Tests gefurulimab in children with a muscle-weakness disease called myasthenia gravis
Tests gefurulimab in children aged 6 to 17 with antibody-positive generalized myasthenia gravis.
Plain-language title generated from the registry record
Registry title: PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Key facts
- Where
- Saitama-shi, JapanSee the site
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 6 and 17 years old?
Source: NCT06607627, who can take part
Where in Japan
- Saitama-shiSaitama-shi, JapanTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Alexion Pharmaceuticals, Inc.
- Trial code
- NCT06607627EU 2024-515157-17-00
- Record updated
- 1 April 2026
- Original 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.Read full original descriptionShow less
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+)
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.
Inclusion: 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: 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.