Parent-reported quality of life measures for young children with primary ciliary dyskinesia
| ISRCTN | ISRCTN87361437 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN87361437 |
| IRAS number | 63800 |
| Secondary identifying numbers | NIHR200470, IRAS 63800 |
- Submission date
- 27/05/2020
- Registration date
- 06/09/2022
- Last edited
- 20/01/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Genetic Diseases
Plain English summary of protocol
Background and study aims
In the airway, cells are lined with many hair-like structures called cilia that work together to sweep away and clear mucus, bacteria and otherdebris from the lungs, nose and ears. In primary ciliary dyskinesia (PCD), problems with the movement of the cilia result in mucus build-up. This causes constant symptoms from birth, which become worse during frequent chest, ear and sinus infections. All children with PCD eventually suffer permanent lung damage. PCD occurs in about 1 in 10,000 people and is caused by a genetic change inherited from both parents.
There is a need for new treatments to prevent children with PCD from getting infections and to delay lung damage. Treatments are also needed to improve how children with PCD feel in their daily life in terms of reducing symptoms and improving their energy levels. Researchers are developing new treatments and now urgently need ways to measure whether these treatments work. Health-related quality of life questionnaires provide a way for patients to report changes in their symptoms and well-being when they start a new treatment. Patients are more likely to stick with a treatment if it makes them feel and function better or if it has fewer side effects. Young children may be unable to explain how they feel, so instead, their parents will be asked about their child's symptoms and how these affect daily living such as sleeping and eating. Asking the child's parent about how their child's disease affects them at a particular time in a standard way also helps to measure the impact of a new treatment.
The aim of this study is to find out the symptoms and burdens that are most important to the child and parent and to create and test a questionnaire that asks about these symptoms in a standard way. This questionnaire will provide a way for patients to report changes in their child's symptoms and well-being when they start a new treatment.
Who can participate?
Parents of children with primary ciliary dyskinesia aged 6 years or below.
What does the study involve?
The researchers have already developed health-related quality of life questionnaires for older children and adults (called QOL-PCD) that are being used to decide whether treatments work in patients with PCD. These questionnaires have been translated into many languages and are being used in research studies across the world. the research team will use this expertise to develop parent-reported questionnaires for younger children. A researcher will interview 20-30 parents to understand how PCD impacts their child’s life. They will use this knowledge to develop a questionnaire called QOL-PCDPR (Parent-Reported). The questionnaires will be tested by 70 parents, to ensure it is a strong and accurate measure for testing whether a particular treatment works in pre-school children with PCD.
What are the possible benefits and risks of participating?
The benefits in participating in this study is that it provides parents of very young children an opportunity to provide their voice and enable their experiences to be incorporated into the parent-reported questionnaire.
The are no risks in participating in this study.
Where is the study run from?
The University of Southampton (UK)
When is the study starting and how long is it expected to run for?
October 2019 to December 2023
Who is funding the study?
National Institute for Health Research (NIHR) (UK)
Who is the main contact?
l.behan@soton.ac.uk
Contact information
Public
National Institute for Health Research
Biomedical Research Centre
PCD Centre
University of Southampton
Tremona Road
Southampton
SO166YD
United Kingdom
 ORCID ID |
0000-0001-8701-9975 |
|---|---|
| Phone | +44 (0)23 8120 6160 |
| jlucas1@soton.ac.uk |
Public
University of Southampton
Southampton General Hospital
Tremona Road
Southampton
SO16 6YD
United Kingdom
| ORCID ID |
0000-0002-0748-6141 |
|---|---|
| Phone | +353 87 9585887 |
| l.behan@soton.ac.uk |
Study information
| Study design | Observational cross sectional international multi center study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Mixed methods study involving both qualitative and quantitative methods to determine what factors impact on parents and very young children with PCD. Data will be analysed to develop and validate a parent-reported outcome measure. |
| Study setting(s) | Community |
| Study type | Quality of life |
| Participant information sheet | Not available in web format, please use contact details to request a participant information sheet |
| Scientific title | Parent-reported Quality of Life measures for young children with Primary Ciliary Dyskinesia (QOL-PCD) |
| Study acronym | QOL-PCD |
| Study objectives | To develop and validate a parent-reported outcome measure to evaluate the impact of PCD in young children: QOL-PCDPR. The QOL-PCDPR will eventually be used for monitoring in clinical practice and for use as an outcome measure in clinical trials. |
| Ethics approval(s) | Approved 27/09/2019, NHS Health Research Authority South Central - Hampshire A Research Ethics Committee (Level 3, Block B, Whitefriars, Lewins Mead, Bristol, BS1 2NT, UK; +44 (0)207 104 8214; hampshirea.rec@hra.nhs.uk), ref: 06/Q1702/109 |
| Health condition(s) or problem(s) studied | Primary ciliary dyskinesia |
