Actively Recruiting
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
Led by Novartis Pharmaceuticals · Updated on 2026-05-27
146
Participants Needed
124
Research Sites
264 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy, safety, and tolerability of iptacopan in adults aged 18 to 85 years with generalized Myasthenia Gravis who are positive for AChR antibodies and are on stable standard-of-care treatment. This randomized, double-blind, placebo-controlled, multicenter Phase III study aims to compare iptacopan with placebo over six months, followed by a long-term open-label extension to further assess treatment effects and safety. Participants will be randomly assigned in equal groups to receive either oral iptacopan or a matching placebo daily for six months while continuing their standard care. After this double-blind phase, all participants are offered open-label iptacopan treatment for up to 60 months. Safety follow-up assessments occur 7 and 30 days after the last study treatment dose. During the study, participants will attend regular visits to assess changes in their Myasthenia Gravis symptoms using several scales, including the MG-ADL score, QMG, MGC, and quality of life questionnaires. Researchers will monitor adverse events and corticosteroid use throughout both the core and extension phases. The total study participation can last up to about five years, including the initial treatment, extension, and follow-up periods.
CONDITIONS
Brief Title
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 85 years with generalized Myasthenia Gravis at screening
- Positive antibody test for AChR+ at screening
- MGFA Class II-IV generalized Myasthenia Gravis and unlikely to need a respirator during the study
- Diagnosis confirmed by at least one test: abnormal neuromuscular transmission, positive acetylcholinesterase inhibitor test, or symptom improvement on oral acetylcholinesterase inhibitors
- Baseline MG-ADL score of 6 or higher with at least 50% from non-ocular symptoms
- On stable treatment for at least 6 months with one or more NSISTs, plasmapheresis, intravenous immunoglobulin, approved FcRN antagonist, rituximab, or other approved disease-modifying therapies excluding complement inhibitors
- Vaccinated against Neisseria meningitidis and Streptococcus pneumoniae before starting study treatment, or receiving appropriate prophylactic antibiotics if needed
You will not qualify if you...
- Treatment with intravenous immunoglobulin or plasma exchange in past month
- Treatment with rituximab in past 6 months
- Treatment with eculizumab in past 2 months or ravulizumab/complement inhibitors in past 3 months
- Treatment with efgartigimod or other anti-FcRn therapies in past 3 months
- Thymectomy within past 6 months or planned during the trial
- Active or uncontrolled infections including Hepatitis B, Hepatitis C, or HIV with AIDS-defining conditions or low CD4 count
- Pregnant or lactating women, or those planning pregnancy
- Women of childbearing potential not using effective contraception during and one week after treatment
- Active systemic infections or major infections requiring hospitalization or injectable therapy within 14 days prior to treatment
- History of recurrent invasive infections from encapsulated organisms
- Fever of 38°C (100.4°F) or higher within 7 days before treatment
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
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