Evaluating gefurulimab for treating pediatric patients with generalized myasthenia gravis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric patients diagnosed with AChR+ generalized myasthenia gravis (gMG). It involves administering the drug to participants aged 6 to 18 years and monitoring its effects over the study duration. The study will evaluate how the drug is processed in the body and its efficacy in alleviating symptoms of gMG. Participants must meet specific vaccination criteria to ensure safety during the trial.

Why it matters

Who can join: the full rules

Inclusion Criteria:

United States of America (USA) specific inclusion criterion:

* Participant must be 12 to \< 18 years of age at the time of signing the informed consent/assent.
* All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.

Rest of World (ROW) specific inclusion criteria:

* Participant must be 6 to \< 18 years of age at the time of signing the informed consent/assent.
* All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs \< 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available)

Global inclusion criteria:

* Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
* Positive serological test for autoantibodies against AChR

Exclusion Criteria:

* History of thymectomy, or any other thymic surgery within 12 months prior to Screening
* Untreated thymic malignancy, carcinoma, or thymoma
* History of Neisseria meningitidis infection
* Pregnancy, breastfeeding, or intention to conceive during the course of the study

Where it's running

• Research Site
  Washington D.C., District of Columbia, United States Recruiting
• Research Site
  Norfolk, Virginia, United States Recruiting
• Research Site
  Joinville, Brazil Recruiting
• Research Site
  Salvador, Brazil Recruiting
• Research Site
  São José do Rio Preto, Brazil Recruiting
• Research Site
  São Paulo, Brazil Recruiting

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Ravulizumab for Generalized Myasthenia Gravis in Chinese Adults

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• ADAPT Forward master protocol testing multiple regimens for generalized myasthenia gravis

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• Adding empasiprubart to efgartigimod for AChR-antibody positive generalized myasthenia gravis

  This trial will test whether adding empasiprubart to efgartigimod infusions helps people with AChR-antibody positive generalized myasthenia gravis who have only had a partial response to efgartigimod.

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• Oral steroid tapering for adults with generalized myasthenia gravis on ravulizumab.

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• Empasiprubart IV in people with AChR-Ab positive generalized myasthenia gravis

  This study tests whether empasiprubart given by IV can help people with AChR-Ab positive generalized myasthenia gravis feel better and have fewer symptoms.

  Recruiting at 14 sites Phase 2 argenx · Carlsbad, California and 13 other locations · NCT07673627

• SHR-2173 injection added to standard care for generalized myasthenia gravis

  This Phase 2 trial tests whether SHR-2173 added to usual treatment helps adults with generalized myasthenia gravis more than a placebo add-on.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.