Status:
RECRUITING
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Generalized Myasthenia Gravis
gMG
Eligibility:
All Genders
6-17 years
Phase:
PHASE3
Brief Summary
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.
Eligibility Criteria
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
Key Trial Info
Start Date :
November 13 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 18 2029
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06607627
Start Date
November 13 2024
End Date
July 18 2029
Last Update
August 27 2025
Active Locations (12)
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1
Research Site
Washington D.C., District of Columbia, United States, 20010
2
Research Site
Norfolk, Virginia, United States, 23507
3
Research Site
Joinville, Brazil, 89202-451
4
Research Site
Salvador, Brazil, 41253-190