ChILD-EU database and observational study
| ISRCTN | ISRCTN23400701 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN23400701 |
| Secondary identifying numbers | N/A |
- Submission date
- 07/11/2013
- Registration date
- 19/12/2013
- Last edited
- 28/09/2020
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Respiratory
Plain English Summary
Background and study aims
Childhood Interstitial Lung Diseases (ChILD) are a group of rare diseases of the lung: most conditions have a poor outcome. There are too few cases in each country to enable adequate research. The ChILD-EU project, funded by the European Commission, is bringing together clinicians and chILD cases from across Europe. The study will gather information into a Europe-wide database and also enable outcomes to be studied.
Who can participate?
Infants and children coming to hospital with suspected interstitial lung disease
What does the study involve?
Information is collected on each patient at diagnosis, who are then observed over the first year following diagnosis (at 1, 2, 3, 6 and 12 months). At the time of diagnosis all patients in the database have their diagnosis and treatment reviewed by an expert team to ensure diagnostic validity. Measurements recorded typically are those routinely monitored during normal clinic visits. Parents and older children are also asked to fill in questionnaires at the start of the study and again after 3, 6 and 12 months. To enable genetic investigation, blood samples are collected from each child and their parents.
What are the possible benefits and risks of participating?
There are no direct benefits to the parents or children taking part in this study. However, the information from this study will show which approaches to treatment give better outcomes.
Where is the study run from?
The study is run from hospitals across Europe
When is the study starting and how long is it expected to run for?
January 2014 to June 2016
Who is funding the study?
European Commission Directorate-General for Research and Innovation, FP7-Health-2012-Innovation-1
Who is the main contact?
Dr Steve Cunningham
Steve.cunningham@ed.ac.uk
Contact information
Scientific
Dept of Respiratory and Sleep Medicine
Royal Hospital for Sick Children
Sciennes Road
Edinburgh
EH9 1LF
United Kingdom
 ORCID ID |
0000-0001-7342-251X |
|---|
Public
ChILD-UK Trial Office
Edinburgh Clinical Trials Unit
Level 2, Outpatients Building
Western General Hospital
Crewe Road South
Edinburgh
EH4 2XU
United Kingdom
| ORCID ID |
0000-0002-4037-0247 |
|---|---|
| Phone | +44 (0)131 537 3846 |
| morag.maclean@ed.ac.uk |
Study information
| Study design | Observational cohort multi-centre study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Hospital |
| Study type | Screening |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | Orphans Unite: ChILD better together EUropean management platform for childhood interstitial lung diseases |
| Study acronym | ChILD-EU |
| Study hypothesis | There are limited studies bringing together children with interstitial lung disease and no studies assessing the response to standardised interventions in Childhood Interstitial Lung Diseases (ChILD). The paucity of cases in each centre and the lack of an evidence-based treatment approach requires a structured observation of current practice to inform future research directions. The aim is to capture interventions and outcomes in well-characterised patients with suspected and proven ChILD. Such information will provide data on outcome in relation to standard interventions and support further research directions. Studies in France, Germany, Italy and Turkey will collect similar information to add to the UK data in the database. |
| Ethics approval(s) | South-East Scotland REC2, 08/11/2013, ref: 13/SS/0195 |
| Condition | Childhood interstitial lung disease (chILD) |
| Intervention | Observations will start from time of presentation at hospital during which the diagnosis is made and participants will continue in the trial for 12 months. Participants will be given the usual treatment for ChILD and data will be collected at seven time points. The data collected will include respiratory measurements, treatments, images of scans and histology samples and patient-reported outcome questionnaires. At study entry blood samples for genetic analysis will be collected from the participant and the participant's parents. Previously diagnosed cases of chILD will only enter the database and biobank study and so will only capture data at study entry and for peer review after 1 year. |
| Intervention type | Other |
| Primary outcome measure | For the database and biobank study - collate detailed information on clinical cases of possible ChILD on a central database and biobank. For the observational study - describe outcomes at 1, 2, 3, 6 and12 months in infants and children with ChILD. Outcomes measured will be: 1. Death 2. Survival on artificial ventilatory support (invasive or non-invasive) 3. Survival in supplemental oxygen 4. Survival breathing room air 5. Quality of life (QoL) |
