What side effects are associated with this type of intervention?

Common treatments for Myasthenia Gravis, such as monoclonal antibodies (e.g., rituximab) and intravenous immunoglobulins (IVIg), often aim to modulate the immune system and reduce pathogenic IgG levels. Known side effects of these treatments can include infusion reactions (e.g., fever, chills, rash), increased risk of infections, and potential allergic reactions. Specific to monoclonal antibodies, patients may experience serum-sickness-like reactions and, rarely, more severe conditions like progressive multifocal leukoencephalopathy. IVIg treatments can also cause headaches, nausea, and, in some cases, kidney dysfunction or thrombotic events.

What prior FDA approvals exist?

The FDA has approved several treatments for Myasthenia Gravis, including eculizumab (Soliris), which inhibits the complement protein C5, and rituximab, which targets CD20 on B cells. These treatments aim to reduce the autoimmune attack on the neuromuscular junction. Similar to Rozanolixizumab, which inhibits the neonatal Fc receptor (FcRn) to reduce pathogenic IgG levels, efgartigimod (Vyvgart) has also been approved. Efgartigimod works by binding to FcRn, leading to a reduction in circulating IgG antibodies. These therapies represent a targeted approach to managing MG by modulating the immune system to decrease the levels of harmful antibodies.

What other types of research exist?

Promising novel alternatives to Rozanolixizumab for Myasthenia Gravis patients in 2024 include: 1) Efgartigimod, another FcRn inhibitor that has shown efficacy in reducing pathogenic IgG levels. 2) Rituximab, a monoclonal antibody targeting CD20-positive B cells, which has been used off-label for MG and is being studied further. 3) Zilucoplan, a complement C5 inhibitor that prevents the formation of the membrane attack complex, reducing muscle damage. 4) Ravulizumab, another complement inhibitor similar to eculizumab, offering a longer dosing interval. These therapies offer different mechanisms of action and have shown promise in clinical trials for MG.

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Monoclonal Antibodies

Self-Administered Rozanolixizumab for Myasthenia Gravis

Phase 3

Waitlist Available

Research Sponsored by UCB Biopharma SRL

Eligibility Criteria Checklist

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

Must have

Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG)

Body weight ≥35 kg

Must not have

Study participant has received a live vaccination within 4 weeks before starting treatment, or a Bacillus Calmette-Guérin (BCG) vaccine within 1 year before starting treatment; or intends to have a live vaccination during the course of the study or within 8 weeks following the last dose of rozanolixizumab

Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring IV antibiotic treatment) within 6 weeks before the Baseline Visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 hours after each administration during the training period (baseline to visit 7 [week 6] and self-administration periods (visit 8 [week 7] to visit 19 [week 18])

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial aims to see if patients with generalized Myasthenia Gravis can successfully learn to inject themselves with rozanolixizumab, a medication that helps reduce muscle weakness by targeting harmful antibodies. Rozanolixizumab is being tested to see how well it works and how safe it is for treating generalized myasthenia gravis.

Who is the study for?

This trial is for individuals with generalized Myasthenia Gravis (gMG) who are capable of self-administering medication and weigh at least 35 kg. Participants must be willing to use a syringe driver or manual push method after training. Those with severe muscle weakness, hypersensitivity to similar drugs, active infections, recent live vaccinations, or certain medical histories cannot join.

What is being tested?

The study tests if patients with gMG can successfully self-administer the drug Rozanolixizumab using a syringe driver or by hand. The focus is on patient independence in managing their treatment after receiving proper instruction.

What are the potential side effects?

While specific side effects are not listed here, potential risks may include reactions related to the immune system due to the nature of Rozanolixizumab as an immunotherapy and local issues at the injection site from self-administration.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with generalized Myasthenia Gravis.

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My body weight is at least 35 kg.

Exclusion Criteria

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

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I have not had a live vaccine recently and do not plan to during the study.

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I haven't had a serious infection or been hospitalized for one in the last 6 weeks.

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I have severe muscle weakness due to myasthenia gravis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 24 hours after each administration during the training period (baseline to visit 7 [week 6] and self-administration periods (visit 8 [week 7] to visit 19 [week 18])

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 24 hours after each administration during the training period (baseline to visit 7 [week 6] and self-administration periods (visit 8 [week 7] to visit 19 [week 18])

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 hours after each administration during the training period (baseline to visit 7 [week 6] and self-administration periods (visit 8 [week 7] to visit 19 [week 18]) for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respectively) during the Self-administration Period at Visit 13

Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respectively) during the Self-administration Period at Visit 19

Secondary study objectives

Occurrence of Treatment-Emergent Adverse Events (TEAEs) after syringe driver or manual push self-administration from Visit 2 up to the End of Study Visit

Occurrence of local site reactions up to 24 hours after each administration during the Training Period and Self-administration Periods

Occurrence of medication errors associated with adverse reactions during the 2 Self-administration Periods of the study

Side effects data

From 2021 Phase 3 trial • 71 Patients • NCT04124965

30%

Headache

12%

Blood immunoglobulin G decreased

12%

Diarrhoea

10%

Urinary tract infection

Nausea

Pyrexia

Myasthenia gravis

Hypertension

Back pain

Nasopharyngitis

Abdominal pain

Hypogammaglobulinaemia

Retinal detachment

Muscular weakness

Cardiac failure congestive

Biopsy kidney abnormal

Rash

100%

80%

60%

40%

20%

Study treatment Arm

Rozanolixizumab ~7 mg/kg

Rozanolixizumab ~10 mg/kg

Awards & Highlights

Pivotal Trial

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

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: Rozanolixizumab Sequence 2: Manual Push - Syringe DriverExperimental Treatment1 Intervention

Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks.

Group II: Rozanolixizumab Sequence 1: Syringe Driver - Manual PushExperimental Treatment1 Intervention

Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rozanolixizumab

2023

Completed Phase 3

~620

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 acetylcholinesterase inhibitors, immunosuppressive drugs, and immunomodulating therapies. Acetylcholinesterase inhibitors, such as pyridostigmine, work by increasing the amount of acetylcholine at the neuromuscular junction, improving muscle contraction. Immunosuppressive drugs, like corticosteroids and azathioprine, reduce the immune system's attack on the neuromuscular junction. Immunomodulating therapies, such as intravenous immunoglobulin (IVIG) and therapeutic plasma exchange, provide rapid but temporary relief by removing or neutralizing pathogenic antibodies. Treatments like Rozanolixizumab, which inhibit the neonatal Fc receptor (FcRn), aim to reduce levels of pathogenic IgG antibodies, thereby decreasing the immune attack on acetylcholine receptors. These mechanisms are crucial for MG patients as they help alleviate muscle weakness and improve overall function by targeting the underlying autoimmune process.