NCT06607627PHASE3Recruiting
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Alexion Pharmaceuticals, Inc.
Start Date
11/13/2024
Completion Date
1/23/2029
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
Age Range: 6 years to 17 years
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
Interventions
COMBINATION_PRODUCT
Gefurulimab
Conditions
Generalized Myasthenia GravisgMG
Locations
Research Site
Washington D.C., District of Columbia 20010
United States
Research Site
Norfolk, Virginia 23507
United States
Research Site
Joinville, 89202-451
Brazil
Research Site
Salvador, 41253-190
Brazil
Research Site
São José do Rio Preto, 15090-000
Brazil
Research Site
São Paulo, 0438-002
Brazil
Research Site
São Paulo, 05403-000
Brazil
Research Site
Saitama-Shi, 330-8777
Japan
Research Site
Bydgoszcz, 85-065
Poland
Research Site
Lodz, 93-338
Poland
Research Site
Warsaw, 02-097
Poland
Research Site
New Taipei City, 23561
Taiwan
Research Site
Taipei, 11101
Taiwan