NCT05549258 · Amgen
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
What this study is about
A Phase 2, where both patients and doctors know the treatment given, conducted at multiple hospitals study to evaluate the how the drug moves through the body (PK), how the drug affects the body (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).
View original scientific description
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).
Interventions
DRUG
Inebilizumab
Inebilizumab administered intravenously (IV) over a total of 28 weeks.
Primary outcome measures
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
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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 criteria
- 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 resu
Where
- La Jolla, California
- Loma Linda, California
- Boston, Massachusetts
- Dallas, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 5, 2025 · Source of record for eligibility and locations