Ravulizumab for Myasthenia Gravis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores how ravulizumab affects children with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. Participants will receive ravulizumab through an IV to observe their body's response over time. Children diagnosed with gMG and anti-AChR antibodies, who are on a stable treatment plan, might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial requires that participants be on a stable dosing regimen of their current medications before and during the screening period, so you will not need to stop taking them.
Is there any evidence suggesting that ravulizumab is likely to be safe for humans?
Research has shown that ravulizumab is usually well-tolerated by people with generalized myasthenia gravis (gMG). In earlier studies, about 23% of patients experienced serious side effects, indicating that while some had significant reactions, many did not.
Other studies indicate that ravulizumab is safe and effective over the long term. Patients have reported noticeable improvements without many severe problems. These findings suggest that although risks exist, ravulizumab could be a safe option for many people with gMG. Discussing any concerns with a healthcare provider is always important.12345Why do researchers think this study treatment might be promising for myasthenia gravis?
Ravulizumab is unique because it offers a new approach for treating myasthenia gravis by targeting the complement system, which plays a role in the immune response. Unlike current treatments like pyridostigmine, corticosteroids, and other immunosuppressants that generally aim to manage symptoms or suppress the immune system broadly, ravulizumab specifically inhibits a part of the immune system involved in the disease process. This specificity may lead to fewer side effects and potentially more effective management of symptoms. Additionally, its infrequent dosing schedule—once every 4 to 8 weeks—offers a more convenient option compared to some existing treatments that require daily administration.
What evidence suggests that ravulizumab might be an effective treatment for myasthenia gravis?
Research has shown that ravulizumab helps treat generalized myasthenia gravis (gMG). Studies have found it makes daily activities easier for people with this condition. Participants who received ravulizumab had better control over their gMG symptoms than those who did not. The CHAMPION MG trial confirmed that ravulizumab was both effective and safe compared to a placebo. Overall, these findings suggest that ravulizumab could be a promising option for managing gMG symptoms.15678
Are You a Good Fit for This Trial?
This trial is for children with generalized Myasthenia Gravis (gMG) who are classified as Class II to IV. They must be on a stable treatment regimen, vaccinated against meningococcal infection, and if previously treated with eculizumab, they should have been on it for at least 6 months. Those with HIV, recent thymic surgery or untreated thymic malignancy cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a weight-based loading dose of ravulizumab IV on Day 1, followed by maintenance doses every 8 weeks or 4 weeks based on weight, for a total of 122 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Ravulizumab
Ravulizumab is already approved in United States, European Union for the following indications:
- Paroxysmal nocturnal hemoglobinuria (PNH)
- Atypical hemolytic uremic syndrome (aHUS)
- Paroxysmal nocturnal haemoglobinuria (PNH)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion
Lead Sponsor
Marc Dunoyer
Alexion
Chief Executive Officer since 2021
PhD in Molecular Biology, University of Brussels
Christophe Hotermans
Alexion
Chief Medical Officer since 2021
MD, University of Leuven
Alexion Pharmaceuticals
Lead Sponsor
Marc Dunoyer
Alexion Pharmaceuticals
Chief Executive Officer since 2021
B.A. in Psychology from the University of New Hampshire
Christophe Hotermans
Alexion Pharmaceuticals
Chief Medical Officer since 2023
MD
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology