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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 mis à jour par: 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

Aperçu de l'étude

Statut

Pas encore de recrutement

Les conditions

Description détaillée

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.

Type d'étude

Observationnel

Inscription (Estimé)

15

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Sauvegarde des contacts de l'étude

Lieux d'étude

  • Royaume-Uni
      • London, Royaume-Uni, SE1 7EU
        • GSTT Evelina Neurosciences
        • Contact:

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Enfant
  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

Méthode d'échantillonnage

Échantillon non probabiliste

Population étudiée

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

La description

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'.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

Cohortes et interventions

Groupe / Cohorte
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

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1.
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Broader issues
Délai: 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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Guy's and St Thomas' NHS Foundation Trust

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Estimé)

19 juin 2026

Achèvement primaire (Estimé)

1 novembre 2026

Achèvement de l'étude (Estimé)

1 novembre 2026

Dates d'inscription aux études

Première soumission

13 mai 2026

Première soumission répondant aux critères de contrôle qualité

13 mai 2026

Première publication (Réel)

19 mai 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

2 juin 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

29 mai 2026

Dernière vérification

1 mai 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • 358595

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

INDÉCIS

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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