The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Ravulizumab will be administered by intravenous (IV) infusion.
Research Site
Los Angeles, California, United States
Research Site
Boston, Massachusetts, United States
Research Site
Chapel Hill, North Carolina, United States
Research Site
Philadelphia, Pennsylvania, United States
Serum Concentration of Ravulizumab
Time frame: Day 1 predose through Week 18 predose
Serum Free C5 Concentration
Time frame: Day 1 predose through Week 18 predose
Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18
Time frame: Week 10, Up to Week 18
Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18
Participants ≥8 years of age will be evaluated.
Time frame: Baseline, Up to Week 18
Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18
Participants \<8 years of age will be evaluated.
Time frame: Baseline, Up to Week 18
Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Research Site
Milan, Italy
Research Site
Roma, Italy
Research Site
Torino, Italy
Research Site
Itabashi-ku, Japan
Research Site
Belgrade, Serbia
Research Site
Belgrade, Serbia
...and 1 more locations
Time frame: Baseline through Week 18
Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18
Time frame: Baseline through Week 18
Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline
Stable is defined as a ±5-point change from Baseline.
Time frame: Baseline through Week 18
Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline
Stable is defined as a ±3-point change from baseline.
Time frame: Baseline through Week 18
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Time frame: Baseline up to Week 126 (8 weeks after last dose of study drug)
Number of Participants With Anti-Drug Antibody (ADA) at Week 18
Time frame: Baseline through Week 18