Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age2-17
SponsorAmgen
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female participants, minimum body weight of 15 kg, age 2 to < 18 years at the time of screening.
Positive serum anti-AQP4-IgG result at screening and diagnosed with NMOSD according to the criteria of Wingerchuk et al, 2015.
Documented history of one or more NMOSD acute relapses within the last year, or 2 or more NMOSD acute relapses within 2 years prior to screening.
Disqualifiers
Any condition that, in the opinion of the Investigator, would interfere with the evaluation or administration of the Investigational Product or interpretation of participant safety or study results.
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.
Evidence of significant hepatic, renal, or metabolic dysfunction or significant hematological abnormality (one repeat test may be conducted to confirm results within the same screening period).
B-cell counts < one-half of the lower limit of normal (LLN) for age according to the central laboratory.
Trial design
Design model
Single group
Treatments tested in this trial
Inebilizumab
DrugInebilizumab administered intravenously (IV) over a total of 28 weeks.
Treatment groups
15 Participants
are divided into 1 treatment groupGroup A: InebilizumabExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Maximum Observed Concentration (Cmax) of Inebilizumab
Time frame
Day 1 to Week 282
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 153
Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 Extrapolated to Infinity (AUC0-Inf)
Time frame
Day 1 to Week 804
Systemic Clearance (CL) of Inebilizumab
Time frame
Day 1 to Week 80Secondary outcomes
1
Disease Activity: Time to First Relapse
Time frame
Day 1 to Week 802
Disease Activity: Proportion of Relapse-free Participants
Time frame
Day 1 to Week 803
Disease Activity: Annualized Relapse Rate
Time frame
Day 1 to Week 804
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 Source ClinicalTrials.gov