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+)
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
Study locations (2)
Research Site
Washington D.C., District of Columbia, 20010
Research Site
Norfolk, Virginia, 23507