Condition category
Haematological Disorders
Date applied
Date assigned
Last edited
Retrospectively registered
Overall trial status
Recruitment status
No longer recruiting

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;
Dr Malcolm Walker;

Trial website

Contact information



Primary contact

Dr Malcolm Walker


Contact details

The Hatter Cardiovascular Institute
67 Chenies Mews
United Kingdom
+44 (0)20 3447 9951

Additional identifiers

EudraCT number number

Protocol/serial number


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



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


Secondary study design

Cohort study

Trial setting


Trial type


Patient information sheet

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


Sickle cell disease


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



Not Applicable

Drug names

Primary outcome measures

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

Time points 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

Time points of measurements: 0 months (baseline), 6 months (mid) and 12 months (end)

Overall trial start date


Overall trial end date


Reason abandoned


Participant inclusion criteria

1. Patients with a diagnosis of homozygous sickle cell disease
2. Aged 18 and over

Participant type


Age group




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


Recruitment end date



Countries of recruitment

United Kingdom

Trial participating centre

The Hatter Cardiovascular Institute
United Kingdom

Sponsor information


University College London (UK)

Sponsor details

c/o Mr David Wilson
Gower Street
United Kingdom
+44 (0)20 3447 5199

Sponsor type




Funder type

Research organisation

Funder name

Biomedical Research Centre (BRC) (UK), Ref: BRC72/CM/MW5982

Alternative name(s)

Funding Body Type

Funding Body Subtype


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