ARGX-113-2207: 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

Conditions

Generalized Myasthenia Gravis

Brief summary

Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of CL and Vd 2. PD parameters: total IgG levels and AChR-Ab as input for PK/PD modeling analysis

Detailed description

Incidence and severity of adverse events (AEs), including serious AEs and AEs of special interest • Changes in vital signs, electrocardiogram (ECGs), and clinical laboratory tests • Efgartigimod serum concentrations • Total IgG and AChR-Ab levels: absolute values, changes from baseline values, and percent (%) reductions from baseline values", Prevalence and incidence of antidrug antibodies (ADA) against efgartigimod and antibodies against rHuPH20 • Myasthenia Gravis Activities of Daily Living (MG-ADL) total score: absolute value and change from baseline appropriate for pediatric use • Quantitative Myasthenia Gravis (QMG): absolute value and change from baseline ", EQ-5D-Y: absolute value and change from baseline • Quality of Life in Neurological Disorders Questionnaire (Neuro-QoL) Pediatric Fatigue Score: change from baseline • Clinical Global Impression of Improvement (CGI-I): change from baseline • Changes in protective antibody titers to vaccines

Related papers

No related papers found yet.

Interventions

DRUGVyvgart 1 000 mg solution for injection

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of CL and Vd 2. PD parameters: total IgG levels and AChR-Ab as input for PK/PD modeling analysis	—

Secondary

Measure	Time frame
Incidence and severity of adverse events (AEs), including serious AEs and AEs of special interest • Changes in vital signs, electrocardiogram (ECGs), and clinical laboratory tests • Efgartigimod serum concentrations • Total IgG and AChR-Ab levels: absolute values, changes from baseline values, and percent (%) reductions from baseline values", Prevalence and incidence of antidrug antibodies (ADA) against efgartigimod and antibodies against rHuPH20 • Myasthenia Gravis Activities of Daily Living (MG-ADL) total score: absolute value and change from baseline appropriate for pediatric use • Quantitative Myasthenia Gravis (QMG): absolute value and change from baseline ", EQ-5D-Y: absolute value and change from baseline • Quality of Life in Neurological Disorders Questionnaire (Neuro-QoL) Pediatric Fatigue Score: change from baseline • Clinical Global Impression of Improvement (CGI-I): change from baseline • Changes in protective antibody titers to vaccines	—

Measure

Time frame

Incidence and severity of adverse events (AEs), including serious AEs and AEs of special interest • Changes in vital signs, electrocardiogram (ECGs), and clinical laboratory tests • Efgartigimod serum concentrations • Total IgG and AChR-Ab levels: absolute values, changes from baseline values, and percent (%) reductions from baseline values", Prevalence and incidence of antidrug antibodies (ADA) against efgartigimod and antibodies against rHuPH20 • Myasthenia Gravis Activities of Daily Living (MG-ADL) total score: absolute value and change from baseline appropriate for pediatric use • Quantitative Myasthenia Gravis (QMG): absolute value and change from baseline ", EQ-5D-Y: absolute value and change from baseline • Quality of Life in Neurological Disorders Questionnaire (Neuro-QoL) Pediatric Fatigue Score: change from baseline • Clinical Global Impression of Improvement (CGI-I): change from baseline • Changes in protective antibody titers to vaccines

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Countries

Belgium, Czechia, France, Germany, Italy, Netherlands, Poland, Spain

Outcome results

None listed