A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis
This research study is investigating a new medication, Remibrutinib, for adults who have Generalized Myasthenia Gravis. The main aim is to understand if Remibrutinib is effective at helping with the symptoms of this condition, how safe it is, and whether people can tolerate it well. Participants will either receive Remibrutinib or a placebo (a dummy medicine that looks the same but has no active ingredient), in addition to their current, stable treatments. This will be done in a way that neither the patient nor their doctor knows who is getting which treatment at first. The study hopes to gather important information that could help improve future treatment options for Myasthenia Gravis.
At a glance
What is this study about?
This study is about an illness called Generalized Myasthenia Gravis, which is a long-term condition that causes muscle weakness. It's looking into a new medicine called Remibrutinib. The main goal is to find out if this medicine can help improve symptoms, if it's safe to use, and if people find it easy to take, especially when they're already on their usual treatments.
To do this, some adult patients with Generalized Myasthenia Gravis will receive the new medicine, Remibrutinib, while others will get a placebo. A placebo looks just like the real medicine but contains no active ingredient. This is a common way to test new medicines, as it helps researchers see if any improvements are truly due to the new drug or something else. Neither the patients nor their doctors will know who is receiving which treatment during the first part of the study; this is called 'double-blind'.
Later, everyone will have the chance to take Remibrutinib. The study aims to involve around 180 people and split them into two groups. By carefully comparing the groups, experts hope to learn a lot about Remibrutinib and whether it could become a helpful new treatment option for people living with Generalized Myasthenia Gravis.
Key takeaways
- This study is testing a new medicine (Remibrutinib) for Generalized Myasthenia Gravis.
- It aims to see how well it works and if it's safe.
- You would continue your existing Myasthenia Gravis treatments.
- Participants will either receive Remibrutinib or a dummy drug (placebo) at first.
- The study could last for several years, with regular check-ups.
- You can stop participating at any time.
Who may be eligible?
This study is looking for adults aged between 18 and 75 who have been diagnosed with Generalized Myasthenia Gravis. Your doctor will need to confirm you have a certain level of Myasthenia Gravis symptoms and are unlikely to need breathing support during the study.
You'll also need to have had specific antibody tests that confirm your Myasthenia Gravis, or show you don't have these antibodies. Your symptoms should include a certain level of daily activity difficulties, with at least half of these difficulties not related to your eyes. You must also be on a steady dose of your current Myasthenia Gravis treatments and be able to swallow the study medication safely.
There are also some reasons why you might not be able to join. For example, if you've recently had certain treatments like IV immunoglobulins, plasma exchange, or specific therapies such as rituximab, eculizumab, or efgartigimod within certain timeframes before the study. Also, if you've had surgery to remove your thymus gland in the last six months or plan to have one, you wouldn't be able to join. Women who could become pregnant must be using effective contraception throughout the study and for a week afterwards.
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.
- Are you an adult between 18 and 75 years old?
- Do you have a confirmed diagnosis of Generalized Myasthenia Gravis?
- Are your Myasthenia Gravis symptoms mostly affecting parts of your body other than your eyes?
- Are you currently on a stable dose of your usual Myasthenia Gravis treatments?
- Can you safely swallow medications, as checked by your doctor?
- Have you avoided certain other Myasthenia Gravis treatments (like IVIg/PLEX, rituximab, eculizumab, etc.) within specific recent timeframes?
What does participation involve?
If you join this study, it will be split into two main parts. For the first six months, you will either receive Remibrutinib or a placebo, and neither you nor your doctors will know which one. This period involves regular visits to the clinic for assessments and to check how you're feeling and if the treatment is helping.
After these first six months, if you wish to continue, you could then enter an 'open-label' part of the study. This means everyone will receive Remibrutinib, and everyone will know they are receiving the active drug. This longer part could last for up to five years. Throughout the study, you'll have appointments to review your health, check for any side effects, and monitor your Myasthenia Gravis symptoms. The study medication will need to be swallowed, and your ability to do so will be assessed.
Potential risks and benefits
Locations (129)
- Neuromuscular Research CenterVerified postcodePhoenix, United States· Recruiting
- Honor Health Research InstituteVerified postcodeScottsdale, United States· Recruiting
- Fullerton Neuro and Headache CtrVerified postcodeFullerton, United States· Recruiting
- University Of Southern CaliforniaVerified postcodeLos Angeles, United States· Recruiting
- Univ Cali Irvine ALS NeuromuscularVerified postcodeOrange, United States· Recruiting
- California Pacific Medical CenterVerified postcodeSacramento, United States· Recruiting
- Medstar Washington Hospital CenterVerified postcodeWashington D.C., United States· Recruiting
- SFM Clinical Research LLCVerified postcodeBoca Raton, United States· Recruiting
- Homestead Assoc In Research IncVerified postcodeHomestead, United States· Recruiting
- Neurology Associates PAVerified postcodeMaitland, United States· Recruiting
- AdventHealthVerified postcodeOrlando, United States· Recruiting
- Neurological Services of Orlando PAVerified postcodeOrlando, United States· Recruiting
Common questions
What is Generalized Myasthenia Gravis?
It's a long-term condition that causes muscle weakness throughout the body.
What is Remibrutinib?
It's a new medicine being tested to see if it can help people with Myasthenia Gravis.
What is a 'placebo'?
A placebo is a dummy medicine that looks like the real drug but contains no active ingredients. It's used for comparison.
Will I know if I'm getting the real medicine or the placebo?
For the first six months, neither you nor your doctor will know. After that, everyone will get the active medicine.
How long will the study last?
The main part is six months, with an option to continue for up to five years.
How to find out more
Novartis Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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