NCT06517758 · Novartis Pharmaceuticals
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.
What this study is about
The study is a randomly assigned, where neither patients nor doctors know which treatment is given, compared against an inactive treatment, conducted at multiple hospitals, Phase III study, to evaluate effectiveness, safety and how well patients handle the treatment of iptacopan in patients with AChR+ gMG who are on stable SOC treatment.
View original scientific description
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.
Interventions
DRUG
Iptacopan
Hard gelatin capsule
OTHER
Matching Placebo
Hard gelatin capsule
Primary outcome measures
Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
Time frame: Baseline to Month 6
The MG-ADL is an 8 item interviewer led patient reporting scale that assesses MG symptoms and their effects on daily activities. MG-ADL is composed of items related to patient's assessment of functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. Each item is assessed on a 4-points scale where a score 0 represents normal function and a score 3 represents loss of ability to perform that function. The scores ranges from 0 to 24, with a higher score indicating more disability.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Adult patients with generalized Myasthenia Gravis (age 18-75 years)
- Positive serology testing for AChR+ antibody at screening
- Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG and likely not in need v 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 positive edrophonium chloride test
- Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.
- Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms
- Participants not optimally controlled for ≥ 6 months on
- just one NSIST; or
- two or more NSISTs; or
- on frequent (at least quarterly) plasmapheresis, plasma exchange, or intrav
Where
- Scottsdale, Arizona
- Fullerton, California
- Pasadena, California
- Sacramento, California
- Boca Raton, Florida
- Hialeah, Florida
- Augusta, Georgia
- Honolulu, Hawaii
- Chicago, Illinois
- Springfield, Illinois
- Bethesda, Maryland
- Boston, Massachusetts
And 9 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 23, 2026 · Source of record for eligibility and locations