Neuromyelitis Optica Spectrum DisorderPhase 2NCT05549258
Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
A Phase 2 study testing Inebilizumab in people with Neuromyelitis Optica Spectrum Disorder, run by Amgen at 19 sites in 11 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- Inebilizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 19 sitesin 11 countries
- Study dates
- 3 Jul 2023to 13 Apr 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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:
- Alemtuzumab
Show all 38Show fewer
- Total lymphoid irradiation
- Bone marrow transplant
- T-cell vaccination therapy
- 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:
- Cyclosporine
- Methotrexate
- Mitoxantrone
- Cyclophosphamide
- Tocilizumab
- Satralizumab
- Eculizumab
- 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.
- Receipt of any of the following:
- Any live or attenuated vaccine within 4 weeks prior to Day 1 (administration of killed vaccines and nucleoside-modified mRNA-based vaccines is acceptable; the Sponsor recommends that Investigators ensure all participants are up to date on required vaccinations prior to study entry).
- Bacillus Calmette Guérin vaccine within one year of screening.
- Blood transfusion within 4 weeks prior to screening or during screening.
- 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 hepatitis B surface antigen (HBsAg), or b. Positive hepatitis B core (HBc) antibody (anti-HBc) plus negative hepatitis B surface (HBs) antibody (anti-HBs).
- 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:
- Unable to undergo an MRI scan (e.g., 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.
Trial locations and contacts
19 sites in 11 countries. Contact the study team or a site near you to ask about taking part.
19 sites in 11 countries
United States4 sites, 4 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02115
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 78701
- Recruiting
UCSD Altman Clinical and Translational Research Institute Building
La Jolla, California, 92037-1337
- Recruiting
Loma Linda University Children's Hospital - PIN
Loma Linda, California, 92354
Brazil4 sites, 4 recruiting
- Recruiting
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
Porto Alegre/RS, 90610-000
- Recruiting
Hospital Santa Izabel-Rua Floriano Peixoto 300
Salvador, Estado de Bahia, 40050-410
- Recruiting
CPQuali Pesquisa Clínica Sao Paulo
São Paulo, 01228-000
- Recruiting
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, 05403-000
United Kingdom3 sites, 3 recruiting
- Recruiting
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, West Midlands, B4 6NH
- Recruiting
Evelina London Children's Hospital
London, London, City of, SE1 7EH
- Recruiting
Great Ormond Street Hospital - PPDS
London, London, City of, WC1N 3JH
Argentina1 site, 1 recruiting
- Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Parque Patricios, Ciudad Autónoma de BuenosAires, C1245AAM
Canada1 site, 1 recruiting
- Recruiting
Hospital For Sick Children
Toronto, Ontario, M5G 1X8
France1 site, 1 recruiting
- Recruiting
Centre Hospitalier Universitaire de Bicêtre
Le Kremlin-Bicêtre, Val-de-Marne, 94275
Netherlands1 site, 1 recruiting
- Recruiting
Erasmus MC Sophia Children's Hospital-Wytemaweg 80
Rotterdam, South Holland, 3015 GD
Poland1 site, 1 recruiting
- Recruiting
Uniwersyteckie Centrum Kliniczne w Gdansku - Smoluchowskiego 17
Gdansk, 80-952
Serbia1 site, 1 recruiting
- Recruiting
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade, Belgrade, 11000
Spain1 site, 1 recruiting
- Recruiting
Hospital Sant Joan de Deu - PIN
Espluges de Llobregat, Barcelona, 08950
Sweden1 site, 1 recruiting
- Recruiting
Karolinska Universitetssjukhuset Solna
Stockholm, Stockholm County, 17176
About this study
Official title: An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
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).
Read the detailed description
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.
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Being tested
- Inebilizumab
- Keywords
- NMOSD
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InebilizumabDrug
Inebilizumab administered intravenously (IV) over a total of 28 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Inebilizumab | Experimental | Infusion of Inebilizumab Receives: Drug: Inebilizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Observed Concentration (Cmax) of Inebilizumab
Measured: Day 1 to Week 28
Area Under the Concentration Versus Time Curve of Inebilizumab from Time 0 to 14 Days Post-dose (AUC0-14d)
Measured: 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)
Measured: Day 1 to Week 80
Systemic Clearance (CL) of Inebilizumab
Measured: Day 1 to Week 80
Terminal Elimination Half-life (t½) of Inebilizumab
Measured: Day 1 to Week 80
Volume of Distribution at Steady State (VSS) of Inebilizumab
Measured: Day 1 to Week 80
Change from Baseline in Peripheral Cluster of Differentiation (CD)20-positive B-cell Counts
Measured: 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)
Measured: Day 1 to Week 80
Change from Baseline in Serum Chemistry
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Hematology
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Serum Immunoglobulins
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Systolic Blood Pressure
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Diastolic Blood Pressure
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Pulse Rate
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Respiratory Rate
Measured: Week 1, Week 2, Week 28, Week 80
Change from Baseline in Body Temperature
Measured: Week 1, Week 2, Week 28, Week 80
Secondary outcomes (8)
Disease Activity: Time to First Relapse
Measured: Day 1 to Week 80
Disease Activity: Proportion of Relapse-free Participants
Measured: Day 1 to Week 80
Disease Activity: Annualized Relapse Rate
Measured: 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.
Measured: 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.
Measured: Day 1 to Week 80
Visual Acuity change from baseline
Measured: 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.
Measured: Day 1 to Week 80
Anti-drug antibody (ADA) rate
Measured: Day 1 to Week 80
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2022
- Study start3 Jul 2023
- Primary completion13 Apr 2027 (estimated)
- Study completion13 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05549258
- Sponsor's protocol ID
- VIB0551.P2.S2.NMO
- Other IDs
- 2023-510007-22, 2023
- Status last verified
- Dec 2025
- First submitted
- 22 Jul 2022
- Last updated
- 5 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder
- ClinicalTrials.gov ID
- NCT05549258
- Acronym / sponsor ID
- VIB0551.P2.S2.NMO
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Keywords
- NMOSD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 3 Jul 2023 (actual)
- Primary completion
- 13 Apr 2027 (estimated)
- Study completion
- 13 Apr 2027 (estimated)
- First posted
- 22 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Inebilizumab | Experimental | Drug: InebilizumabInfusion of Inebilizumab |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (38 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InebilizumabInebilizumab administered intravenously (IV) over a total of 28 weeks. | Drug | Inebilizumab |
Primary outcome
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
8 secondary outcomes
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
Time frame: Day 1 to Week 80
HRQoL change from baseline in Pediatric Quality of Life Inventory
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
Time frame: Day 1 to Week 80
Anti-drug antibody (ADA) rate
Time frame: Day 1 to Week 80
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2025.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
MD study director
Amgen
19 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Inebilizumab in people with Neuromyelitis Optica Spectrum Disorder, run by Amgen at 19 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 38 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Brazil, United Kingdom, Argentina, Canada and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Amgen Call Center (866-572-6436, medinfo@amgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Observed Concentration (Cmax) of Inebilizumab (time frame: Day 1 to Week 28). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2025.