Gefurulimab: Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
Study 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
Sex
ALL
Min age
6 Years
Max age
17 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Maximum Observed Serum Concentration (Cmax) of Gefurulimab — Day 1 predose through Week 18 predose
Serum Free Complement Component 5 (C5) Concentration — Day 1 predose through Week 18 predose
Trial sites (13)
Facility
City
Region
Status
Research Site
Washington D.C.
District of Columbia
Recruiting
Research Site
Norfolk
Virginia
Recruiting
Research Site
Joinville
Brazil
Recruiting
Research Site
Salvador
Brazil
Recruiting
Research Site
São José do Rio Preto
Brazil
Recruiting
Research Site
São Paulo
Brazil
Recruiting
Research Site
São Paulo
Brazil
Recruiting
Research Site
Saitama-Shi
Japan
Recruiting
Research Site
Bydgoszcz
Poland
Withdrawn
Research Site
Lodz
Poland
Withdrawn
Research Site
Warsaw
Poland
Recruiting
Research Site
New Taipei City
Taiwan
Recruiting
Research Site
Taipei
Taiwan
Recruiting
More Alexion Pharmaceuticals, Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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