Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying the diagnosis of generalized Myasthenia Gravis (gMG) and ensuring the patient meets specific criteria, such as age and medical history.

Vaccinations against certain infections are required at least two weeks before starting the medication, unless prophylactic antibiotics are used.

Participants are randomly assigned to receive either the study medication, iptacopan, or a placebo. This process is double-blind, meaning neither the participants nor the researchers know who receives which treatment.

The medication is administered orally in the form of hard gelatin capsules.

The treatment phase lasts for six months. During this time, the effectiveness of iptacopan is evaluated by measuring changes in the Myasthenia Gravis Activity of Daily Living (MG-ADL) score.

Participants continue their stable standard of care (SOC) treatments alongside the study medication.

Regular monitoring is conducted to assess the patient’s health and response to the treatment. This includes clinical laboratory tests, vital signs, and electrocardiograms.

The incidence of any adverse events is recorded, and adjustments to the treatment plan are made if necessary.

At the end of the six-month treatment phase, the primary outcome is the change in the MG-ADL score from baseline.

Secondary outcomes include changes in other health scores and the incidence of adverse events.

Following the initial treatment phase, participants may enter an open label extension phase where all receive iptacopan.

This phase allows for further evaluation of the medication’s long-term effects and safety.