Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
- Conditions
- Myelodysplastic Syndromes (MDS)
- Interventions
- Drug: Sodium Valproate, Bezafibrate, Medroxyprogesterone
- Registration Number
- NCT04997811
- Lead Sponsor
- Prof. Janet Dunn
- 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
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 120
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description VBaP Sodium Valproate, Bezafibrate, Medroxyprogesterone Combination of sodium valproate, bezafibrate, medroxyprogesterone Danzol Danazol Single agent
- Primary Outcome Measures
Name Time Method Haematological improvement (HI) in each arm and in the trial overall, with 25% or more of the participants having HI in each arm and overall. 12 months HI will be assessed in each participant by comparing post randomisation FBC parameters (Haemoglobin, platelet and neutrophil counts) and transfusion requirements, with their individual baseline as determined by the IWG 2018 haematology response criteria in patients with MDS.
- Secondary Outcome Measures
Name Time Method Duration of haematological response 12 months Clinically meaningful haematological responses that persist for 16 weeks or longer.
Overall survival Through study completion, an average of 1 year Overall survival at close of trial will be reported separately for each trial arm
Reduced burden of red cell and/or platelet transfusion in each arm and in the trial overall, as per the IWG 2018 response criteria. 12 months Changes in transfusion requirements will be assessed in each participant by comparison with their individual 16-week lead-in baseline as determined by the IWG 2018 haematology response criteria in patients with MDS.
Reported improved Health Related Quality of Life scores in each arm and in the trial overall. 12 months The four Health Related Quality of Life questions measure 1) self-perceived health (excellent, very good, good, fair, or poor), (2) number of days out of the past 30 that physical health was not good, (3) number of days out of the past 30 that mental health was not good, and (4) number of days out of the past 30 that usual activities were limited by poor physical or mental health of life scores will be evaluated using established protocols.
Trial Locations
- Locations (19)
Royal Hampshire County Hospital, Hampshire Hospitals NHS Foundation Trust
🇬🇧Winchester, Hampshire, United Kingdom
Nottingham City Hospital, Nottingham University Hospitals NHS Trust, Hucknall Road
🇬🇧Nottingham, Nottinghamshire, United Kingdom
Royal Cornwall Hospital NHS Trust
🇬🇧Truro, Cornwall, United Kingdom
Colchester General Hospital
🇬🇧Colchester, Essex, United Kingdom
East Kent Hospitals University Foundation Trust
🇬🇧Canterbury, Kent, United Kingdom
Broomfield Hospital
🇬🇧Chelmsford, Essex, 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
Basingstoke and North Hampshire Hospital,
🇬🇧Basingstoke, Hampshire, United Kingdom
James Paget University Hospitals NHS Foundation Trust
🇬🇧Gorleston-on-Sea, Norfolk, United Kingdom
Grampian Health Board
🇬🇧Aberdeen, Scotland, United Kingdom
Leicester Royal Infirmary, University Hospitals of Leicester NHS Trust
🇬🇧Leicester, United Kingdom
Hull University Teaching Hospitals
🇬🇧Hull, 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
Heartlands Hospital
🇬🇧Birmingham, Bordesley Green East, United Kingdom
Good Hope Hospital
🇬🇧Birmingham, Sutton Coldfield, United Kingdom