RecruitingOBSERVATIONAL

An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)

This study, called Active-NBS, is investigating the muscle development of very young children in the UK who have Spinal Muscular Atrophy (SMA). New medicines for SMA work best when given early, possibly before symptoms appear. This research aims to understand if, and when, the movements of children with SMA, diagnosed at birth, differ from healthy children as they grow. Researchers will use special wearable devices that children wear at home to track their movements over up to 30 months. This will help them learn when new treatments currently being developed might be most effective. The study also includes healthy children to understand normal development using these devices, comparing it to those with SMA. It's all about finding the best way to support muscle development in children with SMA.

At a glance

Status

Recruiting

Sponsor

University of Oxford

Enrolment target

Start

01 May 2026

Estimated completion

01 Jul 2029

Spinal Muscular Atrophy (SMA)

What is this study about?

The Active-NBS study is looking into how young children with a condition called Spinal Muscular Atrophy (SMA) develop their muscle movements. In recent years, new treatments for SMA have become available, and they work best when started as early as possible, ideally before a child even shows any symptoms. However, even with early diagnosis, some children might still develop muscle weakness despite receiving treatment.

This study wants to better understand the muscle development of children diagnosed with SMA at birth. By closely monitoring their movements, researchers hope to discover at what point their development might start to differ from children without SMA. This vital information will help doctors understand the best timing for giving additional new medicines and therapies that are currently being developed to support muscle strength and function.

To do this, the study will involve children wearing special, easy-to-use devices at home that track their movements. These devices are designed to be worn without the need for extra hospital visits. By comparing the information from children with SMA to that from healthy children, the researchers aim to build a clearer picture of how SMA impacts movement and how early treatments can be made as effective as possible. The study is being conducted in the UK and internationally, with the University of Oxford overseeing the collection and analysis of information.

Key takeaways

The study aims to understand muscle development in young children with SMA.
It uses special wearable devices worn at home to track movement.
The goal is to find the best time to give new SMA treatments.
Healthy children will also participate to help understand normal development.
Participation will last up to 30 months and involves no extra hospital visits.
Findings could improve future care for children with SMA.

Who may be eligible?

The study is looking for two main groups of children. The first group includes children with SMA who were diagnosed through newborn screening or other means. They should be between 4 months and under 4 years old when they start the study, though some younger babies might also be included. These children will have a confirmed diagnosis of SMA and might or might not be receiving existing SMA treatments.

For the second group, researchers are looking for healthy children without SMA. These children should be between 6 months and 4 years old. They will help the researchers understand typical development using the special wearable devices.

For both groups, parents or legal guardians must be able to give their consent for their child to take part. Children cannot participate if they have another serious medical condition that would strongly affect their movement or if they are already part of another experimental treatment study.

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 between 4 months and under 4 years old (or a newborn baby)?
Does your child have a confirmed diagnosis of SMA?
Do you, as a parent or guardian, agree to your child taking part?
Is your child otherwise healthy, without serious conditions affecting movement?
Is your child not currently involved in another experimental treatment study?

Answer every question to see your result.

What does participation involve?

If your child takes part, they will be asked to wear special devices called Syde and Motor Assessment of an Infant in a Jumpsuit (MAIJU) at home. These devices track your child's movements. You won't need to make extra trips to the hospital for this part of the study. The study plans to follow children for up to 30 months. There are no specific medications given as part of this study, as it's focused on monitoring movement. The researchers will explain exactly how often and for how long the devices need to be worn. You'll be supported throughout the study by the research team.

Potential risks and benefits

Taking part in this study could help us learn more about SMA and muscle development in young children, which might lead to better timing for new treatments in the future. While there are no direct medical benefits for your child from taking part, your contribution is very valuable for future generations. The risks are expected to be very low, mainly involving the minor inconvenience of wearing the devices. These devices are non-invasive and designed for comfort. You can withdraw your child from the study at any time, for any reason, without it affecting their medical care.

Locations (1)

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

University of Oxford
Verified postcode
Oxford, United Kingdom· Recruiting

Common questions

What is Spinal Muscular Atrophy (SMA)?

SMA is a rare genetic condition that affects the nerves responsible for muscle movement, causing muscles to weaken over time.

What are newborn screening (NBS) and disease-modifying therapy (DMT)?

Newborn screening is a test done shortly after birth to check for certain health conditions. Disease-modifying therapy refers to medicines that can slow down or stop the progression of a disease.

What are 'wearable technologies'?

Wearable technologies are small devices, like smartwatches or activity trackers, that can be worn on the body to collect information, in this case, about movement.

Will my child have to go to the hospital a lot?

No, the main part of this study involving wearable devices is designed so your child can wear them at home, without needing extra hospital visits.

How long will the study last for each child?

Each child will be followed for up to 30 months, which is about two and a half years.

How to find out more

Charlotte Lilien

charlotte.lilien@paediatrics.ox.ac.uk 01865618799 Official trial record

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