Inebilibumab for Pediatric Neuromyelitis Optica Spectrum Disorder
This study is looking at a drug called inebilizumab for children and teenagers (ages 2 to 17) who have neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, lifelong autoimmune disease that affects the central nervous system. Participants must have a specific antibody (anti-AQP4-IgG) in their blood and a history of recent NMOSD attacks. The main goal is to understand how the body handles inebilizumab (pharmacokinetics) and to check its safety. About 15 participants will receive inebilizumab intravenously (through a vein) over 28 weeks. The study will measure the drug levels in the body at different times to see how it's absorbed and cleared.
- Study design
- This is an open-label study, meaning everyone knows what treatment is being given. It aims to enroll about 15 participants.
- What's involved
- You would have 9 visits during a 28-week treatment period, and about 4 visits during a 52-week follow-up period. The total time in the study could be up to 80 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for approximately 52 weeks after the 28-week treatment period, with safety evaluations continuing throughout.
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NCT05549258
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
Recruiting
PHASE2Ages 2–17InterventionalTreatmentAmgen
~15 participants
Updated 2025-12-05 on ClinicalTrials.gov
What's tested:Inebilizumab
At a glance
Recruiting sites
19 of 19 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Maximum Observed Concentration (Cmax) of Inebilizumab
Measured over Day 1 to Week 28
+15 more outcomes measured
Conditions
Where it's being run
19 sites across 15 statesBrazil3
California2
London, City of2
Massachusetts1
Texas1
Ciudad Autónoma de BuenosAires1
Estado de Bahia1
Ontario1
Study leadership
- MD · STUDY_DIRECTOR · Amgen
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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.
Exclusion
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.
Receipt of the following at any time prior to Day 1:
Receipt of rituximab or any experimental B-cell depleting agent within 6 months prior to screening unless B-cell counts have returned to ≥ one-half the LLN.
Receipt of intravenous immunoglobulin (IVIG) within one month prior to Day 1.
Receipt of any of the following within 2 months prior to Day 1:
Receipt of natalizumab (Tysabri®) within 6 months prior to Day 1.
Severe drug allergic history or anaphylaxis to 2 or more food products or medicine (including known sensitivity to acetaminophen/paracetamol, diphenhydramine or equivalent antihistamine, and methylprednisolone or equivalent glucocorticoid).
Diagnosed with a concurrent autoimmune disease that is uncontrolled (unless approved by the medical monitor).
Recent receipt of live/attenuated vaccine or blood transfusion.
Clinically significant serious active or chronic viral, bacterial, or fungal infection that requires treatment with anti-infectives, hospitalization, or, in the Investigator's opinion, represents an additional risk to the participant, within 2 months prior to Day 1.
Known history of congenital or acquired immunodeficiency (e.g., due to human immunodeficiency virus \[HIV\] infection, splenectomy, immunosuppression-related or idiopathic T-cell deficiencies) that predisposes the participant to infection.
Positive test for chronic hepatitis B infection at screening, defined as either:
Positive test for hepatitis C virus antibody.
Negative test for varicella zoster virus (VZV)-IgG.
History of cancer, apart from squamous cell or basal cell carcinoma of the skin treated with documented success of curative therapy \> 3 months prior to Day 1.
History of active or latent tuberculosis (TB), or a positive QuantiFERON®-TB Gold test at screening, unless treatment for TB was completed per local guidelines. Participants with latent TB or a positive QuantiFERON®-TB Gold test who are actively on anti-TB treatment can enroll if they have completed at least one month of anti-TB treatment and intend to complete the full course of anti-TB treatment. Participants with an indeterminate QuantiFERON®-TB Gold test result can enroll if a repeat QuantiFERON®-TB Gold test is negative or a tuberculin skin test is negative.
For participants who may undergo MRI scans:
What this trial measures
- Maximum Observed Concentration (Cmax) of InebilizumabDay 1 to Week 28
- Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 to 14 Days Post-dose (AUC0-14d)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)Day 1 to Week 80
- Systemic Clearance (CL) of InebilizumabDay 1 to Week 80
- Terminal Elimination Half-life (t½) of InebilizumabDay 1 to Week 80
- Volume of Distribution at Steady State (VSS) of InebilizumabDay 1 to Week 80
- Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell CountsWeek 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)Day 1 to Week 80
- Change from Baseline in Serum ChemistryWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in HematologyWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in Serum ImmunoglobulinsWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in Systolic Blood PressureWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in Diastolic Blood PressureWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in Pulse RateWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in Respiratory RateWeek 1, Week 2, Week 28, Week 80
- Change from Baseline in Body TemperatureWeek 1, Week 2, Week 28, Week 80