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

Alexion Pharmaceuticals

Status and phase: Enrolling; Phase 3

Conditions: gMG; Generalized Myasthenia Gravis

Treatments: Combination Product: Gefurulimab

Study type: Interventional

Funder types: Industry

Identifiers

NCT06607627

ALXN1720-MG-302 (Other Identifier)

D6780C00004

2024-515157-17-00 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 12 estimated patients

Sex: All

Ages: 6 to 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Gefurulimab

Experimental group

Description:

On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.

Treatment:

Combination Product: Gefurulimab