Gefurulimab for Myasthenia Gravis

Recruiting at 10 trial locations

Overseen By Alexion Pharmaceuticals, Inc. (Sponsor)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Alexion Pharmaceuticals, Inc.

Disqualifiers: Thymectomy, Thymic malignancy, Pregnancy, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 122 weeks

12 Participants Needed

Trial Summary

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the drug Gefurulimab for treating myasthenia gravis?

Research on a similar drug, eculizumab, shows it helps patients with a specific type of myasthenia gravis (a condition causing muscle weakness) by improving their ability to perform daily activities and reducing symptoms across different muscle groups. ^{1 2 3 4 5}

What is the purpose of this trial?

The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.

Eligibility Criteria

This trial is for pediatric patients aged 6 to less than 18 with generalized muscle weakness due to AChR+ Myasthenia Gravis. They must have tested positive for specific autoantibodies and be vaccinated against certain meningococcal infections, or take antibiotics if recently vaccinated. Those with untreated thymic conditions, recent thymic surgery, a history of certain infections, or who are pregnant cannot participate.

Inclusion Criteria

Positive serological test for autoantibodies against AChR

I have been vaccinated against meningococcal infection within the last 3 years.

I am between 6 and 17 years old.

Exclusion Criteria

I have an untreated cancer of the thymus.

I have not had thymus gland surgery in the last year.

History of Neisseria meningitidis infection

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a weight-based loading dose of gefurulimab followed by a weekly maintenance dose

122 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

Treatment Details

Interventions

• Gefurulimab

Trial Overview The study aims to evaluate the effects and body's handling (pharmacokinetics/pharmacodynamics) of Gefurulimab in children with AChR+ generalized Myasthenia Gravis over the course of the study.

Participant Groups

Group I: Gefurulimab Experimental Treatment 1 Intervention

On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.

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

Findings from Research

Eculizumab, a terminal complement inhibitor, significantly improves muscle strength and daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis, as shown in a study with 125 participants.

Improvements in muscle strength and daily living activities were rapid and sustained over 130 weeks, indicating the long-term efficacy of eculizumab in managing this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Consistent improvement with eculizumab across muscle groups in myasthenia gravis. Mantegazza, R., O'Brien, FL., Yountz, M., et al.[2023]

Eculizumab significantly improved outcomes in patients with anti-acetylcholine receptor-positive refractory generalized myasthenia gravis (gMG), with 25.0% achieving minimal manifestations (MM) after 26 weeks compared to 13.3% in the placebo group.

After 130 weeks of treatment, 88.0% of patients on eculizumab reported improved status, and 57.3% achieved MM, demonstrating both rapid and sustained efficacy without new safety concerns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Mantegazza, R., Wolfe, GI., Muppidi, S., et al.[2021]

A new sum score has been developed to define treatment-refractory generalized myasthenia gravis (TRgMG), which includes criteria such as disease severity and inefficacy of previous therapies, making it easier for clinicians to make treatment decisions.

This scoring system, created through a consensus of 12 experts and based on a thorough literature review, aims to clarify when eculizumab should be used and can also serve as a basis for inclusion criteria in future clinical studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus. Schroeter, M., Berger, B., Blaes, F., et al.[2022]

In a study of 25 patients with anti-muscle-specific kinase (MuSK) myasthenia gravis, the rituximab protocol 4 + 2 resulted in significantly fewer relapses (18.2%) and a longer time to relapse (mean 3.5 years) compared to other protocols.

The protocol 1 + 1 had a much higher relapse rate (80%) and a shorter time to relapse (mean 1.1 years), indicating that the choice of rituximab protocol can greatly affect treatment outcomes in MuSK MG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The impact of rituximab infusion protocol on the long-term outcome in anti-MuSK myasthenia gravis. Cortés-Vicente, E., Rojas-Garcia, R., Díaz-Manera, J., et al.[2022]

In a subgroup of patients with treatment-refractory generalized myasthenia gravis who had previously received chronic intravenous immunoglobulin (IVIg), eculizumab demonstrated sustained clinical improvements over 18 months compared to placebo.

Eculizumab was associated with a lower rate of disease exacerbations and was well tolerated, indicating its efficacy and safety for patients transitioning from IVIg treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. Jacob, S., Murai, H., Utsugisawa, K., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Consistent improvement with eculizumab across muscle groups in myasthenia gravis. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

The impact of rituximab infusion protocol on the long-term outcome in anti-MuSK myasthenia gravis. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. [2022]