What side effects are associated with this type of intervention?

Common treatments for Myasthenia Gravis include acetylcholinesterase inhibitors (e.g., pyridostigmine), immunosuppressive drugs (e.g., corticosteroids, azathioprine), and monoclonal antibodies (e.g., rituximab). Acetylcholinesterase inhibitors can cause gastrointestinal symptoms, muscle cramps, and increased salivation. Immunosuppressive drugs may lead to increased infection risk, liver toxicity, and gastrointestinal issues. Monoclonal antibodies, which target autoantibodies against AChR, can cause infusion reactions, infections, and potential allergic reactions. Specific side effects of ALXN1720 are not detailed, but it is expected to have similar risks associated with monoclonal antibody treatments.

What prior FDA approvals exist?

FDA-approved treatments for Myasthenia Gravis that target the immune response and autoantibodies against acetylcholine receptors include eculizumab (Soliris), which inhibits the complement system to reduce the destruction of AChR. Pyridostigmine, an acetylcholinesterase inhibitor, is also commonly used to improve neuromuscular transmission. Additionally, immunosuppressive therapies such as corticosteroids, azathioprine, and mycophenolate mofetil are used to manage the autoimmune aspect of MG. These treatments aim to reduce the production or effects of autoantibodies against AChR, similar to the mechanism being studied with ALXN1720.

What other types of research exist?

Promising novel alternatives to ALXN1720 for Myasthenia Gravis patients include rituximab, which has shown benefits in treatment-refractory MG, and other investigational drugs such as zilucoplan and verdiperstat, which are being evaluated in ongoing clinical trials. These treatments target different mechanisms involved in MG and may offer new therapeutic options.

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Monoclonal Antibodies

ALXN1720 for Myasthenia Gravis

Phase 3

Recruiting

Research Sponsored by Alexion Pharmaceuticals, Inc.

Eligibility Criteria Checklist

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

Must have

Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV

Be older than 18 years old

Must not have

Untreated thymic malignancy, carcinoma, or thymoma

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to week 26

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new medication called ALXN1720 to help adults with a type of muscle weakness caused by their immune system. The medication aims to stop the immune system from attacking their muscles, which should help reduce their symptoms.

Who is the study for?

This trial is for adults with generalized Myasthenia Gravis (MG), a condition causing muscle weakness. Participants must have specific autoantibodies against AChR and meet certain MG severity criteria. Those who've had thymus surgery within the last year, untreated thymic tumors, or are pregnant/breastfeeding cannot join.

What is being tested?

The study tests ALXN1720's safety and effectiveness in treating generalized MG compared to a placebo. It targets patients with antibodies against acetylcholine receptors which are linked to this muscle weakness disorder.

What are the potential side effects?

While not specified here, potential side effects of treatments like ALXN1720 could include reactions at the injection site, flu-like symptoms, changes in blood counts or immune system responses that might lead to increased risk of infections or other complications.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have been diagnosed with MG and experience widespread muscle weakness.

Exclusion Criteria

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

Select...

I have an untreated cancer of the thymus.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to week 26

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to week 26

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to week 26 for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Percentage of Responders Based on Reduction of the MG-ADL Total Score at Week 26

Percentage of Responders based on Reduction of the QMG Total Score at Week 26

Awards & Highlights

Pivotal Trial

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

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ALXN1720Experimental Treatment1 Intervention

Participants will receive a weight-based initial (loading) dose of ALXN1720 on Day 1, followed by weight-based maintenance treatment with ALXN1720 on Day 8 and once every week (Q1W) thereafter for a total of 26 weeks. Following this randomized controlled treatment (RCT) period, all participants will receive ALXN1720 in an open-label extension (OLE) period of 105 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive placebo during the 26-week RCT period, after which they will enter the OLE period of the study and receive ALXN1720.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ALXN1720

2022

Completed Phase 1

~20

0 / 2Eligibility 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) target the autoimmune response that leads to the destruction of acetylcholine receptors (AChR) at the neuromuscular junction. Acetylcholinesterase inhibitors, such as pyridostigmine, increase the availability of acetylcholine, enhancing neuromuscular transmission. Immunosuppressive drugs, like corticosteroids and azathioprine, reduce the production of autoantibodies. Intravenous immunoglobulin (IVIG) and plasmapheresis are used to remove or neutralize circulating antibodies. Treatments like ALXN1720 specifically inhibit autoantibodies against AChR, directly addressing the underlying cause of the disease. These treatments are crucial for MG patients as they help improve muscle strength and reduce symptoms, significantly enhancing quality of life.