MACS Study - Microparticles and Coagulation in Sickle Cell Disease

NCT01242878 | Unknown

• 1 other identifier: 10/0270
• Study Type: observational
• Target: 360
• Locations: 1 country
• Sites: 3

Brief Summary

Sickle cell disease (SCD) is an inherited disorder of the red blood cell. It is now the commonest genetic disorder in the UK and of childhood stroke, with up to 40% of children having a stroke (clinical or picked up on a scan) by school age. Patients are prone to develop acute crises necessitating hospital admission and resulting in long-term complications. Such events result in considerable morbidity, disability and mortality with its consequent burden on patients, families, the health service and society as a whole. Doctors have very little ability to predict who will get ill and when and so it is very difficult to known when and how to administer treatments. Furthermore there is very little in the way of treatments available and the mainstay of prevention is a chronic blood transfusion programme which is expensive, requires time off work and school and can be fraught with complications. This, in a population who is frequently educationally and socially disadvantaged at the outset. Recent evidence in sickle cell disease and other diseases that have similar underlying processes, points towards the importance of microparticles (circulating broken pieces of cells) and the coagulation system as being important. By comparing levels of these particles and molecules in patients with those found in healthy volunteers and with other measures known to be important, this study hopes to identify their role so as to improve the management of these patients and potentially to lead the way for new therapies. Participants will be required to donate a small amount of blood (1 teaspoon in the very young, two in older children and adults). The investigators aim to take this sample where possible when people are having a blood test in any case.

Conditions

Sickle Cell Disease

Keywords

HbSS; Sickle cell anemia; HbSC; HbSbetathalassemia; Hemoglobinopathy; Hemoglobinopathies

Study Arms (2)

healthy volunteers

ethnically matched people who do not have sickle cell disease

patients

sickle cell disease patients

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This is an observational case-control study in adults and children with sickle cell disease. The cases are any child or adult attending the study centres with a confirmed diagnosis of sickle cell disease that meet the eligibility criteria. The controls are ethnically matched volunteers who meet the appropriate eligibility criteria for controls and who do not have sickle cell disease and will be recruited from the same study sites.

You may qualify if:

• Patient (or parent if child) able to give informed consent Compound heterozygote or homozygote for a sickling disorder (i.e. has sickle cell disease) Having a blood test in any case
• Person (or parent if child) able to give informed consent If a child, having a blood test or cannula inserted in any case Ethnically matched - of African origin Must know their sickle cell status or have it tested as part of the study and agree to have this result given to them and their G.P.

Sponsors & Collaborators

• University College, London: lead
• Guy's and St Thomas' NHS Foundation Trust: collaborator
• The Whittington Hospital NHS Trust: collaborator

Study Sites (3)

Whittington Hospital NHS Trust

London, N19 5NF, United Kingdom

Location

University College London Hospitals NHS Foundation Trust

London, NW1 2PJ, United Kingdom

Location

Guy's and St Thomas' NHS Foundation Trust

London, SE1 7EH, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

plasma will be retained following appropriate informed written consent for further ethically approved studies as stipulated on the consent forms

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinopathies

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• John B Porter, MB BChir FRCPath MD

  University College London and University College London NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

• Baba Inusa, MB BS FRCPCH

  Guy's and St Thomas' NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

• Bernard A Davis, MD FRCPath

  Whittington Hospital NHS Trust

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sara Trompeter, MB ChB BSC MRCPCH FRCPath

CONTACT

sara.trompeter@nhs.net

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: November 16, 2010
• First Posted: November 17, 2010
• Study Start: December 1, 2010
• Last Updated: November 18, 2010

Record last verified: 2010-11

Locations

GB (3)