| Intervention | To inform the development of the patient-reported outcome measure (QOL-PCDPR), individual, semi-structured open-ended interviews will be conducted by telephone with parents by the research fellows who have extensive training and experience in conducting qualitative interviews and has no pre-existing relationships with the study participants. Interviews will be audio-taped and transcribed using content analysis using NVivo (version 8.0, QSR International Pty Ltd). Elements of the coding and analyses will independently conducted by the two Research Fellows who then reach consensus. Thematic coding will identify key symptoms and impacts. These data will be analyzed for their frequency of endorsement and level of impact. Saturation matrices will inform item generation to ensure that data saturation is achieved (i.e. no new themes arising with new interviewees). Agreement on question selection for the questionnaire and wording will be agreed during multi-disciplinary, multinational conference calls using a modified Delphi approach; we will discuss the specific quotes and saturation grids from the interviews. Selected items will be written using parent language as used in the qualitative interviews; the questions will then be combined into subscales based on the research team's conceptual framework. Participants will also be asked to complete questionnaires assessing quality of life to support the validation of the QOL-PCDPR. |
| Intervention type | Other |
| Primary outcome measure | 1. Parental burden assessed using a single semi-structured interview by telephone 2. Parent's assessment of the child's quality of life at a single time point: 2.1. The Infant Toddler Quality of Life Questionnaire (47 item short form) 2.2. The Parent Cough-Specific Quality of Life (8 item short form) 2.3. Sinunasal Questionnaire (SN-5) 2.4. Otitis media-6 questionnaire 2.5. The prototype QOL-PCDPR questionnaire (for validation) |
| Secondary outcome measures | There are no secondary outcome measures. |
| Overall study start date | 01/10/2019 |
| Completion date | 14/12/2023 |
Eligibility
| Participant type(s) | Carer |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 70 participants |
| Key inclusion criteria | Parents or guardians of young children (aged 0-6 years) who have received a diagnosis of primary ciliary dyskinesia |
| Key exclusion criteria | Does not meet inclusion criteria |
| Date of first enrolment | 01/11/2019 |
| Date of final enrolment | 30/09/2023 |
Locations
Countries of recruitment
- Australia
- Canada
- England
- United Kingdom
- United States of America
Study participating centres
Tremona Road
Southampton
S016 6YD
United Kingdom
Leeds
LS1 3EX
United Kingdom
Leicester
LE1 5WW
United Kingdom
Chelsea
London
SW3 6NJ
United Kingdom
Toronto
ON M5G 1X8
Canada
Chapel Hill
ON M5G 1X8
United Kingdom
Parkville
Melbourne
3052
Australia
Sponsor information
Hospital/treatment centre
Tremona Road
Southampton
SO166YD
England
United Kingdom
| Phone | +44 (0)23 8077 7222 |
|---|---|
| sponsor@uhs.nhs.uk | |
| Website | http://www.uhs.nhs.uk/home.aspx |
"ROR" |
https://ror.org/0485axj58 |
Funders
Funder type
Government
Government organisation / National government
- Alternative name(s)
- National Institute for Health Research, NIHR Research, NIHRresearch, NIHR - National Institute for Health Research, NIHR (The National Institute for Health and Care Research), NIHR
- Location
- United Kingdom
Results and Publications
| Intention to publish date | 14/12/2024 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Available on request |
| Publication and dissemination plan | An objective of this study is to prepare outputs, including the report for NIHR, manuscripts for publication, abstracts for conferences, information for PCD Support Group website and social media. In particular, to ensure that the QOL-PCD is freely and widely available, whilst remaining within the scope of the validation, we have copyright for the existing versions. The researchers have informed the PCD community, through international networks and publications, how to gain approval for free use for clinical and research purposes. Applicants provide four clinical researchers who developed QOL-PCD with a short protocol; they sign a declaration that they will administer, score and report the QOL-PCD according to the instruction manual. The researchers plan to make this an online procedure by the time the QOL-PCDPR is ready for use, hosted by University of Southampton. Participation by PCD-clinical leads from other English speaking countries (Australia, Canada, USA) will ensure rapid dissemination for clinical use. The researchers' previous studies for QOL-PCD have resulted in publications in European Respiratory Journal, Thorax and Annals of American Thorax Society and they would target these journals again. The researchers aim to present the QOL-PCDPR at the leading North American and European Respiratory meetings (ATS and ERS). They will use the global network BEAT-PCD to further disseminate their activities using social media and websites. The PCD Support group will ensure patients are informed of the study and its outputs through presentations by researchers at meetings, via social media, the website and newsletter. |
| IPD sharing plan | The datasets generated during the current study will be available upon request from Jane Lucas (jlucas1@soton.ac.uk) and Laura Behan (l.behan@soton.ac.uk). The type of data will include qualitative data generated through semi-structured interviews with all identifiers removed. It will be available from the publication of findings in a scientific journal and in accordance with R&D protocols. |
Editorial Notes
20/01/2025: A contact was removed.
15/12/2022: The following changes have been made:
1. The recruitment end date has been changed from 30/11/2020 to 30/09/2023.
2. The overall trial end date has been changed from 30/11/2021 to 14/12/2023 and the plain English summary was updated accordingly.
3. The intention to publish date has been changed from 30/11/2022 to 14/12/2024.
05/06/2020: Trial's existence confirmed by the National Institute for Health Research (NIHR).
