RecruitingPHASE3INTERVENTIONAL

A Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)

This study is investigating a new medication, Satralizumab, in young children (aged 2 to 11) who have a rare condition affecting the brain and spinal cord called Neuromyelitis Optica Spectrum Disorder (NMOSD). Specifically, these children will have a particular antibody in their blood called anti-aquaporin-4 (AQP4), which is important for this type of NMOSD. The main goal is to understand how the medicine is absorbed, distributed, and removed from the body in these children. The study will also be looking at how effective the drug might be in treating NMOSD, its safety, and whether children can tolerate it well. Due to the small number of participants expected, these findings will be described broadly rather than making definitive conclusions.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Hoffmann-La Roche

Enrolment target

Start

06 May 2026

Estimated completion

12 Sep 2029

Neuromyelitis Optica Spectrum Disorder NMOSD

What is this study about?

This research study is about a condition called Neuromyelitis Optica Spectrum Disorder, or NMOSD. It's a rare, lifelong condition that affects the optic nerves and spinal cord, and sometimes other parts of the brain. It can cause problems with vision, movement, sensation, and other body functions. In NMOSD, the body's immune system mistakenly attacks healthy cells, particularly those that have a protein called aquaporin-4 (AQP4). This study is specifically for children with NMOSD who have these AQP4 antibodies in their blood.

The study is focusing on a medicine called Satralizumab. This medicine aims to help manage NMOSD. Doctors want to understand how this medicine works in children, as it's important to make sure treatments are suitable and safe for younger patients. This type of research helps doctors learn more about diseases and develop new and better ways to help people living with them.

While the main aim is to see how the medicine travels through the body, researchers will also carefully observe if Satralizumab helps reduce NMOSD attacks, if there are any side effects, and generally how well children tolerate the treatment. This information is crucial for deciding if Satralizumab could be a helpful treatment option for children with NMOSD in the future.

Key takeaways

The study tests a new medicine, Satralizumab, for children with NMOSD.
It's for children aged 2-11 who have a specific type of NMOSD (AQP4 antibody positive).
Mainly looks at how the medicine works in the body, also checking safety and effectiveness.
Participation involves regular clinic visits and close medical monitoring.
Your child's health and safety are the top priority.
Joining is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, children need to be between 2 and 11 years old and weigh at least 10 kilograms (about 22 pounds). They must have been officially diagnosed with NMOSD and have the specific anti-AQP4 antibody in their blood. They need to have experienced at least one NMOSD attack in the year before the study starts, and their general neurological condition should have been stable for at least 30 days before joining.

There are also some reasons why a child might not be able to join. For example, if they are pregnant or breastfeeding, or if they have other illnesses that could be confused with NMOSD. Children who currently have serious infections, certain liver conditions, or untreated tuberculosis won't be able to take part. Also, if they've recently had a 'live' vaccine or have a history of severe allergic reactions to similar medicine, they wouldn't be eligible.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child aged between 2 and 11 years?
Does your child weigh at least 10 kilograms (about 22 pounds)?
Has your child been diagnosed with NMOSD and tested positive for the AQP4 antibody?
Has your child had at least one NMOSD attack in the past year?
Has your child's neurological condition been stable for at least 30 days?
Is your child not pregnant or breastfeeding (if applicable)?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive the study medication, Satralizumab. This will involve regular visits to the clinic for assessments, including blood tests, physical examinations, and checks on their NMOSD symptoms. The research team will carefully monitor your child's health throughout the study to track how the medicine is working and to watch for any side effects. These visits will be spread out over a period that ensures thorough monitoring. The total duration of the study and follow-up will be explained in detail by the research team.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as gaining access to a new treatment for NMOSD before it becomes widely available and receiving close medical attention from specialists. However, there are also potential risks, including side effects from the study medication, which may or may not be known yet. It's important to remember that this new treatment might not work for everyone. You will be fully informed about all known risks before deciding to participate, and you always have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (13)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Children's Hospital Colorado.
Verified postcode
Denver, United States· Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Unverified
Ciudad Autonoma Buenos Aires, Argentina· Recruiting
Clinica Universitaria Reina Fabiola
Verified postcode
Córdoba, Argentina· Recruiting
Guangzhou Women and Children's Medical Center
Verified postcode
Guangzhou, China· Recruiting
Children's Hospital of Fudan University
Verified postcode
Shanghai, China· Recruiting
Centre Hospitalier Universitaire de Bicêtre
Verified postcode
Le Kremlin-Bicêtre, France· Recruiting
IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
Verified postcode
Rome, Italy· Recruiting
Fondazione Istituto Neurologico Mondino IRCCS
Verified postcode
Pavia, Italy· Recruiting
Grupo Medico Camino
Unverified
DF, Mexico· Recruiting
Uniwersyteckie Centrum Kliniczne
Unverified
Gda?sk, Poland· Active not recruiting
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Verified postcode
Warsaw, Poland· Recruiting
Kocaeli University Research and Application Hospit
Verified postcode
Kocaeli, Turkey (Türkiye)· Recruiting

Common questions

What is NMOSD?

NMOSD stands for Neuromyelitis Optica Spectrum Disorder, a rare condition that affects the brain, optic nerves, and spinal cord.

What is Satralizumab?

Satralizumab is a medicine being studied as a potential treatment for NMOSD.

Why focus on children aged 2-11?

This study specifically looks at how Satralizumab affects younger children, as treatments need to be carefully tested in different age groups.

What does 'pharmacokinetics' mean?

It means the study will examine how the medicine moves through your child's body – how it's absorbed, distributed, used, and removed.

Can my child stop participating at any time?

Yes, you have the right to withdraw your child from the study at any point without any impact on their usual medical care.

How to find out more

Reference Study ID Number: WN41733 https://forpatients.roche.com/

global-roche-genentech-trials@gene.com 888-662-6728 (U.S.) Official trial record

Always speak to your GP or specialist before deciding to take part in a study.