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

Phase 3

Recruiting

• Conditions: Generalized Myasthenia Gravis; gMG

• Registration Number: NCT06607627
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-19
• Study First Submitted QC: 2024-09-19
• Study First Posted: 2024-09-23
• Study Start: 2024-11-13
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-26
• Last Update Posted: 2025-08-27
• Primary Completion: 2027-04-16
• Study Completion: 2029-07-18

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Serum Free Complement Component 5 (C5) Concentration	Day 1 predose through Week 18 predose
Maximum Observed Serum Concentration (Cmax) of Gefurulimab	Day 1 predose through Week 18 predose

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	Day 1 through Week 134
Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18	Baseline through Week 18
Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18	Baseline through Week 18
Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18	Baseline through Week 18
Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18	Baseline through Week 18
Number of Participants With Anti-drug Antibodies (ADAs)	Baseline through Week 134
Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses	Baseline through Week 134
Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status	Baseline through Week 134

Trial Locations

Locations (1)

Research Site; 🇨🇳 Taipei, Taiwan