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Study on the Effectiveness and Safety of Ravulizumab for Children and Adolescents with Neuromyelitis Optica Spectrum Disorder (NMOSD)

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 called Neuromyelitis Optica Spectrum Disorder (NMOSD), which affects the nervous system, particularly the eyes and spinal cord. The study is testing a treatment called Ravulizumab, also known by its code name ALXN1210. Ravulizumab is given as an infusion, which means it is administered directly into the bloodstream through a vein. The purpose of the study is to evaluate how effective and safe Ravulizumab is for children and adolescents who have NMOSD.

Participants in the study will receive Ravulizumab and will be monitored over a period of time to see how the treatment affects their condition. The study will look at various aspects, such as the frequency of relapses, which are episodes when symptoms worsen, and changes in physical abilities and vision. The study will also assess the overall health and quality of life of the participants. Safety is a key focus, and any side effects or changes in health will be closely observed.

This study is designed to provide important information about the use of Ravulizumab in young patients with NMOSD, aiming to improve understanding of how this treatment can help manage the disease. Participants will be part of a group that receives the treatment, and their progress will be compared to historical data from similar patients who have not received Ravulizumab. The study is expected to continue until 2028, allowing researchers to gather comprehensive data on the treatment's long-term effects.

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 trial begins with an initial assessment to confirm eligibility. Participants must be between 2 and 18 years old, have a diagnosis of Neuromyelitis Optica Spectrum Disorder (NMOSD), and be positive for aquaporin-4 antibodies.

    Participants must have experienced at least one attack or relapse in the past year if they have not previously received complement inhibitor treatment. Those who have been treated with eculizumab must be stable and have been on the treatment for at least 90 days prior to the trial.

  2. Step 2

    Vaccination requirements

    Participants must be vaccinated against meningococcal infection from serogroups A, C, W 135, and Y within three years prior to, or at least 14 days before the first day of the trial. If this requirement is not met, vaccination must occur according to guidelines, and prophylactic antibiotics will be administered for at least two weeks if the trial starts less than two weeks after vaccination.

    Vaccination against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae must also be documented at least 14 days before the trial begins.

  3. Step 3

    Treatment administration

    The treatment involves the administration of ravulizumab, a medication given through an intravenous infusion. The specific dosage and frequency will be determined by the study protocol and the healthcare team overseeing the trial.

  4. Step 4

    Monitoring and assessments

    Throughout the trial, regular monitoring and assessments will be conducted to evaluate the effectiveness and safety of the treatment. This includes measuring changes in the annualized relapse rate, time to first relapse, and other health indicators.

    Additional assessments will include changes in disability status, visual acuity, and quality of life measures. Blood tests will be performed to monitor serum ravulizumab concentrations and other relevant parameters.

  5. Step 5

    Extension period

    An extension period may follow the primary treatment phase, during which the same endpoints will be evaluated to gather further data on the long-term effects and safety of the treatment.

Who can join the trial?

8 criteria

  • Participants must be between 2 and 18 years old at the time of signing the consent form.
  • Participants must test positive for **anti-AQP4 antibodies** and have a diagnosis of **Neuromyelitis Optica Spectrum Disorder (NMOSD)** according to the 2015 international guidelines. **Anti-AQP4 antibodies** are proteins in the blood that are often found in people with NMOSD.
  • Participants who have not been treated with a **complement inhibitor** must have had at least one attack or relapse in the last 12 months before the study starts. A **complement inhibitor** is a type of medication that helps control the immune system.
  • Participants must have an **Expanded Disability Status Scale (EDSS)** score of 7 or less. The **EDSS** is a scale used to measure disability in people with multiple sclerosis and similar conditions.
  • Participants who have experience with **eculizumab** must be stable according to the study doctor for 30 days and have been treated with **eculizumab** for at least 90 days before the study starts, with no missed doses in the 2 months before the first day of the study. **Eculizumab** is a medication used to treat NMOSD.
  • Participants taking supportive **immunosuppressive therapy (IST)**, such as corticosteroids or other medications, must be on a stable dose for a sufficient time before the study and continue on a stable dose during the study's initial period. **Immunosuppressive therapy** helps prevent the immune system from attacking the body.
  • To lower the risk of **meningococcal infection**, all participants must be vaccinated against this infection from specific groups (A, C, W 135, and Y) within 3 years before or at least 14 days before the first day of the study, following local guidelines. If not vaccinated, participants will receive the vaccine and preventive antibiotics for at least 2 weeks if the first day of the study is less than 2 weeks after vaccination. **Meningococcal infection** is a serious bacterial infection that can affect the brain and spinal cord.
  • Participants must have documented vaccinations for **Haemophilus influenzae type b (Hib)** and **Streptococcus pneumoniae** at least 14 days before the first day of the study, according to local guidelines for their age group. These vaccinations help protect against certain bacterial infections.

Who cannot join the trial?

10 criteria

  • Participants who have any other serious health condition that could interfere with the study.
  • Participants who are currently taking medications that might affect the study results.
  • Participants who have had a recent infection or illness that could impact their health during the study.
  • Participants who have a history of allergic reactions to similar medications.
  • Participants who are unable to follow the study procedures or attend scheduled visits.
  • Participants who are pregnant or breastfeeding.
  • Participants who have participated in another clinical trial recently.
  • Participants who have a history of substance abuse or addiction.
  • Participants who have a mental health condition that might affect their ability to participate.
  • Participants who have been diagnosed with a condition that is not stable or controlled.
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Investigated drugs

Ravulizumab is a medication being studied for its effectiveness in treating children and adolescents with a condition called Neuromyelitis Optica Spectrum Disorder (NMOSD) who have tested positive for Aquaporin-4 antibodies. This medication works by targeting specific parts of the immune system to help reduce inflammation and prevent attacks on the nervous system, which are common in NMOSD. The study aims to understand how well ravulizumab works, how it behaves in the body, and its safety for young patients with this disorder.

What is already known about the treatment

Ravulizumab – This medication is administered through an intravenous infusion, meaning it is given directly into a vein. It is currently being studied in clinical trials for its effectiveness in treating Neuromyelitis Optica Spectrum Disorder (NMOSD), particularly in children and adolescents who test positive for the Aquaporin-4 antibody. Ravulizumab works by inhibiting a part of the immune system known as the complement system, which is involved in inflammation and cell destruction. It belongs to a class of drugs called complement inhibitors.

Investigated diseases

Neuromyelitis Optica Spectrum Disorder (NMOSD) – This is a rare autoimmune disease that primarily affects the central nervous system, especially the optic nerves and spinal cord. It is characterized by severe inflammation and demyelination, which can lead to vision loss, paralysis, and other neurological symptoms. The disease often presents with relapses, where symptoms worsen, followed by periods of partial recovery. Over time, these relapses can cause cumulative damage, leading to increased disability. NMOSD is distinct from multiple sclerosis, although they share some similar symptoms. The exact cause of NMOSD is not fully understood, but it involves the immune system mistakenly attacking healthy cells in the nervous system.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2023-508534-33-00Protocol codeALXN1210-NMO-317Estimated enrolment12 patientsSponsorAlexion Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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