Gefurulimab for Myasthenia Gravis

Recruiting in Palo Alto (17 mi)

+10 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Alexion Pharmaceuticals, Inc.

Disqualifiers: Thymectomy, Thymic malignancy, Pregnancy, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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.

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}

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

Trial 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 (Monoclonal Antibodies)

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.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Research Site Washington, United States

Research Site Norfolk, VA

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc. Lead Sponsor

References

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

Consistent improvement with eculizumab across muscle groups in myasthenia gravis. [2023] To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups.

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] To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension.

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

A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus. [2022] In 2017, eculizumab has been approved for treatment-refractory generalised myasthenia gravis (TRgMG). The German Myasthenia Foundation has published a consensus statement on the use of eculizumab, with a recent update. However, a treatment-refractory state is still ill-defined and the term warrants further clarification. We aimed at developing a sum score to operationalise the definition of a TRgMG status, which is easy- to-handle in clinical decision making.

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] To evaluate whether the clinical benefit and relapse rates in anti-muscle-specific kinase (MuSK) myasthenia gravis (MG) differ depending on the protocol of rituximab followed.

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] Label="BACKGROUND" NlmCategory="BACKGROUND">In the phase III eculizumab for refractory generalized myasthenia gravis REGAIN study [ClinicalTrials.gov identifier: NCT01997229] and its open-label extension (OLE) [ClinicalTrials.gov identifier: NCT02301624], patients with treatment-refractory antiacetylcholine receptor antibody-positive generalized myasthenia gravis had clinically meaningful improvements with eculizumab versus placebo. This subgroup analysis evaluated data from patients with a recent history of chronic intravenous immunoglobulin (IVIg) use before study entry.