Plain English Summary
Background and study aims
People with sickle cell disease (SCD) were unlikely to survive childhood in the past but treatment has improved. There are new challenges. It is known that ageing groups of SCD patients in the UK will have complications such as raised pressure in the lungs (pulmonary hypertension) and the same will happen in developing countries. Early intensive treatment of SCD (involving exchange transfusion and drugs designed to increase haemoglobin levels, such as 5-HU) may prevent such complications. The aim of the study is to assess this, by using exercise magnetic resonance imaging and echocardiography in patients undergoing intensive conventional therapy for SCD.
Who can participate?
All adults 18 years of age and older with homozygous SCD.
What does the study involve?
The study will include 3 visits over 12 months. Each visit is expected to last no more than 60 minutes.
Visit 1: patients will be asked to do a symptom limited exercise which will normally last no more than 10 minutes. They will have the following tests: cardiac magnetic resonance scan with exercise (cMR augmented CPEX), exercise echocardiography (ECHO) and Chester Step test with respiratory gas analysis (CPEX).
Visit 2 (after 6 months): ECHO and CPEX
Visit 3 (after 12 months): all tests
The data acquired at rest and during the exercise will be compared at each visit and across the duration of study.
What are the possible benefits and risks of participating?
This study will help the understanding of the effects of sickle cell disease on the heart and lungs of patients with this condition. Magnetic resonance and echocardiography have no proven side effect (no ionising radiation).
Where is the study run from?
This study is run from the University College London Hospital (UCLH), UK.
When is the study starting and how long is it expected to run for?
Recruitment starts on 13th January 2014 and is expected to run for 1 year.
Who is funding the study?
Biomedical Research Centre (BRC) Cardiometabolic programme (UK)
Who is the main contact?
Dr Emmanuel Ako; emmanuel.akoh@uclh.nhs.uk
Dr Malcolm Walker; malcolm.walker@ucl.ac.uk
Trial website
Contact information
Type
Scientific
Primary contact
Dr Malcolm Walker
ORCID ID
Contact details
The Hatter Cardiovascular Institute
67 Chenies Mews
London
WC1E 6HX
United Kingdom
+44 (0)20 3447 9951
m.walker@ucl.ac.uk
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
N/A
Study information
Scientific title
Prospective longitudinal clinical observational study in patients with homozygous sickle cell disease (SCD) to assess if conventional therapy alters cardiopulmonary complications
Acronym
CARE-SCD
Study hypothesis
Intensive conventional therapy can prevent the development of cardiopulmonary complications.
Ethics approval
West London & Gene Therapy Advisory Committee (GTAC) Health Research Authority, ref: 13/LO/1893
Study design
Prospective longitudinal clinical observational study
Primary study design
Observational
Secondary study design
Longitudinal study
Trial setting
Hospitals
Trial type
Diagnostic
Patient information sheet
Not available in web format, please use the contact details to request a patient information sheet
Condition
Sickle cell disease
Intervention
Conventional therapy including hydroxyurea and exchange transfusion. This includes the use of 5-hydroxyurea or exchange transfusions as prescribed by haematology team caring for the patient. This is standard care for sickle cell patients. Patients will be followed up under haematology routinely but have cardiac scans at baseline, 6 months and 12 months.
Intervention type
Other
Phase
Not Applicable
Drug names
Primary outcome measure
1. Cardiac Magnetic Imaging can be used to assess pulmonary vascular parameters in SCD patients
2. The pulmonary vascular responses to exercise are abnormal in patients with SCD
Timepoints of measurements: 0 months (baseline) and 12 months (end)
Secondary outcome measures
Treatment of SCD with transfusion therapy and 5-hydoxyurea, designed to reduce the proportion of sickle red cells, alter vascular responses to exercise
Timepoints of measurements: 0 months (baseline), 6 months (mid) and 12 months (end)
Overall trial start date
13/01/2014
Overall trial end date
18/01/2016
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. Patients with a diagnosis of homozygous sickle cell disease
2. Aged 18 and over
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
30 patients with homozygous sickle cell disease
Participant exclusion criteria
1. Age outside inclusion criteria
2. Impaired left ventricle (LV) function
3. Valvular abnormalities
4. Sickle cell crisis within 2 weeks of recruitment.
5. Acute chest syndrome within 4 weeks of recruitment
6. Principal exclusion criteria to perform an MR scan:
6.1. Permanent pacemaker
6.2. Intracerebral aneurysm clip
6.3. Pregnancy
7. Physical diabilities which don't permit the patient to ride on a bike
8. Principal exclusion criteria to perform a cardiopulmonary exercise test:
8.1. Myocardial infarction (35 days)
8.2. Syncope
8.3. Uncontrolled heart failure
8.4. Uncontrolled Asthma
8.5. Respiratory failure
8.6. Resting saturations <85%
8.7. Uncontrolled arrythmias
8.8. Active endocarditis, myocarditis, pericarditis
Recruitment start date
13/01/2014
Recruitment end date
13/01/2015
Locations
Countries of recruitment
United Kingdom
Trial participating centre
The Hatter Cardiovascular Institute
London
WC1E 6HX
United Kingdom
Sponsor information
Organisation
University College London (UK)
Sponsor details
c/o Mr David Wilson
Gower Street
London
WC1E 6BT
United Kingdom
+44 (0)20 3447 5199
david.wilson@ucl.ac.uk
Sponsor type
University/education
Website
Funders
Funder type
Research organisation
Funder name
Biomedical Research Centre (BRC) (UK), Ref: BRC72/CM/MW5982
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
Basic results (scientific)
Publication list