| Secondary outcome measures | For the database and biobank study: 1. To review each case by an experienced international interdisciplinary peer review team to provide diagnostic oversight and feedback 2. To provide annual updates of diagnosis and outcome in a feedback loop via peer review 3. To store for future research, blood samples for genetic analysis of cases and parents 4. To support paediatricians and families caring for children with ChILD For the observational study: To describe variance in outcome at 1, 2, 3, 6 and 12 months in infants and children with ChILD according to: 1. Diagnosis and presentation 1.1. Diagnosis (peer review) 1.2. Diagnostic certainty (peer review) 1.3. Computed tomography (CT) score by component radiologist (peer review) 1.4. Blood oxygen saturation (SpO2) at rest in room air at presentation 1.5. SpO2 asleep in room air at presentation (nadir) 1.6. Respiratory rate (RR) (z score) at rest in air at presentation 1.7. Heart rate (HR) (z score) in air at presentation 1.8. Blood pressure at rest for 5 minutes at presentation 1.9. Weight (z-score) at presentation 1.10. Leland Fan 5 point severity score (nil, symptoms, SpO2 <90% air asleep, SpO2 at rest, pulmonary hypertension). 2. Time to treatment and improvement 2.1. Time from onset of symptoms/signs of ChILD to first treatment 2.2. Time from onset of symptoms/signs of ChILD to diagnosis (local clinical) 2.3. Time from onset of symptoms/signs of ChILD to normoxia whilst awake (SpO2 ≥94% breathing room air at rest) 2.4. Time from onset of symptoms/signs of ChILD to respiratory rate in normal range for age (Fleming, Thompson et al. 2011) 2.5. Time from onset of first treatment to reduction in RR by 10% 2.6. Time from onset of first treatment to reduction in HR by 20% 2.7. Time from onset of symptoms/signs of ChILD to normoxia whilst asleep (SpO2 ≥94% breathing room air at rest) 2.8. Time from onset of symptoms/signs of ChILD to weight appropriate for age/height without use of calorie supplementation 2.9. Time from onset of treatment to improvement in weight by 10% 3. Treatments 3.1. Steroids: use of steroids, dose, route and frequency of steroid use, time from first presentation to initiation of steroids, number of concomitant ChILD treatments at time of starting steroids 3.2. Hydroxychloroquine: use of hydroxychloroquine, dose and frequency of hydroxychloroquine, time from first presentation to initiation of hydroxychloroquine, number of concomitant ChILD treatments at time of starting hydroxychloroquine 3.3. Azithromycin: use of azithromycin, dose and frequency of azithromycin, time from first presentation to initiation of azithromycin, number of concomitant ChILD treatments at time of starting azithromycin 4. Concomitant medicines 5. Follow-up review 5.1. SpO2 in room air measured 4 weeks after commencing initial treatment 5.2. RR at rest measured 4 weeks after commencing initial treatment 5.3. Heart rate at rest measured 4 weeks after commencing initial treatment 6. Quality of Life score - PEDS QL Generic Core Scales at 0 and 12 months 7. Questionnaire for health care utilisation and costs 7.1. Utilisation of inpatient and outpatient care to calculate direct costs gathered at 0, 3, 6 and 12 months 7.2. Loss of productivity of parents and children to calculate indirect costs gathered at 0, 3, 6 and 12 months |
| Overall study start date | 01/12/2013 |
| Overall study end date | 30/11/2016 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Sex | Both |
| Target number of participants | 32 |
| Total final enrolment | 127 |
| Participant inclusion criteria | Infants and children presenting to hospital with clinician-suspected interstitial lung disease or at least three of the following four criteria present: 1. Respiratory symptoms for at least 14 days 1.1. Cough 1.2. Rapid and/or difficult breathing 1.3. Exercise intolerance 2. Respiratory signs 2.1. Tachypnea 2.2. Adventitious sounds 2.3. Retractions 2.4. Digital clubbing 2.5. Failure to thrive 2.6. Respiratory failure 3. Hypoxemia 4. Diffuse abnormalities on a chest radiograph or computerised tomography (CT) scan |
| Participant exclusion criteria | A participant would be excluded from the database if ineligible to participate in the ChILD-EU Minimal Dataset observation and follow-up study. Exclusion criteria are common causes of diffuse lung disease, including but not exclusively: 1. Cystic fibrosis 2. Respiratory distress syndrome 3. Bronchopulmonary dysplasia 4. Acute infection (viral or bacterial) 5. Inherited or acquired immune deficiency |
| Recruitment start date | 01/04/2014 |
| Recruitment end date | 30/11/2016 |
Locations
Countries of recruitment
- England
- Scotland
- United Kingdom
- Wales
Study participating centres
EH9 1LF
United Kingdom
Oxford
OX3 9DU
United Kingdom
Eaton Road
Liverpool
L12 2AP
United Kingdom
London
SE5 9RS
United Kingdom
Leeds
LS1 3EX
United Kingdom
Derby Road
Nottingham
NG7 2UH
United Kingdom
Aberdeen
AB25 2ZG
United Kingdom
Bristol
BS2 8BJ
United Kingdom
London
SW3 6NP
United Kingdom
Govan
G51 4TF
United Kingdom
Newcastle upon Tyne
NE1 4LP
United Kingdom
Manchester
M13 9WL
United Kingdom
Sheffield
S10 2TH
United Kingdom
Birmingham
B4 6NH
United Kingdom
London
WC1N 3JH
United Kingdom
Southampton
SO16 6YD
United Kingdom
Heath Park
Cardiff
CF14 4XW
United Kingdom
Whitechapel
London
E1 1BB
United Kingdom
Sponsor information
Research organisation
University of Edinburgh & NHS Lothian
The Queens Medical Research Institute
47 Little France Crescent
Edinburgh
EH16 4TJ
United Kingdom
| Website | http://www.accord.ed.ac.uk/ |
|---|---|
"ROR" |
https://ror.org/01x6s1m65 |
Funders
Funder type
Government
No information available
Results and Publications
| Intention to publish date | 30/11/2017 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Stored in repository |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal within one year after the end of the trial. |
| IPD sharing plan | The datasets generated during and/or analysed during the current study will be stored in a non-publically available repository – the Child-EU registry which is administered by the Kids Lung Register Foundation. Access to anonymised datasets should be requested from the Kids Lung Registry Foundation. Contact ChILD-EU.register@med.uni-muenchen.de and Prof Matthias Griese (Matthias.Griese@med.uni-muenchen.de) for further information. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/02/2020 | 28/09/2020 | Yes | No |
| HRA research summary | 28/06/2023 | No | No |
Editorial Notes
28/09/2020: Publication reference and total final enrolment number added, contact details updated.
04/05/2017: The overall trial end date was changed from 31/05/2016 to 30/11/2016.
