A Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of rozanolixizumab in adult participants with ocular myasthenia gravis
Ocular myasthenia gravis is an autoimmune condition that weakens the muscles around the eyes, making everyday tasks like reading or driving difficult due to drooping eyelids and blurred or double vision. This study is testing whether a medicine called rozanolixizumab (brand name Rystiggo) is safe and effective at reducing these eye symptoms. Rozanolixizumab works by lowering the levels of certain antibodies in the blood that attack the nerve-to-muscle signals. Participants will be placed into one of two groups by chance: one group receives the study medicine as an injection under the skin, and the other receives a matching placebo (an inactive saline injection). Neither you nor the doctors will know which you receive. Doctors will check how your eye symptoms change after about six weeks of treatment, using both physical eye exams and quality-of-life questionnaires.
At a glance
What is this study about?
Myasthenia gravis is a long-term condition caused by the body's immune system mistakenly attacking the connections between nerves and muscles. When this weakness only affects the muscles that control the eyes and eyelids, it is known as ocular myasthenia gravis. Common symptoms include one or both eyelids drooping (ptosis) and double vision (diplopia). These symptoms can be frustrating, tiring, and interfere with simple daily activities such as reading, watching television, or driving.
Rozanolixizumab (Rystiggo) is a new targeted medicine designed to help. It works by blocking a specific protein in the body, which helps clear away the harmful antibodies that cause muscle weakness. While this medicine is already approved for generalized myasthenia gravis, researchers need to find out if it also brings clear benefits to people whose symptoms are mainly limited to their eyes.
In this Phase 3 trial, doctors are comparing rozanolixizumab directly against a placebo (an inactive dummy injection). The main goal is to measure how much participants' eye symptoms improve over the first 43 days of treatment. Researchers also want to monitor safety, check for any unwanted side effects, and see if the treatment helps improve people's day-to-day quality of life.
Key takeaways
- Phase 3 trial testing rozanolixizumab (Rystiggo) for ocular myasthenia gravis.
- Open to adults aged 18 and over who have eye muscle weakness like drooping eyelids or double vision.
- Half of participants will receive the active medicine and half will receive a dummy injection (placebo).
- Treatment is given as an injection under the skin.
- The main test of symptom improvement takes place after about six weeks (Day 43).
Who may be eligible?
This study is open to adults aged 18 and older who have been diagnosed with ocular myasthenia gravis and are experiencing active eye symptoms, such as drooping eyelids or double vision.
Because this trial focuses specifically on eye symptoms, it is aimed at individuals whose muscle weakness does not severely affect their limbs, breathing, or ability to swallow. Both men and women can join.
Before taking part, the study team will review your complete medical history and current treatments to ensure it is safe for you to participate. Certain health conditions, serious infections, or recent changes in your regular myasthenia gravis medicines may mean you cannot join.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have been diagnosed with ocular myasthenia gravis.
- I currently have eye symptoms such as droopy eyelids or double vision.
- My muscle weakness is mainly limited to my eyes rather than my whole body.
- I am able to attend regular hospital appointments for check-ups and injections.
What does participation involve?
If you take part, you will be randomly assigned to receive either rozanolixizumab or an inactive placebo injection. The treatment is given as an injection just under the skin. You will visit the clinic regularly so doctors can check your eye movements, examine eyelid drooping, and assess your general health. You will also be asked to fill in simple questionnaires about your vision, your daily activities, and how you are feeling. The main assessment happens around Day 43 (about six weeks in), and you will have follow-up visits to monitor your recovery and safety throughout the study.
Potential risks and benefits
Locations (7)
- —UnverifiedHungary
- —UnverifiedGreece
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedGermany
- —UnverifiedPoland
- —UnverifiedFrance
Common questions
What is a placebo and why is it used?
A placebo is an inactive substance, such as salt water, that looks identical to the real medicine. It helps researchers prove whether any health improvements are genuinely caused by the medicine rather than psychological factors or natural symptom changes.
Will I know if I am receiving the real medicine or the placebo?
No. This is a 'double-blind' trial, which means neither you nor the doctors will know which treatment you are receiving until the study is over. This ensures the results are completely fair.
How is the study medicine given?
The medicine is given as a subcutaneous injection, meaning it is injected into the fatty tissue just under your skin.
Will taking part cost me anything?
No, the study medication and all trial tests are free. Most clinical trials also offer help to cover your travel expenses to and from the hospital.
Can I leave the study early if I change my mind?
Yes. You have the right to withdraw from the trial at any point, for any reason, without losing any of your normal medical care or rights.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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