REDRESS V1.0
Research type
Research Study
Full title
A multi-centre open randomised controlled trial to assess the effect of related haplo-donor haematopoietic stem cell transplantation versus standard of care (no transplant) on treatment failure at 24 month in adults with severe sickle cell disease.
IRAS ID
312212
Contact name
Victoria Potter
Contact email
Sponsor organisation
King’s College Hospital NHS Foundation Trust
Duration of Study in the UK
4 years, 2 months, 30 days
Research summary
Sickle Cell Disease (SCD) is a serious inherited condition, in which red blood cells become an abnormal sickle shape under stressful conditions causing blood vessels to be easily blocked. This leads to health problems such as: sickle cell crises (painful episodes around the body), a higher risk of stroke, heart issues and many other complications.
People with SCD need treatment throughout their lives. Currently available treatments for SCD include regular blood transfusions (when blood from a donor is given to replace a patient’s red blood cells) and a medicine called hydroxycarbamide. Sometimes painkillers are given to manage painful episodes. These treatments are not sufficient because some people may not respond well or fully. Stem Cell Transplant (SCT) is a procedure that may cure SCD. This process destroys damaged blood cells and replaces them with stem cells from the blood or bone marrow of a genetically matched family donor.
Unfortunately, many patients do not have a fully matched donor available. Research has shown that SCT from haplo-identical (half-matched) family donors has the same potential to cure severe SCD, but this is only approved for use in children due to concerns about side effects in adults. New transplant protocols mean it is now possible for haploidentical SCT to be done safely in adults. This treatment is expensive, so it is important to show that this is good value for money for the NHS.
We will carry out a clinical trial to find out whether haplo-identical SCT is safe, effective, and cost worthy in treating adults with severe SCD. We will invite 120 participants; half will be assigned by chance to receive either haploidentical SCT or standard of care. We will monitor participants for 2 years to see if haploidentical SCT cures people of SCD and is good value for the NHS.
REC name
London - Chelsea Research Ethics Committee
REC reference
22/LO/0702
Date of REC Opinion
29 Nov 2022
REC opinion
Further Information Favourable Opinion