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Brief Title: Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
Official Title: Evaluation of Haematological Improvement in Patients With Low-risk MDS by Comparing VBaP With Danazol in Patients Who Have Either Received Erythropoiesis Stimulating Agents (ESA) and Lost Response, Not Responded to ESA or Are Deemed Unlikely to Respond to ESA
Study ID: NCT04997811
Brief Summary: Over 7,000 people in the UK are living with Myelodysplastic Syndromes (MDS). Approximately 1,600 of these individuals (23%) die each year from their disease. MDS affects the production of blood cells by the bone marrow, causing chronic fatigue, bleeding, and recurrent infections. Many patients die because their disease transforms into acute myeloid leukaemia (AML) an even more aggressive blood cancer. The general outlook for AML is poor, but when AML arises from MDS it is worse. REPAIR-MDS seeks to repurpose existing drugs in order to dramatically improve the outlook, health and quality of life of people with MDS. The trial treatments aim to improve the production of healthy functioning blood and immune cells that will fight against infections and boost the immune system's action against the MDS clone. REPAIR-MDS design is a is a multicentre open label phase 2 randomised controlled trial which will compare VBaP (sodium valproate, bezafibrate, medroxyprogesterone) with danazol in patients who have received either Erythropoiesis Stimulating Agents (ESAs) and lost response, not responded to ESAs or are deemed unlikely to respond to ESAs.
Detailed Description:
Minimum Age: 18 Years
Eligible Ages: ADULT, OLDER_ADULT
Sex: ALL
Healthy Volunteers: No
Heartlands Hospital, Birmingham, Bordesley Green East, United Kingdom
Royal Cornwall Hospital NHS Trust, Truro, Cornwall, United Kingdom
University Hospitals Dorset NHS Foundation Trust, Poole, Dorset, United Kingdom
Russells Hall Hospital, Dudley, England, United Kingdom
University College London Hospitals NHS Foundation Trust, London, England, United Kingdom
King's College Hospital NHS Foundation Trust, London, England, United Kingdom
Broomfield Hospital, Chelmsford, Essex, United Kingdom
Colchester General Hospital, Colchester, Essex, United Kingdom
Basingstoke and North Hampshire Hospital,, Basingstoke, Hampshire, United Kingdom
Royal Hampshire County Hospital, Hampshire Hospitals NHS Foundation Trust, Winchester, Hampshire, United Kingdom
East Kent Hospitals University Foundation Trust, Canterbury, Kent, United Kingdom
James Paget University Hospitals NHS Foundation Trust, Gorleston-on-Sea, Norfolk, United Kingdom
Nottingham City Hospital, Nottingham University Hospitals NHS Trust, Hucknall Road, Nottingham, Nottinghamshire, United Kingdom
Grampian Health Board, Aberdeen, Scotland, United Kingdom
Good Hope Hospital, Birmingham, Sutton Coldfield, United Kingdom
Hull University Teaching Hospitals, Hull, , United Kingdom
Leicester Royal Infirmary, University Hospitals of Leicester NHS Trust, Leicester, , United Kingdom
James Cook University Hospital, South Tees Hospitals NHS Foundation Trust, Middlesbrough, , United Kingdom
Royal Gwent Hospital, Aneurin Bevan University Health Board, Newport, , United Kingdom