Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
Summary
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
Arms & interventions
- BiologicalEfgartigimod IV
Intravenous infusion of Efgartigimod
Outcome measures
Primary
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Clearance (CL)
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Time frame: up to 26 weeks
Efgartigimod concentrations as input for compartmental, model-driven analysis to determine (age and size dependency of) Volume of Distribution (Vd)
Blood samples will be collected from each participant for measurement of serum concentrations of efgartigimod
Time frame: up to 26 weeks
Total Immunoglobulin G (IgG) levels as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
Total Immunoglobulin G levels will be measured from blood samples
Time frame: up to 26 weeks
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK) and pharmacodynamics (PD) modeling analysis
Total Immunoglobulin G (IgG) levels will be measured from blood samples
Time frame: up to 26 weeks
Secondary
Incidence and severity of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs)
Time frame: up to 28 weeks
Efgartigimod serum concentrations from blood samples
Time frame: up to 26 weeks
Absolute values of levels of total Immunoglobulin G (IgG) from blood samples
Time frame: up to 26 weeks
Change from baseline of levels of total Immunoglobulin G (IgG) from blood samples
Time frame: up to 26 weeks
Percentage change from baseline of total Immunoglobulin G (IgG) from blood samples
Time frame: up to 26 weeks
Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: up to 26 weeks
Change from baseline of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: up to 26 weeks
Percentage change from baseline of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: up to 26 weeks
Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: up to 28 weeks
Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: up to 28 weeks
Absolute values of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score. Total score can range from 0 to 24, with higher total scores indicating more impairment.
Time frame: up to 26 weeks
Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score. Total score can range from 0 to 24, with higher total scores indicating more impairment.
Time frame: up to 26 weeks
Absolute values of total Quantitative Myasthenia Gravis Score (QMG score). The total possible score is 39, where higher total scores indicate more severe impairments.
Time frame: up to 26 weeks
Change from baseline of total Myasthenia Gravis Score (QMG score). The total possible score is 39, where higher total scores indicate more severe impairments.
Time frame: up to 26 weeks
Absolute values of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Time frame: up to 26 weeks
Change from baseline of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Time frame: up to 26 weeks
Values of Neurological Quality of Life (Neuro-QoL) pediatric fatigue questionnaire
Time frame: up to 26 weeks
Change from baseline of Neurological Quality of Life (Neuro-QoL) pediatric fatigue questionnaire
Time frame: up to 26 weeks
Change in protective antibody titers to vaccines received before or during the trial from blood samples
Time frame: up to 28 weeks
Eligibility criteria
Study locations (3)
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois, 60611
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Virginia (UVA) Health - Developmental Pediatrics Clinic
Charlottesville, Virginia, 22903