Active not recruitingNAINTERVENTIONAL

Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).

This study is investigating a new, enhanced rehabilitation program for children with Spinal Muscular Atrophy (SMA) in the UK. The goal is to find out if this updated program is helpful, can be easily used by families, and is safe for children aged 1 to 10 years old. The new program offers more hands-on physiotherapy and provides special equipment for use at home. This is designed to better support children and their families who are currently managing rehabilitation largely on their own. Researchers want to compare this new program to the current rehabilitation methods to see if it makes a difference in how well children can move and function, and if families find it more acceptable and easier to stick with. The study will last for 12 months for each participant.

At a glance

Status

Active not recruiting

Phase

Sponsor

University of Oxford

Enrolment target

Start

01 Jun 2024

Estimated completion

01 Dec 2026

Spinal Muscular Atrophy

What is this study about?

This research study is about finding better ways to help children in the UK who have Spinal Muscular Atrophy (SMA). Specifically, it's looking into a new, improved rehabilitation program. Rehabilitation involves exercises and therapies that help people improve their movement and strength. The current way rehabilitation is often done can be a lot for families to manage, especially providing therapy at home by themselves. This study wants to see if an updated approach can make things easier and more effective for these children and their families.

The new rehabilitation program is designed to be more focused on individual goals and will include more frequent, direct physiotherapy sessions. This means a physiotherapist will work hands-on with the child more often, specifically every two weeks. On top of this, families will be given special equipment to use at home once a week. The main idea is to give children with SMA the most suitable therapy for their specific needs, aiming to see if this extra support and equipment can lead to better results than the current standard of care.

Over 12 months, all children in this study will follow the new rehabilitation program. Researchers will check if it's something families find acceptable and if it's practical to do. They will also carefully watch to make sure it's safe. To understand if the new program is making a positive difference, they will compare the children's progress to a group of children not in this study, who are receiving the usual care. Ultimately, the study hopes to show if this 'optimized' program could become a new, better standard for SMA rehabilitation.

Key takeaways

This study investigates a new rehabilitation program for children with SMA.
It aims to improve therapy with more frequent physiotherapy and home equipment.
The study lasts 12 months and involves three visits.
It's for children aged 1-10 already on SMA medication.
The goal is to provide better support for children and families.
Participants will not receive new medication.

Who may be eligible?

To join this study, children must be between 1 and 10 years old at the start of the study and have a confirmed diagnosis of Spinal Muscular Atrophy (SMA). They must have been taking a market-approved SMA medicine for at least 12 months and be on a steady dose. This study is open to children with SMA who are considered 'non-sitters', 'sitters', or 'walkers', meaning those who can't sit independently, those who can, and those who can walk.

Certain situations would prevent a child from joining. For example, if they have another serious medical condition that would make rehabilitation difficult, or if they need breathing support all day (not just for naps). Children already taking part in another treatment study or receiving an experimental treatment are also not eligible. Also, if a child has had surgery or a medical procedure within 3 months before the start of the study, or needs one during the study that might interfere with their rehabilitation, they would not be able to participate.

For children under 16, a parent or legal guardian must agree to the study by signing a consent form. The parent or guardian who will be the main caregiver for the child during the study also needs to agree to follow the study's requirements. Basically, everyone involved must be willing and able to participate fully in the program.

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 my child between 1 and 10 years old?
Does my child have a confirmed diagnosis of SMA?
Has my child been on a stable dose of an approved SMA medicine for at least 12 months?
Does my child NOT require full-time breathing support (only naps are okay)?
Has my child NOT had major surgery or a medical procedure in the last 3 months, or will they need one during the study?
Am I, as the parent/guardian, willing to commit to the study requirements?

Answer every question to see your result.

What does participation involve?

If you or your child decide to take part in this study, you will follow the new rehabilitation program for 12 months. This program involves receiving hands-on physiotherapy every two weeks and using a special rehabilitation device at home once a week. You will also have three study visits: one at the very beginning, then at 6 months, and finally at 12 months.

During these visits, your child will have a general health check-up. The research team will also ask you and your child (if they are old enough) about your experiences with the rehabilitation program, how satisfied you are, and how easy it is to stick with. To see how much the program is helping, your child will also have tests to check their movement and how they can function, appropriate for their age and whether they can sit or walk. There are no new medications given as part of this study; it focuses only on the rehabilitation program. After the final 12-month visit, your involvement with the study will end.

Potential risks and benefits

This study aims to find out if an improved rehabilitation program can offer benefits to children with SMA, potentially leading to better movement and function. There's a chance it could provide more frequent and targeted therapy than usual care, and better support for families. As with any new program, there might be some risks, such as the rehabilitation device causing minor discomfort or the increased physiotherapy leading to temporary tiredness, but the study will carefully monitor for any issues. You have the right to withdraw from the study at any time, for any reason, without it affecting your child's medical care.

Locations (1)

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

Oxford Brookes University
Verified postcode
Oxford, United Kingdom

Common questions

What is Spinal Muscular Atrophy (SMA)?

SMA is a genetic condition that affects the nerves that control muscle movement, leading to muscle weakness and wasting.

What is 'rehabilitation' in this study?

Rehabilitation refers to exercises and therapies designed to improve a child's movement, strength, and overall physical ability.

How long will the study last for my child?

If your child joins, they will be part of the rehabilitation program for 12 months.

Will my child receive any new medication in this study?

No, this study is only looking at a rehabilitation program; no new medications will be given. Children must already be on a stable dose of an approved SMA medicine.

What does 'hands-on physiotherapy' mean?

It means a physiotherapist will directly work with your child to guide their exercises and movements, rather than just giving instructions.