Condition category
Respiratory
Date applied
07/11/2013
Date assigned
19/12/2013
Last edited
24/09/2014
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

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 project 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 can participate in this study.

What does the study involve?
We will collect information on each case at diagnosis and also observe cases over the first year following diagnosis (at 1, 2, 3, 6 and 12 months). At the time of diagnosis all patients in the database will have their diagnosis and treatment reviewed by an expert team to ensure diagnostic validity. Measurements recorded will typically be those routinely monitored during normal clinic visits. Parents and older children will also be asked to fill in questionnaires at the start of the study and again after 3, 6 and 12 months. To enable genetic investigation, we will collect blood samples 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 that we get from this study will help us to learn what approaches to treatment may give better outcomes, and use these in future studies.

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?
The study will start in January 2014 and will run until June 2016.

Who is the main contact?
Dr Steve Cunningham, steve.cunningham@nhs.net
Child UK trial office child-uk@ed.ac.uk; Tel: +44 (0)131 537 3846 / 3878

Trial website

http://www.childeu.net

Contact information

Type

Scientific

Primary contact

Dr Steve Cunningham

ORCID ID

Contact details

Dept of Respiratory and Sleep Medicine
Royal Hospital for Sick Children
Sciennes Road
Edinburgh
EH9 1LF
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

N/A

Study information

Scientific title

Orphans Unite: ChILD better together – EUropean management platform for childhood interstitial lung diseases

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. We aim 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

South-East Scotland REC2, 08/11/2013, REC ref: 13/SS/0195

Study design

Observational cohort multi-centre study

Primary study design

Observational

Secondary study design

Cohort study

Trial setting

Hospitals

Trial type

Screening

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

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

Phase

Not Applicable

Drug names

Primary outcome measures

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 trial start date

01/12/2013

Overall trial end date

31/05/2016

Reason abandoned

Eligibility

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 type

Patient

Age group

Child

Gender

Both

Target number of participants

32

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

01/12/2013

Recruitment end date

31/05/2016

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Dept of Respiratory and Sleep Medicine
Edinburgh
EH9 1LF
United Kingdom

Sponsor information

Organisation

Academic and Clinical Centre Office for Research and Development (ACCORD) (UK)

Sponsor details

University of Edinburgh & NHS Lothian
The Queen’s Medical Research Institute
47 Little France Crescent
Edinburgh
EH16 4TJ
United Kingdom

Sponsor type

Research organisation

Website

http://www.accord.ed.ac.uk/

Funders

Funder type

Government

Funder name

European Commission Directorate-General for Research and Innovation, FP7-Health-2012-Innovation-1

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Funder name

Funding ref nr 305653

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes