An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
Summary
The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.
Arms & interventions
- BiologicalEfgartigimod PH20 SC
Subcutaneous injections
Outcome measures
Primary
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL)
Time frame: Up to 12 weeks
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd)
Time frame: Up to 12 weeks
Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis
Time frame: Up to 12 weeks
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis
Time frame: Up to 12 weeks
Secondary
Incidence of adverse events (AEs)
Time frame: Up to 14 weeks
Severity of adverse events (AEs)
Time frame: Up to 14 weeks
Incidence of serious adverse events (SAEs)
Time frame: Up to 14 weeks
Severity of serious adverse events (SAEs)
Time frame: Up to 14 weeks
Incidence of adverse events of special interest (AESI)
Time frame: Up to 14 weeks
Severity of adverse events of special interest (AESI)
Time frame: Up to 14 weeks
Efgartigimod serum concentrations
Time frame: Up to 12 weeks
Absolute values of total Immunoglobulin G (IgG) from blood samples
Time frame: Up to 12 weeks
Change from baseline values of total Immunoglobulin G (IgG) from blood samples
Time frame: Up to 12 weeks
Percentage change from baseline values of total Immunoglobulin G (IgG) from blood samples
Time frame: Up to 12 weeks
Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: Up to 12 weeks
Change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: Up to 12 weeks
Percentage change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: Up to 12 weeks
Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: Up to 12 weeks
Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: Up to 12 weeks
Incidence of antibodies against rHuPH20 in serum samples
Time frame: Up to 12 weeks
Prevalence of antibodies against rHuPH20 in serum samples
Time frame: Up to 12 weeks
Absolute value of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use
Time frame: Up to 12 weeks
Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use
Time frame: Up to 12 weeks
Absolute value of Quantitative Myasthenia Gravis (QMG) score
Time frame: Up to 12 weeks
Change from baseline of Quantitative Myasthenia Gravis (QMG) score
Time frame: Up to 12 weeks
Absolute value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score
Time frame: Up to 12 weeks
Change from baseline value of Neuro-QoL Pediatric Fatigue Score
Time frame: Up to 12 weeks
Change from baseline value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score
Time frame: Up to 12 weeks
Change from baseline value of Clinical Global Impression of Improvement (CGI-I)
Time frame: Up to 12 weeks
Changes in protective antibody titers to vaccines
Time frame: Up to 12 weeks
Eligibility criteria
Study locations (3)
Carolinas HealthCare System Neurosciences Institute - Neurology
Charlotte, North Carolina, 28207
Neurology Rare Disease Center
Denton, Texas, 76208
University of Virginia
Charlottesville, Virginia, 22903