Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
REPAIR-MDS
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
2 other identifiers
interventional
120
1 country
19
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Dec 2021
Typical duration for phase_2
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 23, 2021
CompletedFirst Posted
Study publicly available on registry
August 10, 2021
CompletedStudy Start
First participant enrolled
December 21, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 31, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2025
CompletedOctober 3, 2023
October 1, 2023
3.1 years
July 23, 2021
October 2, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
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.
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.
12 months
Secondary Outcomes (4)
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
Duration of haematological response
12 months
Reported improved Health Related Quality of Life scores in each arm and in the trial overall.
12 months
Overall survival
Through study completion, an average of 1 year
Study Arms (2)
VBaP
EXPERIMENTALCombination of sodium valproate, bezafibrate, medroxyprogesterone
Danzol
EXPERIMENTALSingle agent
Interventions
Sodium valproate tablet 1 x 500mg bd, (starting 1 x 200mg bd) Bezafibrate standard release tablet 2 x 200mg tds, (starting 1 x 200mg tds) Medroxyprogesterone acetate tablet 1 x 400mg bd (starting 1 x 400mg od)
Eligibility Criteria
You may qualify if:
- Provision of written informed consent
- Age ≥ 18 years and able to give informed consent
- Diagnosis of Myelodysplastic Syndrome with an IPSS-R score of less than or equal to 3.51
- Haematological parameters:
- Mean haemoglobin \< 100 g/l over 16 weeks (pre transfusion) OR
- Mean platelets \< 100 x 109/l over 16 weeks + evidence of bleeding (assessed using the ISTH Bleeding Assessment Tool) OR
- Mean neutrophils \< 1.0 x 109/l over 16 weeks + history of infection (the requirement for antimicrobial therapy and hospital admissions associated with infection)
- No response to Erythroid Stimulating Agents (ESAs) OR Have Ceased to respond to ESAs OR are predicated not to respond to ESAs by current UK guidelines2,3 (NB Patients with thrombocytopenia and/or neutropenia, without anaemia, are eligible as they are predicated not to respond to ESAs).
- ECOG performance status 0-3
- Expected survival \> 12months
You may not qualify if:
- \. Currently receiving Vitamin K-Antagonist Anticoagulation (though patients receiving DOACs (direct oral anticoagulants) can be included) 13. History of Venous Thrombo-Embolism (VTE) 14. Cardiac Failure NYHA Class III or IV 15. Women of childbearing potential, pregnant or lactating 16. The physician or patient consider VBaP or danazol to be inappropriate for the patient 17. Known HIV 18. Abnormally high CK level 19. Presence of isolated del 5q 20. Acute Porphyria 21. Contraindications to any of the trial medications or known hypersensitivity to any of the investigational products (see Appendix C for contraindications) 22. Previous randomisation in the REPAIR-MDS trial 23. Participation in a clinical trial of an investigational medicinal product in the last 16 weeks
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Prof. Janet Dunnlead
- Blood Cancer UKcollaborator
- Dudley Group NHS Foundation Trustcollaborator
- University of Birminghamcollaborator
- University of Manchestercollaborator
- King's College Hospital NHS Trustcollaborator
Study Sites (19)
Heartlands Hospital
Birmingham, Bordesley Green East, B9 5SS, United Kingdom
Royal Cornwall Hospital NHS Trust
Truro, Cornwall, TR1 3LJ, United Kingdom
University Hospitals Dorset NHS Foundation Trust
Poole, Dorset, BH15 2JB, United Kingdom
Russells Hall Hospital
Dudley, England, DY1 2HQ, United Kingdom
University College London Hospitals NHS Foundation Trust
London, England, NW1 2PG, United Kingdom
King's College Hospital NHS Foundation Trust
London, England, SE5 9RS, United Kingdom
Broomfield Hospital
Chelmsford, Essex, CM1 7ET, United Kingdom
Colchester General Hospital
Colchester, Essex, CO4 5JL, United Kingdom
Basingstoke and North Hampshire Hospital,
Basingstoke, Hampshire, RG24 9NA, United Kingdom
Royal Hampshire County Hospital, Hampshire Hospitals NHS Foundation Trust
Winchester, Hampshire, SO22 5DG, United Kingdom
East Kent Hospitals University Foundation Trust
Canterbury, Kent, CT1 3NG, United Kingdom
James Paget University Hospitals NHS Foundation Trust
Gorleston-on-Sea, Norfolk, NR31 6LA, United Kingdom
Nottingham City Hospital, Nottingham University Hospitals NHS Trust, Hucknall Road
Nottingham, Nottinghamshire, NG5 1PB, United Kingdom
Grampian Health Board
Aberdeen, Scotland, AB15 6RE, United Kingdom
Good Hope Hospital
Birmingham, Sutton Coldfield, B75 7RR, United Kingdom
Hull University Teaching Hospitals
Hull, HU3 2JZ, United Kingdom
Leicester Royal Infirmary, University Hospitals of Leicester NHS Trust
Leicester, LE1 5WW, United Kingdom
James Cook University Hospital, South Tees Hospitals NHS Foundation Trust
Middlesbrough, TS4 3BW, United Kingdom
Royal Gwent Hospital, Aneurin Bevan University Health Board
Newport, NP18 3XQ, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Professor of Clinical Trials
Study Record Dates
First Submitted
July 23, 2021
First Posted
August 10, 2021
Study Start
December 21, 2021
Primary Completion
January 31, 2025
Study Completion
June 30, 2025
Last Updated
October 3, 2023
Record last verified: 2023-10