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Evaluating gefurulimab for treating pediatric patients with generalized myasthenia gravis

Q: Who is a good fit for trial NCT06607627?

Ideal candidates are pediatric patients aged 6 to 18 years with a diagnosis of AChR+ generalized myasthenia gravis.

Q: Who should not enroll in trial NCT06607627?

Patients who do not have generalized myasthenia gravis or are outside the specified age range may not benefit from this study.

Q: What is the potential benefit of this trial?

If successful, this study could provide a new treatment option for pediatric patients suffering from generalized myasthenia gravis.

Q: How have similar studies performed?

Other studies have shown promise in treating generalized myasthenia gravis with similar approaches, but this specific intervention is being evaluated for the first time in this pediatric population.

Q: Where is this trial running?

Washington D.C., District of Columbia and 12 other locations

An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)

PHASE3 · Alexion Pharmaceuticals, Inc. · NCT06607627

This study is testing a new drug called gefurulimab to see if it can help children and teens with generalized myasthenia gravis feel better.

Quick facts

Phase	PHASE3
Study type	Interventional
Enrollment	12 (estimated)
Ages	6 Years to 17 Years
Sex	All
Sponsor	Alexion Pharmaceuticals, Inc. (industry)
Drugs / interventions	gefurulimab
Locations	13 sites (Washington D.C., District of Columbia and 12 other locations)
Trial ID	NCT06607627 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients aged 6 to 18 years with a diagnosis of AChR+ generalized myasthenia gravis.

Not a fit: Patients who do not have generalized myasthenia gravis or are outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for pediatric patients suffering from generalized myasthenia gravis.

How similar studies have performed: Other studies have shown promise in treating generalized myasthenia gravis with similar approaches, but this specific intervention is being evaluated for the first time in this pediatric population.

Eligibility criteria

Show full inclusion / exclusion criteria

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 this trial is running

Washington D.C., District of Columbia and 12 other locations

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)
Research Site — São Paulo, Brazil (RECRUITING)
Research Site — Saitama-Shi, Japan (RECRUITING)
Research Site — Bydgoszcz, Poland (WITHDRAWN)
Research Site — Lodz, Poland (WITHDRAWN)
Research Site — Warsaw, Poland (RECRUITING)
Research Site — New Taipei City, Taiwan (RECRUITING)
Research Site — Taipei, Taiwan (RECRUITING)

Study contacts

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

How to participate

Review the eligibility criteria above with your treating physician.
Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Generalized Myasthenia Gravis, gMG, Acetylcholine Receptor antibodies, AChR+, ALXN1720, anti-acetylcholine receptor antibody-positive, acetylcholine receptor, AChR

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.