Neuromyelitis optica spectrum disorder (NMOSD; also known as Devic's syndrome and previously known as neuromyelitis optica [NMO])
Conditions
Brief summary
1. PK parameters, including maximum observed concentration (Cmax), area under the concentration-time curve from time 0 to 14 days post dose (AUC0-14d) and from time 0 extrapolated to infinity (AUC0-inf), systemic clearance, terminal elimination half-life (t½), and volume of distribution at steady state (Vss), 2. CD20-positive B-cell counts on Days 1, 8, 15, 29, 57, 85, 113, 155, and 197, 3. Safety and tolerability assessments, including incidence of adverse events (AEs)/serious adverse events (SAEs)/adverse events of special interest (AESIs) and changes in laboratory parameters and vital signs
Detailed description
1. Disease activity endpoints include: − Time to first relapse − Proportion of relapse-free subjects − Annualized relapse rate, 2. HRQoL endpoints include: − Change in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) score − Change in Pediatric Quality of Life Inventory (PedsQL), 3. Change in visual acuity, 4. Change in EDSS, 5. Presence of anti-drug antibody (ADA)
Interventions
DRUGINEBILIZUMAB
Sponsors
Horizon Therapeutics Ireland Designated Activity Company
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. PK parameters, including maximum observed concentration (Cmax), area under the concentration-time curve from time 0 to 14 days post dose (AUC0-14d) and from time 0 extrapolated to infinity (AUC0-inf), systemic clearance, terminal elimination half-life (t½), and volume of distribution at steady state (Vss), 2. CD20-positive B-cell counts on Days 1, 8, 15, 29, 57, 85, 113, 155, and 197, 3. Safety and tolerability assessments, including incidence of adverse events (AEs)/serious adverse events (SAEs)/adverse events of special interest (AESIs) and changes in laboratory parameters and vital signs | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Disease activity endpoints include: − Time to first relapse − Proportion of relapse-free subjects − Annualized relapse rate, 2. HRQoL endpoints include: − Change in Euro Quality of Life-5 Dimension Youth (EQ-5D-Y) score − Change in Pediatric Quality of Life Inventory (PedsQL), 3. Change in visual acuity, 4. Change in EDSS, 5. Presence of anti-drug antibody (ADA) | — |
Countries
France, Netherlands, Poland, Spain, Sweden
Outcome results
None listed