What is the mechanism of action of this treatment?

The most common treatments for Myasthenia Gravis (MG) include complement inhibitors, acetylcholinesterase inhibitors, immunosuppressants, and plasmapheresis. Complement inhibitors, such as eculizumab and the investigational ALXN2050, block the complement system to prevent the immune-mediated destruction of acetylcholine receptors, which is crucial for reducing muscle weakness in MG patients. Acetylcholinesterase inhibitors increase acetylcholine availability, enhancing muscle contraction. Immunosuppressants lower the immune response, reducing antibody production against acetylcholine receptors. Plasmapheresis removes these harmful antibodies from the blood. These treatments collectively help manage the immune attack on the neuromuscular junction, improving muscle function and quality of life for MG patients.

What side effects are associated with this type of intervention?

Common treatments for Myasthenia Gravis include acetylcholinesterase inhibitors, corticosteroids, immunosuppressants, plasmapheresis, and intravenous immunoglobulin (IVIG). Acetylcholinesterase inhibitors can cause gastrointestinal symptoms and muscle cramps. Corticosteroids may lead to weight gain, diabetes, and osteoporosis. Immunosuppressants like azathioprine and mycophenolate mofetil can cause liver toxicity and increased infection risk. Plasmapheresis and IVIG are generally well-tolerated but can cause headaches, dizziness, and allergic reactions. Complement inhibitors, such as eculizumab, used in MG can lead to increased risk of infections, particularly meningococcal infections, and infusion-related reactions.

What prior FDA approvals exist?

The FDA has approved several treatments for Myasthenia Gravis, including complement inhibitors like eculizumab (Soliris). Eculizumab was the first complement inhibitor approved for generalized Myasthenia Gravis in adults who are anti-acetylcholine receptor (AChR) antibody-positive. This drug works by inhibiting the complement system, which is part of the immune response that contributes to the symptoms of Myasthenia Gravis. Other treatments include acetylcholinesterase inhibitors like pyridostigmine, immunosuppressants such as azathioprine and mycophenolate mofetil, and monoclonal antibodies like rituximab. These treatments aim to reduce the immune system's attack on the neuromuscular junction, thereby alleviating symptoms.

What other types of research exist?

Promising novel alternatives to ALXN2050 for Myasthenia Gravis patients include: 1) Zilucoplan, a complement C5 inhibitor, which has shown potential in clinical trials. 2) Efgartigimod, an FcRn antagonist that reduces pathogenic IgG antibodies. 3) Rozanolixizumab, another FcRn antagonist under investigation for its efficacy in MG. These therapies are being studied for their ability to improve symptoms and quality of life in MG patients.

← Back to Search

Monoclonal Antibodies

ALXN2050 for Myasthenia Gravis

Phase 2

Waitlist Available

Research Sponsored by Alexion Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at the Screening Visit.

MG-ADL total score must be ≥ 5 at the Screening Visit and at randomization (Day 1) with at least 50% of the score attributed to non-ocular elements

Must not have

Use of intravenous immunoglobulin G or subcutaneous immunoglobulin within the 4 weeks (28 days) prior to the Screening Visit

Untreated thymic malignancy, carcinoma, or thymoma

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing a medication called ALXN2050 to see if it helps people with generalized myasthenia gravis, a condition that causes muscle weakness. The study will monitor safety and effectiveness over time and includes a phase where all participants will receive the medication.

Who is the study for?

This trial is for adults with generalized myasthenia gravis (gMG), a condition causing muscle weakness. Participants must have had gMG symptoms scored at a certain level, been diagnosed at least 3 months prior, and be on stable doses of specific treatments without expecting changes during the study. Those with recent thymic surgery or certain severe kidney issues cannot join.

What is being tested?

The trial is testing ALXN2050's effectiveness and safety in treating gMG compared to a placebo. Two different doses of ALXN2050 (120 mg and 180 mg) are being studied to see if they can improve muscle strength in participants.

What are the potential side effects?

While the side effects of ALXN2050 are not detailed here, typically such trials monitor for any adverse reactions including but not limited to allergic responses, injection site reactions, fatigue, or exacerbation of gMG symptoms.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My myasthenia gravis is moderate to severe.

Select...

My MG-ADL score is 5 or more, mostly due to non-eye symptoms.

Select...

My myasthenia gravis is mild to severe but not requiring intubation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I haven't used intravenous or subcutaneous immunoglobulin in the last 4 weeks.

Select...

I have an untreated cancer of the thymus.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: ALXN2050: 180 mgExperimental Treatment1 Intervention

Participants will receive ALXN2050.

Group II: ALXN2050: 120 mgExperimental Treatment1 Intervention

Participants will receive ALXN2050.

Group III: PlaceboPlacebo Group2 Interventions

Participants will receive placebo followed by ALXN2050.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ALXN2050

2021

Completed Phase 1

~250

0 / 5Eligibility criteria met

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 complement inhibitors, acetylcholinesterase inhibitors, immunosuppressants, and plasmapheresis. Complement inhibitors, such as eculizumab and the investigational ALXN2050, block the complement system to prevent the immune-mediated destruction of acetylcholine receptors, which is crucial for reducing muscle weakness in MG patients. Acetylcholinesterase inhibitors increase acetylcholine availability, enhancing muscle contraction. Immunosuppressants lower the immune response, reducing antibody production against acetylcholine receptors. Plasmapheresis removes these harmful antibodies from the blood. These treatments collectively help manage the immune attack on the neuromuscular junction, improving muscle function and quality of life for MG patients.

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