What side effects are associated with this type of intervention?

Common treatments for Myasthenia Gravis include intravenous immune globulin (IVIG), plasmapheresis, and immunosuppressive drugs such as corticosteroids, azathioprine, and mycophenolate mofetil. IVIG can cause side effects like headache, fever, chills, and infusion reactions. Plasmapheresis may lead to citrate reactions and vascular access issues. Immunosuppressive drugs can result in increased infection risk, gastrointestinal symptoms, and liver toxicity. For treatments similar to Zilucoplan, such as other complement inhibitors, potential side effects may include infusion reactions, infections, and hypersensitivity reactions.

What prior FDA approvals exist?

The FDA has approved several treatments for Myasthenia Gravis, including acetylcholinesterase inhibitors like pyridostigmine, immunosuppressants such as azathioprine and mycophenolate mofetil, and monoclonal antibodies like eculizumab, which is a complement C5 inhibitor similar to Zilucoplan. Eculizumab works by inhibiting the complement system, thereby reducing the destruction of acetylcholine receptors at the neuromuscular junction, which is a key mechanism in MG. This makes it a relevant comparison to Zilucoplan, which is also being studied for its effects on the complement pathway in MG.

What other types of research exist?

Promising novel alternatives to Zilucoplan for Myasthenia Gravis patients include Eculizumab and Ravulizumab, both of which are complement C5 inhibitors. Additionally, treatments targeting other pathways, such as Efgartigimod (an FcRn antagonist) and Rozanolixizumab (an anti-FcRn monoclonal antibody), are also emerging as effective options. These therapies have shown efficacy in clinical trials and may provide new options for MG patients in 2024.

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Monoclonal Antibodies

Zilucoplan for Myasthenia Gravis (RAISE-XT Trial)

Phase 3

Waitlist Available

Research Sponsored by Ra Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline (day 1) to safety follow-up visit (up to 36 months)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing zilucoplan, a medication for muscle weakness, in patients with generalized Myasthenia Gravis who were in previous studies. It aims to see if the drug is safe and effective over time by calming the immune system to prevent it from attacking muscles. Zilucoplan is currently being tested for its potential to improve disease control in generalized myasthenia gravis.

Who is the study for?

This trial is for individuals with generalized Myasthenia Gravis (gMG) who have already completed a qualifying study involving zilucoplan. They should not be part of any other clinical trials testing new treatments, although observational studies are okay.

What is being tested?

The RAISE-XT study is testing the long-term effects of zilucoplan on people with gMG. It's an open-label extension, meaning everyone knows they're getting zilucoplan and there's no placebo comparison.

What are the potential side effects?

While specific side effects aren't listed here, common ones for drugs like zilucoplan may include injection site reactions, muscle or joint pain, headaches, and potential immune system impacts.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline (day 1) to safety follow-up visit (up to 36 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline (day 1) to safety follow-up visit (up to 36 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline (day 1) to safety follow-up visit (up to 36 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of treatment-emergent adverse events (TEAEs)

Secondary study objectives

Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score

Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score

Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score

+1 more

Side effects data

From 2021 Phase 3 trial • 174 Patients • NCT04115293

16%

Injection site bruising

15%

Headache

10%

Diarrhoea

Injection site pain

Myasthenia gravis

Urinary tract infection

Contusion

Lipase increased

Nasopharyngitis

Amylase increased

Rash

Vomiting

Aphthous ulcer

Basal cell carcinoma

Angioedema

COVID-19

Pneumonia

Pulmonary embolism

Oesophageal candidiasis

Oral candidiasis

COVID-19 pneumonia

Sepsis

Anaemia

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Zilucoplan 0.3 mg/kg

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: 0.3 mg/kg zilucoplan (RA101495)Experimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

zilucoplan (RA101495)

2022

Completed Phase 3

~250

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Myasthenia Gravis (MG) include acetylcholinesterase inhibitors, immunosuppressive drugs, and complement inhibitors. Acetylcholinesterase inhibitors, such as pyridostigmine, increase the availability of acetylcholine at the neuromuscular junction, improving muscle contraction. Immunosuppressive drugs like corticosteroids and azathioprine reduce the immune system's attack on acetylcholine receptors, decreasing symptoms. Complement inhibitors, such as Zilucoplan, block the complement system's role in the destruction of acetylcholine receptors, preventing further muscle weakness. These treatments are crucial for MG patients as they target different aspects of the disease's pathophysiology, offering symptomatic relief and slowing disease progression.

Update in immunosuppressive therapy of myasthenia gravis.[Myasthenia gravis].