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New hope for kids with rare nerve disease: drug trial launches

NCT ID NCT05549258

Disease control Sponsor: Amgen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called inebilizumab in 15 children aged 2 to 17 with neuromyelitis optica spectrum disorder (NMOSD), a rare condition where the immune system attacks the optic nerves and spinal cord. The drug aims to reduce relapses by targeting certain immune cells. Researchers will measure how the drug moves through the body, its safety, and whether it helps control the disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
inebilizumab
What this could lead to
If successful, this trial could show that inebilizumab is a safe and effective option to reduce relapses in children with NMOSD.
What could go wrong
This is a small, early-phase study with only 15 participants, so results may not apply to all patients. Side effects are possible, and the drug may not work as well in children as in adults.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2023

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 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: 1. Alemtuzumab 2. Total lymphoid irradiation 3. Bone marrow transplant 4. 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: 1. Cyclosporine 2. Methotrexate 3. Mitoxantrone 4. Cyclophosphamide 5. Tocilizumab 6. Satralizumab 7. 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: 1. 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). 2. Bacillus Calmette Guérin vaccine within one year of screening. 3. 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: a. 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: 1. 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 2. Unable to tolerate or comply with the MRI procedure.

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Conditions

The condition(s) this trial relates to.

neuromyelitis optica

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Neuromyelitis optica spectrum disorder

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    19 sites in 11 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Birmingham Women's and Children's NHS Foundation Trust

    RECRUITING

    Birmingham, West Midlands, B4 6NH, United Kingdom

  • CPQuali Pesquisa Clínica Sao Paulo

    RECRUITING

    São Paulo, 01228-000, Brazil

  • Centre Hospitalier Universitaire de Bicêtre

    RECRUITING

    Le Kremlin-Bicêtre, Val-de-Marne, 94275, France

  • Clinic for Neurology and Psychiatry for Children and Youth

    RECRUITING

    Belgrade, Belgrade, 11000, Serbia

  • Erasmus MC Sophia Children's Hospital-Wytemaweg 80

    RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Evelina London Children's Hospital

    RECRUITING

    London, London, City of, SE1 7EH, United Kingdom

  • Great Ormond Street Hospital - PPDS

    RECRUITING

    London, London, City of, WC1N 3JH, United Kingdom

  • Hospital For Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • Hospital Sant Joan de Deu - PIN

    RECRUITING

    Espluges de Llobregat, Barcelona, 08950, Spain

  • Hospital Santa Izabel-Rua Floriano Peixoto 300

    RECRUITING

    Salvador, Estado de Bahia, 40050-410, Brazil

  • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)

    RECRUITING

    Porto Alegre/RS, 90610-000, Brazil

  • Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

    RECRUITING

    São Paulo, 05403-000, Brazil

  • Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

    RECRUITING

    Parque Patricios, Ciudad Autónoma de BuenosAires, C1245AAM, Argentina

  • Karolinska Universitetssjukhuset Solna

    RECRUITING

    Stockholm, Stockholm County, 17176, Sweden

  • Loma Linda University Children's Hospital - PIN

    RECRUITING

    Loma Linda, California, 92354, United States

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • UCSD Altman Clinical and Translational Research Institute Building

    RECRUITING

    La Jolla, California, 92037-1337, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 78701, United States

  • Uniwersyteckie Centrum Kliniczne w Gdansku - Smoluchowskiego 17

    RECRUITING

    Gdansk, 80-952, Poland

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