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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
Research Site
Washington D.C., District of Columbia, United States
RECRUITINGResearch Site
Norfolk, Virginia, United States
RECRUITINGMaximum 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
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
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Research Site
Joinville, Brazil
RECRUITINGResearch Site
Salvador, Brazil
RECRUITINGResearch Site
São José do Rio Preto, Brazil
RECRUITINGResearch Site
São Paulo, Brazil
RECRUITINGResearch Site
São Paulo, Brazil
RECRUITINGResearch Site
Saitama-shi, Japan
NOT_YET_RECRUITINGResearch Site
Bydgoszcz, Poland
NOT_YET_RECRUITINGResearch Site
Lodz, Poland
NOT_YET_RECRUITING...and 3 more locations
Time frame: Baseline through Week 134