ISRCTN - ISRCTN63190358: Cardiopulmonary Assessment at Rest and during Exercise in patients with Sickle Cell Disease

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

Cohort study

Trial setting

Other

Trial type

Diagnostic

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

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 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

13/01/2014

Overall trial end date

18/01/2016

Reason abandoned

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