Clinical Trials for Neuromyelitis Optica Spectrum Disorder
There are currently 4 ongoing clinical trials investigating new treatments for neuromyelitis optica spectrum disorder. These studies are testing different medications aimed at reducing inflammation and preventing relapses in both children and adults with this rare autoimmune condition. The trials are being conducted across several European countries including France, Italy, the Netherlands, Poland, Spain, and Sweden. (Also known as: NMOSD, Devic disease, Devic’s syndrome)
Clinical trial locations
- France
- Study of Inebilizumab for Children and Adolescents with Neuromyelitis Optica Spectrum Disorder
- Study of Satralizumab for Children with Neuromyelitis Optica Spectrum Disorder (NMOSD)
- Study on the Effectiveness and Safety of Ravulizumab for Children and Adolescents with Neuromyelitis Optica Spectrum Disorder (NMOSD)
- Italy
- Netherlands
- Poland
- Spain
- Sweden
Study of Inebilizumab for Children and Adolescents with Neuromyelitis Optica Spectrum Disorder
This study is investigating Inebilizumab, a monoclonal antibody treatment, in children and adolescents between 2 and 18 years of age. The medication is administered through an intravenous injection and works by targeting specific B cells in the immune system that contribute to inflammation.
Main inclusion criteria: Participants must weigh at least 15 kg and have a confirmed diagnosis with positive anti-AQP4-IgG antibodies. They must have experienced at least one acute relapse within the past year, or two or more relapses within the past two years. Both males and females who are sexually active must agree to use effective birth control methods throughout the study and for several months after the last treatment dose.
Main exclusion criteria: Children without a confirmed diagnosis, those outside the specified age range, pregnant or breastfeeding individuals, and those with allergies to the study medication cannot participate. Recent participation in other clinical trials may also prevent eligibility.
Study focus: The trial aims to understand how Inebilizumab behaves in the body, measure its effects on CD20-positive B-cell counts, and assess its safety in young patients. Regular follow-up visits are scheduled to monitor blood cell counts, track any relapses, and evaluate changes in quality of life and visual function.
Study of Satralizumab for Children with Neuromyelitis Optica Spectrum Disorder (NMOSD)
This trial focuses on Satralizumab, a medication given as an injection under the skin for children aged 2 to 11 years who test positive for aquaporin-4 antibodies. The drug works by blocking the interleukin-6 receptor, which plays a role in the inflammatory process.
Main inclusion criteria: Children must be between 2 and 11 years old and weigh at least 10 kilograms. They must have a confirmed diagnosis with positive AQP4 antibodies and have been neurologically stable for at least 30 days before the study begins. Their disability score on the EDSS scale should range from 0 to 6.5. Girls who have started menstruating must agree to use reliable birth control or abstain from sexual activity.
Main exclusion criteria: Children outside the specified age range, those without a confirmed diagnosis, those unable to follow study procedures, and those with known allergies to the medication are excluded. Pregnant or breastfeeding participants and those with recent substance abuse histories cannot join.
Study focus: The research will monitor how the medication is absorbed and processed over 24 weeks, track the proportion of relapse-free participants by week 48, and measure changes in disability levels, visual acuity, pain, and quality of life at weeks 24 and 48.
Study on the Effectiveness and Safety of Ravulizumab for Children and Adolescents with Neuromyelitis Optica Spectrum Disorder (NMOSD)
This study evaluates Ravulizumab, a complement inhibitor given as an intravenous infusion, in children and adolescents aged 2 to 18 years. The medication targets the complement system, part of the immune system involved in inflammation.
Main inclusion criteria: Participants must be between 2 and 18 years old with a confirmed diagnosis and positive anti-AQP4 antibodies. They must have an EDSS score of 7 or less. Those not previously treated with complement inhibitors must have had at least one attack in the past year. Participants already on eculizumab must have been stable and on treatment for at least 90 days. All participants must be vaccinated against meningococcal infection, Haemophilus influenzae type b, and Streptococcus pneumoniae according to specific timelines.
Main exclusion criteria: Individuals with serious health conditions that could interfere with the study, those taking medications that might affect results, those with recent infections, pregnant or breastfeeding individuals, and those who have recently participated in other trials are excluded.
Study focus: The trial will measure the annualized relapse rate, time to first relapse, changes in disability status, visual acuity, and quality of life. Blood tests will monitor medication concentrations and other health parameters throughout the study, which continues until 2028.
Study on Imlifidase for Treating Acute Inflammation in Patients with Neuromyelitis Optica Spectrum Disorder
This trial investigates Imlifidase, an enzyme-based therapy administered intravenously, for adults experiencing acute inflammation. The medication works by breaking down harmful antibodies that target the aquaporin-4 protein.
Main inclusion criteria: Participants must be at least 18 years old with a confirmed diagnosis and positive anti-AQP4 IgG antibodies. They must have experienced a recent worsening of symptoms within 14 days before treatment, such as weakness from myelitis or vision loss from optic neuritis. Immediate steroid treatment must be required. Participants can be newly diagnosed or already receiving maintenance treatments. They must test negative for hepatitis B, hepatitis C, and HIV. Women of childbearing age must use effective birth control for at least 6 months after treatment, and men must use two forms of birth control for at least 2 months after treatment.
Main exclusion criteria: Individuals without a confirmed diagnosis, those in vulnerable populations unable to give proper consent, and those with certain disabilities are excluded.
Study focus: The primary goal is to determine if Imlifidase can reduce harmful antibody levels to undetectable amounts within 6 hours of treatment. This rapid reduction could help manage severe symptoms associated with acute attacks. The study will conclude by December 2025.
Summary
Three of the four ongoing trials focus specifically on pediatric populations, reflecting an important need for treatment options in children and adolescents with this rare condition. France participates in three trials, making it the most represented country in this research effort, followed by Italy, Poland, and Spain with two trials each. The Netherlands and Sweden each host one trial.
The trials investigate four different therapeutic approaches: Inebilizumab and Satralizumab target specific immune pathways, Ravulizumab inhibits the complement system, and Imlifidase offers a rapid antibody-reduction approach for acute attacks in adults. All participants must have positive anti-AQP4 antibodies, confirming the autoimmune nature of their condition. The studies share a common focus on reducing relapse rates, improving disability outcomes, and closely monitoring safety, particularly regarding infections that may arise from immune system suppression.
