Alexion C5 Inhibition Therapies for Myasthenia Gravis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial gathers long-term data on the safety and effectiveness of two treatments, eculizumab and ravulizumab (also known as Ultomiris), for people with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. By observing patients already using these treatments, researchers aim to understand how well these therapies work in real-world settings. People with gMG who are currently on or have previously used these treatments and can provide past health information related to their condition may be a good fit for this trial. As a Phase 4 trial, this research focuses on understanding how these FDA-approved treatments benefit more patients in everyday settings.
Do I need to stop my current medications for this trial?
The trial does not specify if you need to stop your current medications. However, since it involves patients already treated with specific therapies, you might be able to continue your current treatment.
What is the safety track record for these treatments?
Research has shown that both eculizumab and ravulizumab are generally safe for people with generalized myasthenia gravis (gMG). Eculizumab has low acute toxicity, making serious harm from a single dose unlikely. Clinical trials mostly support its safety, although real-world data remains limited.
For ravulizumab, studies indicate that diarrhea and upper respiratory infections are the most common side effects, affecting at least 10% of patients. Most side effects are mild to moderate. However, ravulizumab can weaken the immune system, increasing the risk of serious infections, particularly from bacteria like Neisseria meningitidis, which can lead to meningitis, a serious infection of the brain and spinal cord coverings.
Both medications have received approval for use, indicating thorough safety testing. However, like all medicines, they carry some risks. Discuss any concerns with a healthcare provider.12345Why are researchers excited about this trial?
Researchers are excited about Eculizumab and Ravulizumab for generalized myasthenia gravis because they target the complement system, specifically inhibiting the C5 protein. This is different from many traditional treatments that primarily focus on modulating the immune response or managing symptoms. By specifically blocking the C5 protein, these treatments can prevent the breakdown of the neuromuscular junction, which is a key problem in myasthenia gravis. Additionally, Ravulizumab has a longer dosing interval compared to Eculizumab, which could mean fewer hospital visits and more convenience for patients.
What is the effectiveness track record for Eculizumab and Ravulizumab in treating gMG?
Research has shown that eculizumab can greatly help people with generalized myasthenia gravis (gMG). Clinical trials found that patients using eculizumab experienced better muscle strength and improved quality of life. Those treated had fewer symptoms, making daily life easier. Similarly, studies have shown that ravulizumab effectively keeps gMG symptoms under control. Patients reported lasting improvements, indicating that both treatments can effectively manage gMG symptoms over time.678910
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Enrollment and Retrospective Data Collection
Participant records are queried for retrospective information about medical history and gMG disease treatment history
Prospective Data Collection
Prospective data collection using routine clinical care data and patient-reported outcome methods
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Eculizumab
- Ravulizumab
Find a Clinic Near You
Who Is Running the Clinical Trial?
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
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
Citations
Data from the International PNH Registry
We demonstrate that treatment with eculizumab conferred a 49% relative benefit in survival and an approximately 60% reduction in TE/MAVE risk.
Real-World Eculizumab Effectiveness on US Patient ...
Results from this study suggest that treatment with eculizumab reduced patients' treatment burden (shown by the decrease in concomitant prednisone use) and ...
Long‐term effectiveness of eculizumab: Data from the ...
Long-term effectiveness data of eculizumab on patient survival and prevention of clinical outcomes such as TEs have been previously ...
First analysis of the Myasthenia Gravis SPOTLIGHT Registry
Clinical trials have demonstrated improved functional ability, muscle strength, and quality of life (QOL) in patients treated with eculizumab or ...
The effectiveness and value of eculizumab ...
At week 26, the proportion of patients with minimal symptom expression (MG-ADL score of 0 or 1) was much greater in the eculizumab group (21.4% vs 1.7%, P = ...
Safety Data Sheet
Wear appropriate protective eyeglasses or chemical safety goggles as described by OSHA. Body Protection. Wear compatible chemical-resistant ...
7.
file.medchemexpress.com
file.medchemexpress.com/batch_PDF/HY-P9914/Eculizumab-SDS-MedChemExpress.pdfSafety Data Sheet
Eculizumab. Catalog No. : HY-P9914. CAS No. : 219685-50-4. 1.2 Relevant identified uses of the substance or mixture and uses advised against.
8.
abmole.com
abmole.com/literature/eculizumab-msds.html?srsltid=AfmBOopTMM4US34FCAhcDLW6XX6RLbWDkIlvYiM0WltHuFxJUKY2uf8tMaterial Safety Data Sheet of Eculizumab
Avoid inhalation, contact with eyes and skin. Avoid dust and aerosol formation. Use only in areas with appropriate exhaust ventilation. 7.2, Conditions for safe ...
SAFETY DATA SHEET
Based on composition, the mixture has low acute toxicity and no adverse effects for human health are expected under applicable conditions of ...
Real-world safety profile of eculizumab: an analysis of FDA ...
Most of the safety data regarding eculizumab came from clinical trials, while its safety information in the real world is still limited.
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.