A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase
Novartis Pharmaceuticals
Summary
The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC treatment. The randomization will be stratified based on region.
Description
The study consists of a 6-month double-blind treatment period for the primary efficacy and safety analysis followed by a maximum duration of 60 month open label extension period. A safety follow up assessment will be performed, one 7 days after the last administration of study treatment and one 30 days after the last administration of study treatment for all participants.
Eligibility
- Age range
- 18–85 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Adult patients with generalized Myasthenia Gravis (age 18-85 years) at screening * Positive serology testing for AChR+ antibody at screening * 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. * The confirmation of the diagnosis of gMG should be documented and supported by ≥1 of the following 3 tests: * History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation. * History of positi…
Interventions
- DrugIptacopan
Hard gelatin capsule
- OtherMatching Placebo
Hard gelatin capsule
Locations (112)
- Honor Health Research InstituteScottsdale, Arizona
- Fullerton Neuro and Headache CtrFullerton, California
- SC3 Research PasadenaPasadena, California
- California Pacific Medical CenterSacramento, California
- Neurology Offices Of South FloridaBoca Raton, Florida
- Superior Associates in Research LLCHialeah, Florida