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RecruitingInterventionalPhase 3

An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)

NCT ID: NCT06607627Sponsor: Alexion Pharmaceuticals, Inc.Last updated: 2026-04-01

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.

Arms & interventions

  • Combination ProductGefurulimab

    Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.

Outcome measures

Primary

  • Maximum Observed Serum Concentration (Cmax) of Gefurulimab

    Time frame: Day 1 predose through Week 18 predose

  • Serum Free Complement Component 5 (C5) Concentration

    Time frame: Day 1 predose through Week 18 predose

Secondary

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

    Time frame: Day 1 through Week 134

  • Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18

    Time frame: Baseline through Week 18

  • Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18

    Time frame: Baseline through Week 18

  • Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18

    Time frame: Baseline through Week 18

  • Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18

    Time frame: Baseline through Week 18

  • Number of Participants With Anti-drug Antibodies (ADAs)

    Time frame: Baseline through Week 134

  • Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses

    Time frame: Baseline through Week 134

  • Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status

    Time frame: Baseline through Week 134

Eligibility criteria

Sex: AllAge: 6 Years to 17 YearsHealthy 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

Study locations (2)

Research Site

Washington D.C., District of Columbia, 20010

Recruiting

Research Site

Norfolk, Virginia, 23507

Recruiting