Condition category
Haematological Disorders
Date applied
03/06/2014
Date assigned
18/07/2014
Last edited
26/08/2015
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Background and study aims
Sickle cell disease (SCD) is an inherited blood disorder where the red blood cells do not develop normally. Instead of being flexible and disc-shaped like normal blood cells, they are rigid and shaped like a crescent (or sickle). They also contain defective haemoglobin, the iron-rich protein that enables red blood cells to carry oxygen. This results in lower than normal oxygen levels in the tissues and organs of the body and can cause people to feel lethargic (lack of energy), tired and breathless, particularly after exercise. Many complications of the condition are thought to be caused by low daytime and night time oxygen levels. These can be made worse if the patient also suffers from obstructive sleep apnoea (OSA) where the muscles and soft tissues in the windpipe relax and collapse during sleep, causing a total blockage of the airway; this results in an extra dip in oxygen levels during sleep. Overnight oxygen is already used in patients with chronic lung disease who suffer from low oxygen levels during sleep. Automatic positive airway pressure (APAP) machines, which work by blowing air at a pressure that keeps the windpipe open, are used successfully for OSA patients of all ages. It is possible that these treatments may also help people suffering from SCD. Early studies have shown that SCD patients do find overnight oxygen safe and easy to use. There is also some evidence that APAP can be beneficial to children with SCD, resulting in improved attention span and fewer crises. However, further work is needed to decide whether the inconvenience of these treatments outweigh any benefits from using them for treating dips in night time oxygen levels for SCD patients. The aim of this small scale (pilot) study is to find out which intervention (the overnight oxygen or APAP) is the most acceptable for patients by asking them to use them for one week each and then have an interview describing their experience. The most acceptable intervention will then be used for further study.

Who can participate?
English speaking SCD patients aged over 8 years and who are able to (or their parents are able to) use a smart phone.

What does the study involve?
Participants are first asked to fill in a daily pain diary for a week. They are then are randomly allocated to receive one of the two interventions. A sleep physiotherapist sets up the intervention and shows the participant how to use it. The participant them uses the intervention for a week, filling in their pain diary daily and will have phone interviews to discuss any symptoms suffered. After the seven day treatment, the intervention is stopped and the participant attends hospital for a review, which includes having blood and urine tests taken, and having their lung function and oxygen levels measured. Afterwards, the participant continues to fill in their pain diary for another week before repeating the procedure with the second intervention.

What are the possible benefits and risks of participating?
Participants may find that their condition improves from one or both interventions given in this trial. If this proves to be the case, we will leave the machine that provides the most benefit with the patient (with approval from their doctor). As regards to risks, some people may find the tests to check lung function uncomfortable and may make them feel dizzy. Blood tests can also be slightly painful. Overnight APAP and oxygen therapies may also be uncomfortable and inconvenient to the participant. There is evidence that higher doses of oxygen can stop new red blood cells from being made, but not with the doses and timing of the oxygen being used for this study.

Where is the study run from?
University Hospital, Southampton (UK)
Guy's and St Thomas' NHS Foundation Trust (UK)
UCL Institute of Child Health (UK)

When is the study starting and how long is it expected to run for?
July 2014 to December 2014

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

Who is the main contact?
Professor Fenella Kirkham
Fenella.Kirkham@ucl.ac.uk

Trial website

Contact information

Type

Scientific

Primary contact

Prof Fenella Kirkham

ORCID ID

Contact details

University Hospital Southampton
Child Health
Tremona Road
Southampton
SO16 6YD
United Kingdom
+44 (0)23 8120 4765
Fenella.Kirkham@ucl.ac.uk

Additional identifiers

EudraCT number

2014-002181-67

ClinicalTrials.gov number

Protocol/serial number

RHMCHI0706

Study information

Scientific title

Prevention Of Morbidity in Sickle cell disease 2a - pilot phase (POMS 2a): Improvement of pain and quality of life in patients with sickle cell disease with nocturnal oxygen therapy or auto-adjusting continuous positive pressure: pilot phase

Acronym

POMS

Study hypothesis

The principal research question is to assess whether APAP or overnight oxygen therapy is more acceptable to patients and to assess whether there are any physiological effects of either therapy. This information will influence the choice of intervention in a second, proof of concept trial.

Ethics approval

NRES Committee East of England - Cambridge South; 03/06/2014; ref. 14/EE/0163

Study design

Randomised controlled trial. Block randomisation by an independent statistician at RDS Southampton.

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Quality of life

Patient information sheet

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

Condition

Sickle Cell Disease

Intervention

Participants will have two interventions, APAP and overnight oxygen, for a week each in randomized order (week 2 and 4). There will be a week of baseline data collection (week 1), and a week of washout between the interventions (week 3).

Intervention type

Other

Phase

Not Applicable

Drug names

Primary outcome measures

Patient feasibility, acceptability and preference will be explored using qualitative interviews

Secondary outcome measures

1. Pain: Using available Smartphone technology, information on:
1.1. Pain characteristics (intensity, location, quality)
1.2. Pain medications and non-pharmacological strategies used for pain
1.3. Healthcare visits will be collected in the pilot to test out the methodology and to determine whether there is any obvious effect of either intervention or its withdrawal, particularly if detrimental. This is important as the first outcome measure for trial 2b will be average pain intensity during the two observation periods.
2. Adverse events: The Clinical Report Forms (CRFs) for reporting adverse events will be trialed during this pilot to show efficacy in recording and reporting adverse events.
3. Daytime oxygen saturation will be collected before and after each intervention to determine whether there is any obvious effect of either intervention or its withdrawal, particularly if detrimental.
4. Lung volume will be collected before and after each intervention to determine whether there is any obvious effect of either intervention or its withdrawal, particularly if detrimental.

Overall trial start date

01/07/2014

Overall trial end date

01/12/2014

Reason abandoned

Eligibility

Participant inclusion criteria

1. Age over 8 years
2. Informed consent with assent in accordance with the institutional policies (UK ethical committee) and European or US Federal guidelines
3. Sickle cell haemoglobin (HbSS) diagnosed by standard techniques
4. Able to speak and understand English
5. Patient or parent/guardian able to use smart phone

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

20

Participant exclusion criteria

1. Patient already on overnight respiratory support or has used it in the past
2. Hospital admission for acute sickle complication within the past 1 month
3. Patient with > 6 admissions for acute sickle complications within the past 12 months
4. Existing Respiratory Failure
5. Decompensated Cardiac Failure
6. History of Severe Epistaxis
7. Transsphenoidal

Recruitment start date

01/07/2014

Recruitment end date

01/12/2014

Locations

Countries of recruitment

United Kingdom

Trial participating centre

University Hospital Southampton
Southampton
SO16 6YD
United Kingdom

Trial participating centre

Guy's and St Thomas' NHS Foundation Trust

London
-
United Kingdom

Trial participating centre

UCL Institute of Child Health
London
-
United Kingdom

Sponsor information

Organisation

University Hospital Southampton NHS Foundation Trust (UK)

Sponsor details

R&D Office
E Level
Southampton Centre for Biomedical Research
Southampton General Hospital
Tremona Road,Southampton
Southampton
SO16 6YD
United Kingdom
+44 (0)23 8120 4765
sharon.atwill@uhs.nhs.uk

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

National Institute for Health Research (NIHR) (UK) - CCR- RfPB grant

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

2015 protocol in: http://www.ncbi.nlm.nih.gov/pubmed/26303626

Publication citations

Additional files

Editorial Notes