Neuromyelitis optica spectrum disorder (NMOSD
Conditions
Sponsors
Horizon Therapeutics Ireland DAC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent and any locally required data privacy authorization obtained from the subject’s legally authorized representative in accordance with regional laws or regulations and the subject’s assent, when applicable, prior to performing any protocol-related procedures. 2. Male or female subjects, minimum body weight of 15 kg, age 2 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the IP or interpretation of subject safety or study results 2. Concurrent/previous enrollment in another clinical study involving an investigational treatment within 4 weeks or 5 published half-lives of the investigational treatment, whichever is the longer, prior to Day 1 3. Females who are breastfeeding, pregnant, or who intend to become pregnant at any time from screening until 6 months after the final dose of IP 4. Known history of allergy or reaction to any component of the IP formulation or history of anaphylaxis following any biologic therapy 5. Evidence of alcohol, drug, or chemical abuse, or a recent history of such abuse 3 months prior to Day 1 22. History of active or latent tuberculosis 23. For subjects who may undergo MRI scans: Unable to undergo an MRI scan (eg, hypersensitivity to Gd containing MRI contrast agents, implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans), or unable to tolerate or comply with the MRI procedure.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To characterize the PK of inebilizumab administered in pediatric subjects with NMOSD 2. To characterize the PD of inebilizumab administered in pediatric subjects with NMOSD 3. To assess the safety and tolerability of inebilizumab administered in pediatric subjects with NMOSD;Secondary Objective: 1. To assess disease activity when inebilizumab is administered in pediatric subjects with NMOSD 2. To assess health-related quality of life (HRQoL) when inebilizumab is administered in pediatric subjects with NMOSD 3. To assess visual acuity when inebilizumab is administered in pediatric subjects with NMOSD 4. To assess disability when inebilizumab is administered in pediatric subjects with NMOSD 5. To characterize the immunogenicity of inebilizumab administered in pediatric subjects with NMOSD ;Primary end point(s): 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 ;Timepoint(s) of evaluation of this end point: From randomization until end of the trial | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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) ;Timepoint(s) of evaluation of this end point: From randomization until end of the trial | — |
Countries
Argentina, Brazil, Canada, France, Netherlands, Poland, Serbia, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactAlan Mac Neice
Horizon Therapeutics Ireland DAC
Outcome results
None listed