Condition category
Nutritional, Metabolic, Endocrine
Date applied
30/10/2017
Date assigned
29/11/2017
Last edited
29/11/2017
Prospective/Retrospective
Retrospectively registered
Overall trial status
Ongoing
Recruitment status
Recruiting

Plain English Summary

Background and study aims
Urea cycle disorders are a group of genetic metabolic conditions that prevents correct conversion of nitrogen to urea and, instead, produces ammonia. These patients cannot effectively control the level of ammonia in their blood. If the ammonia reaches high levels, this can cause severe illness and even death in these patients. This is a lifelong debilitating condition which is difficult to manage, as it is hard to tell when a patient is well or when they are sick. This study investigates whether a device (called the AmBeR device) for measuring ammonia in people’s breath relates to the concentration of ammonia in their blood. If successful, this device would allow a simple, painless way of monitoring these patients in their own home. As well as significantly improving their quality of life, this would also be a significant reduction in the burden on the healthcare system.

Who can participate?
This study involves children aged 5 years and younger who have a Urea Cycle Disorder or another hyper-ammonaemic condition as well as a parent or carer who is willing to help collect breath samples.

What does the study involve?
This study includes two parts: a clinic study and a home study. The clinic study asks participants to provide breath samples and to complete questionnaires on dietary intake and quality of life. The breath samples are collected using the AmBeR device. The home study involves participants collecting breath samples two-three times per day for three months using the AmBeR breath ammonia device. Patients or carers are asked to record the participants’ health status, time of last food/drink and any issues they experience using the device. Participants are followed up after three months to measure their quality of life.

What are the possible benefits and risks of participating?
There are no significant benefits or risks in taking part in this study.

Where is the study run from?
1. Bristol Royal Hospital for Children (UK)
2. Great Ormond Street Hospital for Children NHS Foundation Trust (UK)
3. Evelina London Children’s Hospital (UK)
4. Birmingham Children's Hospital (UK)

When is the study starting and how long is it expected to run for?
December 2016 to April 2019

Who is funding the study?
National Institute for Health Research (UK)

Who is the main contact?
Dr Fiona Lithander

Trial website

N/A

Contact information

Type

Public

Primary contact

Dr Fiona Lithander

ORCID ID

Contact details

University of Bristol
NIHR Bristol Biomedical Research Centre (Nutrition Theme)
University Hospitals Bristol Education & Research Centre
Upper Maudlin Street
Bristol
BS2 8AE
United Kingdom

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

35123

Study information

Scientific title

Assessment of a breath ammonia monitoring device for its potential to improve the management and care of patients with urea cycle disorders

Acronym

AmBeR UCD

Study hypothesis

The IMPRESS-Study evaluates a process-intervention on the basis of two comparing groups. This trial primarily aims to answer if mortality improves due to an organized collegial conference ("peer Review"). The aim is to find and eradicate error-potentials and improve in-house-quality.

Ethics approval

London – Riverside Research Ethics Service, 15/08/2017, ref: 17/LO/1077

Study design

Observational; Design type: Validation of outcome measures

Primary study design

Observational

Secondary study design

Cross sectional study

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

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

Condition

Specialty: Children, Primary sub-specialty: Inherited Metabolic Disorders; UKCRC code/ Disease: Metabolic and Endocrine/ Metabolic disorders

Intervention

There are two parts to this research project: Clinic Study and Home Study.

Clinic Study: Once a participant has provided written consent and assent as appropriate, the participant is asked to provide breath samples and to complete two questionnaires, one which records dietary intake and the second records quality of life. Breath samples are collected using the using the AmBeR device to measure breath ammonia. Each measurement involves a set of exhalations (typically 8) into the mouthpiece of the AmBeR device; this is similar to natural breathing and does not involve a forced exhale in older children. However, in younger children, it may require a larger than normal inhale and exhale, the same as taking a deep breath. The AmBeR device measures how much ammonia is in the breath and this will be recorded by the device. Dietary intake is measured using a 24 hour recall online tool called Intake24 and quality of life is recorded using PedSQL.

