Not yet recruitingOBSERVATIONAL

Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication

This study aims to understand the daily lives of families raising children with Spinal Muscular Atrophy Type 1 (SMA1). SMA1 is a severe condition that affects muscle strength, including those needed for swallowing and speaking. While new treatments can help children with SMA1 live longer and move better, we don't fully understand how these treatments affect their feeding and communication abilities. This research will involve talking to parents and carers to learn about their experiences, challenges, and support needs. The goal is to gather insights that can help improve care and support for families of children with SMA1 in the future.

At a glance

Status

Not yet recruiting

Sponsor

Guy's and St Thomas' NHS Foundation Trust

Enrolment target

Start

19 Jun 2026

Estimated completion

01 Nov 2026

Spinal Muscular Atrophy 1

What is this study about?

Imagine being a parent of a child with Spinal Muscular Atrophy Type 1 (SMA1). This is a serious condition that makes muscles very weak, which can make things like eating, drinking, and talking very difficult. In the past, these problems often led to children becoming very poorly quite early in life.

Now, there are new medicines available (like nusinersen, zolgensma, and risdiplam) that have made a big difference. These treatments are helping children with SMA1 live longer and even improve their movement. However, we still have a lot to learn about how these medicines affect their ability to eat, swallow, and communicate. This study wants to hear directly from families about their everyday experiences managing these challenges.

The researchers will be talking to parents and carers through interviews. They want to understand the emotional and social challenges families face, as well as the practical support they need when it comes to feeding and communicating with their child who has SMA1. By listening to these personal stories, the study hopes to find out what really matters to families and use this information to help doctors and nurses offer better care and support in the future.

Key takeaways

This study focuses on families of children with SMA1.
It aims to understand daily experiences with feeding and communication.
New treatments for SMA1 are changing families' lives, and researchers want to know how.
Participation involves a one-hour online interview.
Your insights could help improve support for future families.
You must be an English-speaking parent or guardian of a child with SMA1 who has received treatment.

Who may be eligible?

This study is looking for parents or guardians of children who have been diagnosed with Spinal Muscular Atrophy Type 1 (SMA1). Your child must have received at least one of the newer treatments for SMA1.

You should be able to speak and understand English well enough to take part in an interview without an interpreter. This is because the researchers want to make sure you feel comfortable sharing your feelings openly and honestly.

Unfortunately, if you are a foster carer or looking after a child for a local authority, you won't be able to join the study. This is because the study focuses on parents who have full decision-making responsibilities for their child's long-term care.

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 a parent or guardian of a child with SMA Type 1?
Has your child received one or more of the new SMA treatments (like Spinraza, Zolgensma, or Evrysdi)?
Can you speak and understand English well enough for an interview?
Are you the primary carer and have full parental responsibility?
Are you 16 years old or older?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will have an interview with one of the researchers. This interview will be done using video conferencing, which means you can do it from the comfort of your own home. The interview is expected to last about 60 minutes (one hour). During the interview, you'll be asked about your experiences with your child's feeding and communication, as well as the emotional and practical side of things. The interview will be recorded so the researchers can accurately capture your story. There aren't any medicines or follow-up visits involved; your participation ends after the interview.

Potential risks and benefits

Taking part in this study won't offer a direct medical benefit to you or your child, but your experiences can help future families by improving our understanding of SMA1 and how to support them. The main 'risk' is that talking about difficult experiences might be upsetting, but researchers will handle your story with care and respect. You are free to stop the interview at any time or withdraw from the study without giving a reason.

Locations (1)

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

GSTT Evelina Neurosciences
Verified postcode
London, United Kingdom

Common questions

What is SMA Type 1?

SMA Type 1 is a serious genetic condition that causes muscles to become very weak, affecting a child's ability to move, eat, and breathe.

What are 'disease-modifying therapies'?

These are newer treatments like nusinersen, zolgensma, and risdiplam (also known as Spinraza, Zolgensma, and Evrysdi) that can help improve the life and movement of children with SMA Type 1.

Will I have to travel for this study?

No, the interviews will be conducted online via video conferencing, so you can participate from home.

How long will the interview take?

The interview is expected to last about 60 minutes, or one hour.

Will my child also be interviewed?

No, only parents or primary carers will be interviewed for this study.

How to find out more

Anne Breaks, PhD

anne.breaks@nhs.net +447808723798 Official trial record

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