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PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
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+)
Who can join
Ages 6 to 17 · All sexes
Full eligibility 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
About the study
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.
What is being tested
- Gefurulimab (combination product)
Sponsor: Alexion Pharmaceuticals, Inc. · Participants: 12 · Started: Nov 13, 2024
Contact the study team
- Alexion Pharmaceuticals, Inc. (Sponsor) · Phone: 1-855-752-2356
Official record on ClinicalTrials.gov — NCT06607627
Locations in the U.S.
| District of Columbia | Research Site, Washington D.C. |
| Virginia | Research Site, Norfolk |
Conditions
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.