NCT06607627 · Alexion Pharmaceuticals, Inc.
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
What this study is about
The primary objective of this study is to assess the how the drug moves through the body and how the drug affects the body of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
View original scientific description
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.
Interventions
COMBINATION_PRODUCT
Gefurulimab
Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
Primary outcome measures
Maximum Observed Serum Concentration (Cmax) of Gefurulimab
Time frame: Day 1 predose through Week 18 predose
Serum Free Complement Component 5 (C5) Concentration
Time frame: Day 1 predose through Week 18 predose
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- Washington D.C., District of Columbia
- Norfolk, Virginia
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 1, 2026 · Source of record for eligibility and locations