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Study of Inebilizumab for Children and Adolescents with Neuromyelitis Optica Spectrum Disorder

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Neuromyelitis Optica Spectrum Disorder (NMOSD), which is also referred to as Devic's syndrome. NMOSD is a rare disease that affects the central nervous system, particularly the optic nerves and spinal cord, leading to symptoms such as vision loss and muscle weakness. The study involves a treatment using a medication called Inebilizumab, which is a type of drug known as a monoclonal antibody. Monoclonal antibodies are proteins designed to target specific cells in the body, and in this case, Inebilizumab is being tested for its effects on NMOSD in children and adolescents.

The purpose of this study is to understand how Inebilizumab behaves in the body, its effects on the disease, and its safety for young patients with NMOSD. Participants in the study will receive Inebilizumab through a direct injection into a vein. The study will monitor how the drug is processed by the body, its impact on the disease, and any side effects that may occur. This will help researchers determine the best way to use Inebilizumab for treating NMOSD in younger patients.

Throughout the study, participants will have regular check-ups to assess their health and the effectiveness of the treatment. These check-ups will include measuring the levels of certain cells in the blood and evaluating any changes in symptoms or overall health. The study aims to provide valuable information that could lead to better treatment options for children and adolescents living with NMOSD.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    The study begins with an initial assessment to confirm eligibility. This includes a review of medical history and a blood test to check for a positive serum anti-AQP4-IgG result, which is necessary for participation.

  2. Step 2

    First dose administration

    The first dose of inebilizumab is administered through a direct intravenous injection. This is done under medical supervision to monitor any immediate reactions.

  3. Step 3

    Follow-up visits

    Follow-up visits are scheduled on Days 1, 8, 15, 29, 57, 85, 113, 155, and 197. During these visits, blood samples are taken to measure CD20-positive B-cell counts and other parameters.

    These visits also include safety assessments to monitor for any adverse events or changes in health status.

  4. Step 4

    Ongoing monitoring

    Throughout the study, regular monitoring is conducted to assess the safety and effectiveness of the treatment. This includes tracking any relapses of the condition and changes in quality of life and visual acuity.

  5. Step 5

    Final assessment

    At the end of the study period, a final assessment is conducted. This includes a comprehensive review of the treatment's impact on the condition and any long-term effects.

Who can join the trial?

6 criteria

  • The patient's legally authorized representative must provide written informed consent, and the patient must agree to participate if they are able to understand.
  • The patient must be a boy or girl, weigh at least 15 kg, and be between 2 and less than 18 years old at the time of screening.
  • The patient must have a positive result for a specific antibody called **anti-AQP4-IgG** during screening and be diagnosed with **neuromyelitis optica spectrum disorder (NMOSD)**.
  • The patient must have a documented history of one or more **NMOSD acute relapses** (sudden worsening of symptoms) within the last year, or two or more relapses within the last two years before screening.
  • Female patients who can have children and are sexually active with a male partner who has not been sterilized must agree to use a highly effective method of birth control from screening until six months after the last dose of the study medication.
  • Male patients who have not been sterilized and are sexually active with a female partner who can have children must agree to use a male condom from the first day of the study until three months after the last dose of the study medication.

Who cannot join the trial?

8 criteria

  • Patients who do not have **Neuromyelitis Optica Spectrum Disorder (NMOSD)**, which is a condition affecting the nerves of the eyes and spinal cord, cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not willing to follow the study procedures and requirements cannot participate.
  • Patients with other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently may not be eligible.
  • Patients who have allergies or reactions to the study medication cannot participate.
  • Patients who are unable to provide consent or do not have a legal guardian to provide consent cannot participate.
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Investigated drugs

Inebilizumab is a medication being studied for its effects on children with Neuromyelitis Optica Spectrum Disorder (NMOSD). This study aims to understand how the body processes the medication (pharmacokinetics) and how the medication affects the body (pharmacodynamics). Additionally, the study evaluates the safety and how well children tolerate the medication.

What is already known about the treatment

Inebilizumab – Inebilizumab is administered as an intravenous infusion, typically given in a clinical setting under the supervision of healthcare professionals. It is currently being studied in clinical trials for its effectiveness and safety in treating neuromyelitis optica spectrum disorder (NMOSD), particularly in pediatric patients. The medication works by targeting and depleting B cells, a type of immune cell involved in the inflammatory process of NMOSD, thereby reducing disease activity. It is classified as a monoclonal antibody, which is a type of biologic therapy designed to specifically target certain cells in the immune system.

Investigated diseases

Neuromyelitis optica spectrum disorder – This is a rare autoimmune disease that primarily affects the optic nerves and spinal cord. It leads to inflammation and damage in these areas, causing symptoms such as vision loss, weakness, and paralysis. The disease can progress with episodes of relapses, where symptoms worsen, followed by periods of remission. Unlike multiple sclerosis, which it can resemble, neuromyelitis optica spectrum disorder typically involves more severe attacks and less recovery between episodes. The condition can also affect other parts of the central nervous system, leading to a variety of neurological symptoms. It is important to monitor the disease closely to understand its progression and impact on the individual.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2023-510007-22-00Protocol codeVIB0551.P2.S2.NMOEstimated enrolment15 patientsSponsorHorizon Therapeutics Ireland Designated Activity Company

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