A Phase 2 Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children From 2 Years to Less Than 18 Years of Age With Generalized Myasthenia Gravis (gMG)
Summary
The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.
Arms & interventions
- DrugInebilizumab
Inebilizumab will be administered IV.
Outcome measures
Primary
Maximum Observed Concentration (Cmax) of Inebilizumab
Time frame: Up to Week 52
Area Under the Concentration-time Curve (AUC) of Inebilizumab
Time frame: Up to Week 52
Half-life (t1/2) of Inebilizumab
Time frame: Up to Week 52
Clearance (CL) of Inebilizumab
Time frame: Up to Week 52
Volume of Distribution at Steady State (Vss) of Inebilizumab
Time frame: Up to Week 52
Change from Baseline in Cluster of Differentiation 20 (CD20)+ B-cell Counts
Time frame: Baseline and Week 78
Number of Participants Experiencing Treatment-emergent Adverse Events
Time frame: Up to Week 78
Number of Participants Experiencing Clinically Significant Changes in Laboratory Parameters
Time frame: Up to Week 78
Number of Participants Experiencing Clinically Significant Changes in Vital Signs
Time frame: Up to Week 78
Secondary
Change from Baseline in Quantitative Myasthenia Gravis (QMG) Score
Time frame: Baseline and Week 78
Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score
Time frame: Baseline and Week 78
Change from Baseline in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) Score
Time frame: Baseline and Week 78
Change from Baseline in Neurological Quality of Life (Neuro-QoL) Pediatric Fatigue Score
Time frame: Baseline and Week 78
Number of Participants with Anti-drug Antibodies (ADAs) Present
Time frame: Up to Week 78
Eligibility criteria
Study locations (1)
Austin Neuromuscular Center
Austin, Texas, 78759