Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Rega… (NCT07596277) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
United Kingdom15 participantsStarted 2026-06-19
Plain-language summary
Spinal Muscular Atrophy Type 1 (SMA )Type 1 is a severe, early-onset neuromuscular condition that typically leads to profound weakness and impaired bulbar function-affecting swallowing, feeding, speech, and airway protection. Historically, bulbar decline contributed significantly to early morbidity and mortality.
The advent of disease-modifying therapies (DMTs) such as nusinersen, zolgensma and risdiplam (also known as Spinraza, Zolgensma, and Evrysdi) sinersinhas altered the clinical course of SMA Type 1, with emerging evidence of motor improvement and increased survival. However, the impact of these therapies on bulbar function remains poorly understood, and standardised tools for its assessment are lacking.
Qualitative research which uses interviews with parents and carers offers an opportunity to capture nuanced caregiver perspectives, identify meaningful functional outcomes, and explore daily lived experiences in a way quantitative tools currently cannot.
This study will investigate the lived experiences of families managing feeding and communication in children with SMA Type 1.
The research will also aim to
1 Identify emotional, social issues experienced by families and practical support needs related to feeding and communication.
2\. Provide insights that can inform healthcare interventions and support
Who can participate
Age range
16 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Parents/guardians of children with a diagnosis of SMA1 who have received any one or more disease modifying therapies\[CE7.1\]\[BA7.2\]\[AB7.3\]
* Participants need to be able to carry out interview in English In addition to parents, grandparents or other relatives with full parental responsibility will be included
Exclusion Criteria:
* Parents/carers who require an interpreter will not be included within the study for reasons of time and cost and because parents may feel less able to be open and honest when communicating with the researcher through a third party.
* Primary carer who is a foster carer or corporate parent (i.e. a looked after child) as they are not likely to have the same decision-making 'freedoms'.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1.
Timeframe: From interview to completion of thematic analysis 2 weeks later