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)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Intravenous infusion of Efgartigimod
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois, United States
RECRUITINGUniversity of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
RECRUITINGUniversity of Virginia (UVA) Health - Developmental Pediatrics Clinic
Charlottesville, Virginia, United States
RECRUITINGMedizinische Universitat Wien
Vienna, Austria
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
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)
Description of the participant's health state is done by digits for 5 dimensions combined in a 5-digit number. A unique health state is defined by combining 1 level from each of the 5 dimensions. Each state is referred to in terms of a 5-digit code, whereas code 11111 would indicate no problems in any of the 5 dimensions and 33333 would indicate worst problems in any of the 5 dimensions.
Time frame: up to 26 weeks
Change from baseline of total score EuroQoL 5 Dimensions Youth (EQ-5D-Y)
Description of the participant's health state is done by digits for 5 dimensions combined in a 5-digit number. A unique health state is defined by combining 1 level from each of the 5 dimensions. Each state is referred to in terms of a 5-digit code, whereas code 11111 would indicate no problems and 33333 would indicate worst problems in any of the 5 dimensions.
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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Universitair Ziekenhuis Antwerpen
Antwerp, Belgium
RECRUITINGBritish Columbia Children's Hospital
Vancouver, Canada
RECRUITINGAP-HM - Hopital de la Timone
Marseille, France
RECRUITINGAssistance Publique Hopitaux de Paris (AP-HP) - Hopital Necker-Enfants Malades
Paris, France
RECRUITINGVian - M. Iashvili Children's Central Hospital
Tbilisi, Georgia
COMPLETEDTbilisi State Medical University - Givi Zhvania Pediatric Academic Clinic
Tbilisi, Georgia
COMPLETED...and 14 more locations