NCT07596277Not yet recruiting
Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication
Lived Experiences of Families of Children With Spinal Muscular Atrophy Type 1: A Qualitative Investigation Into Feeding and Communication
Source: ClinicalTrials.gov · Last updated 2 Jun 2026
Brief summary (registry, in English)
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
- Status (registry)
- Not yet recruiting
- Study type
- Observational
- Enrollment
- 15 (estimated)
- First submitted
- 13 May 2026
- Start date
- 19 Jun 2026
- Primary completion date
- 1 Nov 2026
- Lead sponsor
- Guy's and St Thomas' NHS Foundation Trust
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07596277
What you commit to
- Study type: Observational
- Planned participants: 15
- From 19 Jun 2026 to the primary completion date, 1 Nov 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- 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'.
Who the study is looking for
- 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
Age in the registry: 16 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| GSTT Evelina Neurosciences Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.