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

NCT07596277 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 15

Last updated 2026-05-19

No results posted yet for this study

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

Conditions

  • Spinal Muscular Atrophy 1

Sponsors & Collaborators

  • Guy's and St Thomas' NHS Foundation Trust

    lead OTHER

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-19
Primary Completion
2026-11-01
Completion
2026-11-01

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07596277 on ClinicalTrials.gov