RecruitingPHASE2INTERVENTIONAL

Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

This research study is investigating a drug called inebilizumab for children and adolescents between 2 and 17 years old who have a rare condition called Neuromyelitis Optica Spectrum Disorder (NMOSD). These young people must also have specific antibodies in their blood called AQP4-IgG. The main goals of the study are to understand how the medicine moves through the body (pharmacokinetics), what effects it has on the body (pharmacodynamics), and how safe it is. Participants will receive the medicine intravenously over 28 weeks, and their health will be closely monitored throughout the study, which could last up to 80 weeks.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Amgen

Enrolment target

Start

03 Jul 2023

Estimated completion

13 Apr 2027

Neuromyelitis Optica Spectrum Disorder

What is this study about?

This study is focused on a rare condition called Neuromyelitis Optica Spectrum Disorder, or NMOSD. It's a condition where the body's immune system mistakenly attacks its own healthy cells, particularly in the brain and spinal cord, leading to a range of symptoms. This particular study is for children and teenagers aged between 2 and 17 years old who have been diagnosed with NMOSD and have a specific type of antibody in their blood.

The research aims to find out more about a new treatment called inebilizumab. It's important to understand how new medicines work in young people because their bodies can react differently compared to adults. The study will look at how the medicine is absorbed and used by the body, what effects it has, and how safe it is for this age group.

By carefully studying inebilizumab, researchers hope to gather important information that could help in the development of new and effective treatments for children and teenagers with NMOSD in the future. This kind of research is vital for improving the lives of those affected by rare conditions.

Key takeaways

This study is for children and teenagers with a specific type of NMOSD.
It's testing a new medicine called inebilizumab given intravenously.
The main goals are to check the medicine's safety and how it works in the body.
Participation involves regular clinic visits over a period of up to 80 weeks.
Your health will be closely monitored throughout the study.
You can withdraw from the study at any time.

Who may be eligible?

To be considered for this study, participants must be between 2 and 17 years old, weigh at least 15 kg (about 2 stone 5 pounds), and have a clear diagnosis of NMOSD. They also need to have tested positive for specific antibodies (called AQP4-IgG) in their blood and have experienced at least one NMOSD relapse in the past year, or two or more relapses in the past two years.

There are also some reasons why someone might not be able to join. For example, if they have other significant health problems affecting their liver, kidneys, or blood, or if they are currently taking part in another drug study. Also, if they have recently received certain other treatments for NMOSD or similar conditions, they might not be eligible. The study team will review all your medical information carefully to see if this study is right for you.

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.

Are you between 2 and 17 years old?
Do you weigh at least 15 kg?
Have you been diagnosed with Neuromyelitis Optica Spectrum Disorder?
Have you had a positive blood test for AQP4-IgG antibodies?
Have you experienced an NMOSD relapse in the last year, or two or more in the last two years?
Are you currently free from other major health issues or recent specific treatments that might prevent your participation?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive the study medicine, inebilizumab, directly into your vein (intravenously) over a period of 28 weeks. The study involves several visits to the clinic. First, there's a screening period of up to 4 weeks to check if you're suitable. Then, during the 28-week treatment period, you'll have about 9 visits. After that, there's a follow-up period of about 52 weeks, with approximately 4 visits to monitor your health. The total time you would be involved in the study could be up to 80 weeks (about a year and a half). Your health will be regularly checked throughout this time to ensure your safety and monitor the effects of the treatment.

Potential risks and benefits

Participating in a clinical trial could offer the potential benefit of accessing a new treatment for your condition that is not yet widely available. However, there are also potential risks involved, as with any new medicine. These could include side effects from the medication or discomfort from procedures like blood tests or infusions. Your safety will be closely monitored by the study team, and you will receive regular medical check-ups. You are completely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (19)

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

UCSD Altman Clinical and Translational Research Institute Building
Verified postcode
La Jolla, United States· Recruiting
Loma Linda University Children's Hospital - PIN
Verified postcode
Loma Linda, United States· Recruiting
Massachusetts General Hospital
Verified postcode
Boston, United States· Recruiting
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States· Recruiting
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Verified postcode
Parque Patricios, Argentina· Recruiting
Hospital Santa Izabel-Rua Floriano Peixoto 300
Verified postcode
Salvador, Brazil· Recruiting
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
Unverified
Porto Alegre/RS, Brazil· Recruiting
CPQuali Pesquisa Clínica Sao Paulo
Verified postcode
São Paulo, Brazil· Recruiting
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
Verified postcode
São Paulo, Brazil· Recruiting
Hospital For Sick Children
Verified postcode
Toronto, Canada· Recruiting
Centre Hospitalier Universitaire de Bicêtre
Verified postcode
Le Kremlin-Bicêtre, France· Recruiting
Erasmus MC Sophia Children's Hospital-Wytemaweg 80
Verified postcode
Rotterdam, Netherlands· Recruiting

Common questions

What is NMOSD?

NMOSD is a rare condition where your body's immune system mistakenly attacks healthy cells, mainly in the brain and spinal cord.

What is inebilizumab?

Inebilizumab is the medicine being investigated in this study. It's a potential new treatment for NMOSD that aims to help manage the condition.

How will the medicine be given?

The medicine will be given directly into a vein (intravenously), meaning through a drip.

How long will I be in the study?

If you join, the study participation could last for up to 80 weeks, which includes treatment and follow-up periods.

Can I leave the study at any time?

Yes, you have the right to withdraw from the study at any point, and it won't affect your ongoing medical care.

How to find out more

Amgen Call Center

medinfo@amgen.com 866-572-6436 Official trial record

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