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

29. mai 2026 oppdatert av: Guy's and St Thomas' NHS Foundation Trust

Lived Experiences of Families of Children With Spinal Muscular Atrophy Type 1: A Qualitative Investigation Into Feeding and Communication

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

Studieoversikt

Status

Har ikke rekruttert ennå

Forhold

Detaljert beskrivelse

Methodology This study will adopt a qualitative phenomenological approach to gain in-depth insights into the experiences of families of children with SMA Type 1. It is anticipated that the study will interview 10-15 participants, the numbers will be determined by how many parents need to be interviewed to reach the point of saturation i.e. the point at which no new themes arise. The interviews will be aim to be inductive and discursive.

Prior to the interview parents will have received the information leaflet and consent form. A broad interview guide will be used to enable the interviewer to ensure all areas are covered within each interview, whilst providing flexibility to ensure all participant feedback is gathered. The interviews will be recorded digitally.

Participants The study will be advertised on the SMAUK website and their other social media platforms. Families will be invited to contact the CI if they are interested in taking part. Participants will be caregivers (parents, guardians) of children diagnosed with SMA Type 1.

The CI will then contact the potential participants and share the full information sheet and consent form. The participant will then complete and return the consent form and the PI will contact them to arrange a time for the interview. Interviews are expected to last approximately 60 minutes.

Data Collection The CI will carry out semi-structured interviews conducted via video conferencing, focusing on feeding and communication experiences. Interviews will be recorded, and questions will be based upon a broad interview guide to ensure all key areas are covered whilst being flexible to enable participants to share their experiences.

Data Analysis Recordings from interviews will be transcribed using Thematic Analysis (Braun and Clarke 2006)Thematic analysis software will be used to support the identification of recurring patterns and themes. Coding consistency will be checked by another researcher who will recode an anonymised random sample of the transcriptions.

Ethical Considerations: Informed consent will be obtained from all participants. The study will adhere to ethical guidelines to ensure confidentiality, voluntary participation, and sensitivity to emotional distress.

Studietype

Observasjonsmessig

Registrering (Antatt)

15

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studer Kontakt Backup

Studiesteder

  • Storbritannia
      • London, Storbritannia, SE1 7EU
        • GSTT Evelina Neurosciences
        • Ta kontakt med:

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

Parent/carers of children with SMA1 who have had a disease modifying treatment

Beskrivelse

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'.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Kohorter og intervensjoner

Gruppe / Kohort
Parents/guardians of children with a diagnosis of SMA1
Parents/guardians of children with a diagnosis of SMA1 who have received any one or more disease modifying therapies 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

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1.
Tidsramme: From interview to completion of thematic analysis 2 weeks later
The primary outcome is the set of emergent qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1. Data will be collected via a single semi-structured interview per participant (approximately one hour) conducted post-consent, and analysed using inductive thematic analysis (NVivo) following completion of data collection.
From interview to completion of thematic analysis 2 weeks later

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Broader issues
Tidsramme: From interview to 2 weeks after when thematic analysis is completed

Social, emotional, and practical challenges related to feeding and communication.

Caregiver perceptions of bulbar function following disease-modifying therapies (DMTs).

Identification of perceived barriers and support systems impacting feeding and communication, assessed during the same single interview session and analysed thematically.
From interview to 2 weeks after when thematic analysis is completed

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Guy's and St Thomas' NHS Foundation Trust

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

19. juni 2026

Primær fullføring (Antatt)

1. november 2026

Studiet fullført (Antatt)

1. november 2026

Datoer for studieregistrering

Først innsendt

13. mai 2026

Først innsendt som oppfylte QC-kriteriene

13. mai 2026

Først lagt ut (Faktiske)

19. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

2. juni 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

29. mai 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 358595

Plan for individuelle deltakerdata (IPD)

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UBESLUTTE

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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