Home Study: Patients are asked to collect 2-3 breath samples, two times per day, for three months using the AmBeR breath ammonia device at home. They are asked to provide these samples in the morning and in the evening. The patient/ carer are also asked to record patient health status information, time of last food/drink and any issues with the use of the device. There will be no change in the care model used during the home evaluation period and patients, carers and clinicians act independently of any data gathered by the instruments in a manner which continues to ensure optimum care of the patient during this study. During the three month period, parents/ carers are asked to record time off work data to cater to the child’s health care needs, number and reason for hospital visits and contacts with primary care. Parents/carers are also be asked to complete a quality of life questionnaire (EQ-5D-5L) at baseline and at the three month follow up. Children are also be asked to complete a PedsQL Quality of life questionnaire at baseline and follow up. At the end of the three month period, the parent/carer will be asked if they would like to take part in a phone interview in which qualitative data will be recorded. This interview explore the parent/carer’s user experiences of the AmBeR device.

Intervention type

Device

Phase

Drug names

Primary outcome measures

Clinic Study:
Breath ammonia is measured using an ammonia breath monitor (AmBeR) at one occasion.

Home Study:
Breath ammonia is measured using an ammonia breath monitor (AmBeR) daily for a 3 month period.

Secondary outcome measures

There are no secondary outcome measures.

Overall trial start date

16/12/2016

Overall trial end date

30/04/2019

Reason abandoned

Eligibility

Participant inclusion criteria

Clinic Study: Inclusion criteria for patients with Urea Cycle Disorder and other hyper-ammonaemic conditions
1. Aged ≥ 5 years
2. Diagnosis of a UCD or other hyper-ammonaemic condition
3. Capable of providing a valid breath sample
4. Providing a blood sample as part of their clinical care

Clinic Study: Inclusion criteria for control patients
1. Patients without a UCD or another hyper-ammonaemic condition attending for a routine inborn error of metabolism (IEM) hospital appointment and providing a blood sample as part of their clinical care. An additional 1-2ml whole blood is required for the measurement of blood ammonia.
2. Aged ≥ 5 years
3. Capable of providing a valid breath sample

Home Study: Inclusion criteria (patients with Urea Cycle Disorder or other hyper-ammonaemic conditions)
1. Aged ≥ 5 years
2. Diagnosis of a UCD or other hyper-ammonaemic condition
3. Capable of providing valid breath samples
4. Family willing to help patient collect breath samples and record data

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Planned Sample Size: 126; UK Sample Size: 126

Participant exclusion criteria

Children under five years of age.

Recruitment start date

10/11/2017

Recruitment end date

28/02/2019

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Bristol Royal Hospital for Children
University Hospitals Bristol NHS Foundation Trust Upper Maudlin Street
Bristol
BS2 8BJ
United Kingdom

Trial participating centre

Great Ormond Street Hospital for Children NHS Foundation Trust
Great Ormond Street
London
WC1N 3JH
United Kingdom

Trial participating centre

Evelina London Children’s Hospital
Guy's and St Thomas' NHS Foundation Trust Westminster Bridge Road St Thomas’ Hospital
London
SE1 7EH
United Kingdom

Trial participating centre

Birmingham Children's Hospital
Birmingham Women’s and Children’s NHS Foundation Trust Steelhouse Lane
Birmingham
B4 6NH
United Kingdom

Sponsor information

Organisation

University Hospitals Bristol NHS Foundation Trust

Sponsor details

Research & Innovation
Level 3
Education and Research Centre
Upper Maudlin Street
Bristol
BS1 3NU
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

NIHR Central Commissioning Facility (CCF)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

The trialists will disseminate their research findings at national and international conferences, invited talks, at relevant stakeholder groups including CLIMB, and via original research articles in high-end peer-reviewed journals.

Relevant conferences include:
1. British Inherited Metabolic Diseases Group Annual Conference
2. International Congress of Inborn Errors of Metabolism Annual conferences

IPD sharing statement:
The datasets generated and/or analysed during the current study during this study will be included in the subsequent results publication.

Intention to publish date

30/04/2020

Participant level data

Other

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes

17/11/2017: The ISRCTN prospective/retrospective flag compares the date of registration with the recruitment start date and does not include any grace period. The registration of this study was requested through the NIHR Portfolio and was finalised within 6 weeks of the recruitment starting.