Study of Satralizumab for Children with Neuromyelitis Optica Spectrum Disorder (NMOSD)

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What is this study about?

This clinical trial is focused on studying a condition called Neuromyelitis Optica Spectrum Disorder (NMOSD), which is a rare disease that affects the nerves in the eyes and spinal cord. The study is specifically looking at children who have a positive test for a specific antibody called Aquaporin-4 (AQP4). The treatment being tested in this study is a medication called Satralizumab, which is given as a solution for injection under the skin. Satralizumab is a type of protein that works by blocking a receptor involved in inflammation, and it is also known by its code name RO 533-3787.

The purpose of this study is to understand how Satralizumab behaves in the body, its safety, and how well it works in children with NMOSD. The study will observe how the medication is absorbed and processed in the body over a period of 24 weeks. Participants will receive regular injections of Satralizumab and will be monitored for any changes in their condition, including the frequency of relapses, which are episodes when symptoms get worse. The study will also look at changes in disability levels, vision, pain, and overall quality of life.

Throughout the study, participants will have regular check-ups to monitor their health, including tests to measure their vision, pain levels, and general well-being. The study will also track any side effects or adverse reactions to the medication. The goal is to gather information that could help improve treatment options for children with NMOSD in the future.

1 joining the study

Upon joining the study, the participant will be assessed to ensure they meet the eligibility criteria. This includes being between 2 and 11 years old, having a body weight of at least 10 kg, and being diagnosed with neuromyelitis optica spectrum disorder (NMOSD) with aquaporin-4 (AQP4) antibody positivity.

2 initial assessment

An initial assessment will be conducted to evaluate the participant’s neurological stability and overall health status. This includes a review of the Expanded Disability Status Scale (EDSS) score, which ranges from 0 to 6.5.

3 treatment administration

The participant will receive satralizumab as a solution for injection. The medication is administered via subcutaneous injection. The frequency and dosage will be determined by the study protocol and the participant’s specific needs.

4 monitoring and follow-up

Throughout the study, the participant’s response to the treatment will be monitored. This includes regular assessments of serum concentration of satralizumab to evaluate pharmacokinetics over 24 weeks.

Secondary outcomes such as the proportion of relapse-free participants by week 48, annualized relapse rate, and changes in visual acuity and pain rating will be measured at weeks 24 and 48.

5 safety and efficacy evaluation

The study will also assess the safety and tolerability of satralizumab. This includes monitoring for adverse events, changes in vital signs, weight, height, and clinical laboratory test results.

The presence of anti-drug antibodies (ADAs) will be evaluated at baseline and throughout the study.

6 completion of the study

The study is expected to conclude by February 28, 2029. Upon completion, a final assessment will be conducted to summarize the participant’s overall response to the treatment and any changes from baseline measurements.

Who Can Join the Study?

The child must be between 2 and 11 years old.
The child must weigh at least 10 kilograms.
If the child is a girl who has started menstruating, she must agree to either not have sexual intercourse or use a reliable form of birth control.
The child must have been diagnosed with Neuromyelitis Optica Spectrum Disorder (NMOSD) and have a positive test for AQP4 antibodies. These are specific proteins in the blood related to the condition.
The child’s neurological condition must have been stable for at least 30 days before the screening and the start of the study. This means there should be no significant changes in their symptoms.
The child can have normal or abnormal neurological status, which is measured by a scale called EDSS. This scale ranges from 0 to 6.5, where 0 means no disability and 6.5 indicates more severe disability.

Who Cannot Join the Study?

Participants who are not within the age range of 2 to 11 years old.
Participants who do not have a diagnosis of Neuromyelitis Optica Spectrum Disorder (NMOSD). This is a condition that affects the nerves in the eyes and spinal cord.
Participants who are unable to comply with the study procedures or follow-up requirements.
Participants who have any other medical condition that the study doctors believe would make it unsafe for them to participate.
Participants who are currently participating in another clinical trial or have recently participated in one.
Participants who have a known allergy or adverse reaction to the study medication or any of its ingredients.
Participants who are pregnant or breastfeeding.
Participants who have a history of substance abuse or dependency that could interfere with the study.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Fondazione Istituto Neurologico Nazionale Casimiro Mondino	Pavia	Italy
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Hopital Beaujon	Clichy	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy

Trial status

Country	Status	Recruitment Start
France	Recruiting	28.06.2024
Italy	Recruiting	28.06.2024
Poland	Recruiting	28.06.2024

Trial locations

Investigated drugs:

Satralizumab

Satralizumab is a medication being studied for its effects on children with a condition called Neuromyelitis Optica Spectrum Disorder (NMOSD), which is associated with certain antibodies. This study aims to understand how the body processes the medication, its effectiveness, safety, and how well it is tolerated by children aged 2 to 11 years. Satralizumab works by targeting specific parts of the immune system to help reduce inflammation and prevent attacks related to NMOSD.

Investigated diseases:

Neuromyelitis optica spectrum disorder

Neuromyelitis Optica Spectrum Disorder (NMOSD) – Neuromyelitis Optica Spectrum Disorder is a condition that primarily affects the central nervous system, particularly the optic nerves and spinal cord. It is characterized by inflammation and demyelination, which can lead to symptoms such as vision loss, weakness, and paralysis. The disease often presents with episodes of attacks or relapses, followed by periods of partial recovery. Over time, these attacks can accumulate, potentially leading to more severe neurological impairments. NMOSD is distinct from multiple sclerosis, although they share some similar features. The progression of the disease can vary significantly among individuals, with some experiencing frequent relapses and others having long periods of remission.

Trial ID:

2023-507817-85-00

Protocol code:

WN41733

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of Satralizumab for Children with Neuromyelitis Optica Spectrum Disorder (NMOSD)

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