Alexion C5 Inhibition Therapies for Myasthenia Gravis

Recruiting at 55 trial locations

Overseen By Alexion Pharmaceuticals, Inc. (Sponsor)

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Alexion Pharmaceuticals, Inc.

Must be taking: C5 inhibitors

Disqualifiers: Other Alexion studies

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

500 Participants Needed

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. ^{1 2 3 4 5}

Why 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. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

Inclusion Criteria

Participants with a diagnosis of gMG who are treated with Alexion C5IT at the time of enrollment, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.

Capable of giving signed informed consent, which includes compliance with the protocol requirements and restrictions.

Participants must have myasthenia gravis historical data, such as MG-ADL and MGFA class, available to be enrolled in the Registry.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Enrollment and Retrospective Data Collection

Participant records are queried for retrospective information about medical history and gMG disease treatment history

At enrollment

Prospective Data Collection

Prospective data collection using routine clinical care data and patient-reported outcome methods

Up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Eculizumab
• Ravulizumab

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

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

Trials

247

Recruited

38,600+

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

Trials

231

Recruited

36,700+

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

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37712908/

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.

2. neurology.org neurology.org/doi/10.1212/WNL.98.18_supplement.868

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 ...

3. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1111/ejh.14080

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 ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S0022510X2500245X

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 ...

5. jmcp.org jmcp.org/doi/10.18553/jmcp.2022.28.1.119

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 = ...

6. biosynth.com biosynth.com/Files/MSDS/BE/16/MSDS_BE165588_5000_EN.pdf

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.pdf

Safety 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=AfmBOopTMM4US34FCAhcDLW6XX6RLbWDkIlvYiM0WltHuFxJUKY2uf8t

Material 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 ...

9. iwaichem.com iwaichem.com/wp-content/uploads/ECU-SPEC-ECU_SDS.pdf

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 ...

10. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39140260

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.