PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis

Launched by ALEXION PHARMACEUTICALS, INC. · Sep 19, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a medication called gefurulimab to see how it works and how safe it is for children and teens aged 6 to 17 who have a type of muscle weakness known as generalized myasthenia gravis (gMG). The trial aims to understand how the body processes the medication and its effects on the symptoms of gMG. Researchers are looking for participants who have been diagnosed with this condition and have specific test results showing they have antibodies related to it.

To join the study, participants must be between 6 and 17 years old, diagnosed with gMG, and have received certain vaccinations against meningococcal infections in the past few years. Children who have had recent surgery on their thymus gland or have certain health conditions will not be eligible. If chosen to participate, families can expect close monitoring during the study to ensure the child’s safety and to track how well the medication works. This trial is currently recruiting participants, and it offers a chance to help improve treatments for gMG in young patients.

Gender

ALL

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