A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to \< 18 years of age with recently active neuromyelitis optica spectrum disorder (NMOSD) who are seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin \[Ig\]G).
Approximately 15 participants to be enrolled and receive Inebilizumab administered intravenously over 28 weeks. The maximum trial duration per participant is approximately 80 weeks, including up to 4 week screening period, 9 visits during a 28 week open-label treatment period, and approximately 4 visits during a 52 week follow-up period. Safety evaluations will be performed regularly throughout the course of the study. Acquired from Horizon in 2023.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Inebilizumab administered intravenously (IV) over a total of 28 weeks.
UCSD Altman Clinical and Translational Research Institute Building
La Jolla, California, United States
Maximum Observed Concentration (Cmax) of Inebilizumab
Time frame: Day 1 to Week 28
Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 to 14 Days Post-dose (AUC0-14d)
Time frame: Day 1 to pre-dose on Day 15
Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 Extrapolated to Infinity (AUC0-Inf)
Time frame: Day 1 to Week 80
Systemic Clearance (CL) of Inebilizumab
Time frame: Day 1 to Week 80
Terminal Elimination Half-life (t½) of Inebilizumab
Time frame: Day 1 to Week 80
Volume of Distribution at Steady State (VSS) of Inebilizumab
Time frame: Day 1 to Week 80
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell Counts
Time frame: Week 1, Week 2, Week 28, Week 80
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs)
Time frame: Day 1 to Week 80
Change from Baseline in Serum Chemistry
Time frame: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Hematology
Time frame: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Serum Immunoglobulins
Time frame: Week 1, Week 2, Week 28, Week 80
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Loma Linda University Children's Hospital - PIN
Loma Linda, California, United States
RECRUITINGMassachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGUniversity of Texas Southwestern Medical Center
Dallas, Texas, United States
RECRUITINGHospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Parque Patricios, Ciudad Autónoma de BuenosAires, Argentina
RECRUITINGHospital Santa Izabel-Rua Floriano Peixoto 300
Salvador, Estado de Bahia, Brazil
RECRUITINGHospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
Porto Alegre/RS, Brazil
RECRUITINGCPQuali Pesquisa Clínica Sao Paulo
São Paulo, Brazil
RECRUITINGHospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, Brazil
RECRUITINGHospital For Sick Children
Toronto, Ontario, Canada
RECRUITING...and 9 more locations
Change from Baseline in Systolic Blood Pressure
Time frame: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Diastolic Blood Pressure
Time frame: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Pulse Rate
Time frame: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Respiratory Rate
Time frame: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Body Temperature
Time frame: Week 1, Week 2, Week 28, Week 80
Disease Activity: Time to First Relapse
Time frame: Day 1 to Week 80
Disease Activity: Proportion of Relapse-free Participants
Time frame: Day 1 to Week 80
Disease Activity: Annualized Relapse Rate
Time frame: Day 1 to Week 80
Health-Related Quality of Life (HRQoL) change from baseline in Euro Quality of Life-5 Dimension Youth score
Change in baseline for the 5 dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. A higher score indicates onset or worsening of an affective disorder.
Time frame: Day 1 to Week 80
HRQoL change from baseline in Pediatric Quality of Life Inventory
Change in baseline comprised from 4 generic core scales: Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. A higher score indicates a better quality of life.
Time frame: Day 1 to Week 80
Visual Acuity change from baseline
Time frame: Day 1 to Week 80
Change From Baseline in Expanded Disability Status Scale
Change in baseline comprised from results of 8 Functional Systems: Visual, Brainstem, Pryamidal, Cerebellar, Sensory, Bowel, Bladder, and Cerebral. A higher score indicates a higher grade of impairment and disability.
Time frame: Day 1 to Week 80
Anti-drug antibody (ADA) rate
Time frame: Day 1 to Week 80