
Eligibility
- Age range
- 18–75 years
- Sex
- All
- 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 spec…
Interventions
- DrugRemibrutinib (Blinded)
Remibrutinib (Blinded) active treatment
- OtherPlacebo
Placebo
- DrugRemibrutinib (Open Label)
Remibrutinib (Open Label) active treatment
Locations (100)
- Neuromuscular Research CenterPhoenix, Arizona
- Honor Health Research InstituteScottsdale, Arizona
- Fullerton Neuro and Headache CtrFullerton, California
- University Of Southern CaliforniaLos Angeles, California
- Univ Cali Irvine ALS NeuromuscularOrange, California
- SFM Clinical Research LLCBoca Raton, Florida