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
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
Přehled studie
Postavení
Podmínky
Detailní popis
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.
Typ studie
Zápis (Odhadovaný)
Kontakty a umístění
Studijní kontakt
- Jméno: Anne Breaks, PhD
- Telefonní číslo: +447808723798
- E-mail: anne.breaks@nhs.net
Studijní záloha kontaktů
- Jméno: Heinz Jungbluth, PhD
- Telefonní číslo: 02071883998
- E-mail: heinz.jungbluth@nhs.net
Studijní místa
-
-
-
London, Spojené království, SE1 7EU
- GSTT Evelina Neurosciences
-
Kontakt:
- Ming Lim, PhD
- Telefonní číslo: 02071883992
- E-mail: ming.lim3@nhs.net
-
-
Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dítě
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Metoda odběru vzorků
Studijní populace
Popis
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'.
-
Studijní plán
Jak je studie koncipována?
Detaily designu
Kohorty a intervence
Skupina / kohorta |
|---|
|
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
|
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Qualitative themes describing caregiver experiences of feeding and communication in children with SMA Type 1.
Časové okno: 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
|
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Broader issues
Časové okno: 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
|
Spolupracovníci a vyšetřovatelé
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Odhadovaný)
Primární dokončení (Odhadovaný)
Dokončení studie (Odhadovaný)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Klíčová slova
Další relevantní podmínky MeSH
- Onemocnění centrálního nervového systému
- Nemoci nervového systému
- Neuromuskulární onemocnění
- Genetické choroby, vrozené
- Nemoci trávicího systému
- Gastrointestinální onemocnění
- Neurodegenerativní onemocnění
- Nemoci jícnu
- Otorinolaryngologická onemocnění
- Heredodegenerativní poruchy, nervový systém
- Faryngeální onemocnění
- Nemoci míchy
- Nemoc motorických neuronů
- Svalová atrofie, Spinální
- Vrozené, dědičné a neonatální nemoci a abnormality
- Chování
- Slovní chování
- Poruchy deglutace
- Spinální svalové atrofie dětství
- Sdělení
- Mluvený projev
- Jazyk
Další identifikační čísla studie
- 358595
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .