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Study on the Effects of Iptacopan in Patients with Generalized Myasthenia Gravis

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a disease called generalized Myasthenia Gravis (gMG), which is a condition that causes muscle weakness. The study will evaluate a treatment called iptacopan, which is taken as a capsule. The purpose of the study is to assess how effective and safe iptacopan is for people with gMG. Participants in the study will either receive iptacopan or a placebo, which looks like the treatment but does not contain the active medicine.

The study will begin with a phase where neither the participants nor the researchers know who is receiving iptacopan and who is receiving the placebo. This is known as a "double-blind" study. After this phase, there will be an "open label" phase where all participants will receive iptacopan. The study will last for several months, and participants will be monitored to see how their symptoms change over time. The main goal is to see if iptacopan can help reduce the symptoms of gMG, as measured by a specific scale that assesses daily living activities affected by the disease.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. The study will also look at any side effects that might occur with the use of iptacopan. This research aims to provide more information about the potential benefits and risks of using iptacopan for treating generalized Myasthenia Gravis.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    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.

  2. Step 2

    Randomization and treatment assignment

    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.

  3. Step 3

    Treatment phase

    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.

  4. Step 4

    Monitoring and assessments

    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.

  5. Step 5

    Evaluation of outcomes

    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.

  6. Step 6

    Open label extension phase

    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.

Who can join the trial?

12 criteria

  • Adults aged 18 to 75 years with generalized Myasthenia Gravis (gMG).
  • Positive test results for AChR+ antibody during screening. This means a specific blood test shows the presence of certain antibodies related to gMG.
  • Classified as Class II-IV gMG by the Myasthenia Gravis Foundation of America (MGFA), and not likely to need a breathing machine during the study, as determined by the study doctor.
  • Diagnosis of gMG must be confirmed by at least one of the following tests:
    • History of abnormal nerve-muscle communication shown by special tests like single-fiber electromyography or repetitive nerve stimulation.
    • History of a positive edrophonium chloride test, which is a test used to diagnose gMG.
    • Improvement in gMG symptoms when taking oral medications called acetylcholinesterase inhibitors, as noted by the doctor.
    • Baseline score of 6 or more on the Myasthenia Gravis Activity of Daily Living (MG-ADL) scale, with at least 50% of the score due to symptoms other than eye-related ones.
    • Participants whose symptoms are not well controlled for 6 months or more with:
      • Just one non-steroidal immunosuppressive therapy (NSIST).
      • Two or more NSISTs.
      • Frequent treatments like plasmapheresis, plasma exchange, or intravenous immunoglobulin, despite using steroids and NSISTs.
      • One of the following gMG treatments: an FcRN antagonist approved for gMG, rituximab, or other approved gMG therapies, excluding complement inhibitors.
      • Participants must be vaccinated against certain bacteria: Neisseria meningitidis and Streptococcus pneumoniae. Depending on local rules and vaccine availability, they may also need a vaccine against Haemophilus influenzae. Vaccination should be done at least 2 weeks before starting the study medication. If the study medication starts earlier, antibiotics must be taken until 2 weeks after vaccination.

Who cannot join the trial?

5 criteria

  • Patients who have a different condition than generalized Myasthenia Gravis cannot participate. This is a condition that affects the muscles and causes weakness.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not on a stable treatment plan for their condition cannot participate. A stable treatment plan means that their current treatment has not changed recently.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
  • Patients who do not meet other specific health criteria set by the study cannot participate.
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Investigated drugs

Iptacopan is a medication being studied for its effectiveness in treating patients with generalized Myasthenia Gravis (gMG). This condition affects the muscles, causing weakness and fatigue. The trial aims to see if iptacopan can help reduce the symptoms of gMG, making daily activities easier for patients. The study will also look at the safety and how well patients tolerate the medication over a period of six months.

What is already known about the treatment

Iptacopan – Iptacopan is administered orally and is currently being studied in a phase III clinical trial for its effectiveness in treating generalized Myasthenia Gravis (gMG). This medication is not yet widely available in medical practice, as it is still under investigation. It is primarily indicated for patients with gMG, a chronic autoimmune neuromuscular disorder. Iptacopan works by inhibiting a specific protein in the immune system, which helps reduce the symptoms of muscle weakness associated with gMG. It belongs to the pharmacological class of complement inhibitors, which are designed to modulate the immune response.

Investigated diseases

Generalized Myasthenia Gravis – Generalized Myasthenia Gravis is a chronic autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the body's own tissues, specifically targeting the receptors that receive nerve signals. This results in fluctuating muscle weakness and fatigue, which can affect various muscle groups, including those controlling eye movements, facial expressions, and swallowing. The severity of symptoms can vary, often worsening with activity and improving with rest. Over time, the condition may progress, affecting more muscle groups and leading to increased difficulty in performing daily activities.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-507064-39-00Protocol codeCLNP023Q12301Estimated enrolment146 patientsSponsorNovartis Pharma AG

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