Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
NCT ID: NCT04997811
Last Updated: 2023-10-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2
120 participants
INTERVENTIONAL
2021-12-21
2025-06-30
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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VBaP
Combination of sodium valproate, bezafibrate, medroxyprogesterone
Sodium Valproate, Bezafibrate, Medroxyprogesterone
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)
Danzol
Single agent
Danazol
Danazol 1 x 200mg capsules tds, (starting 1 x 200mg od)
Interventions
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Sodium Valproate, Bezafibrate, Medroxyprogesterone
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)
Danazol
Danazol 1 x 200mg capsules tds, (starting 1 x 200mg od)
Eligibility Criteria
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Inclusion Criteria
2. Age ≥ 18 years and able to give informed consent
3. Diagnosis of Myelodysplastic Syndrome with an IPSS-R score of less than or equal to 3.51
4. Haematological parameters:
1. Mean haemoglobin \< 100 g/l over 16 weeks (pre transfusion) OR
2. Mean platelets \< 100 x 109/l over 16 weeks + evidence of bleeding (assessed using the ISTH Bleeding Assessment Tool) OR
3. Mean neutrophils \< 1.0 x 109/l over 16 weeks + history of infection (the requirement for antimicrobial therapy and hospital admissions associated with infection)
5. 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).
6. ECOG performance status 0-3
7. Expected survival \> 12months
Exclusion Criteria
12\. 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
18 Years
ALL
No
Sponsors
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Blood Cancer UK
UNKNOWN
Dudley Group NHS Foundation Trust
OTHER
University of Birmingham
OTHER
University of Manchester
OTHER
King's College Hospital NHS Trust
OTHER
Prof. Janet Dunn
OTHER
Responsible Party
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Prof. Janet Dunn
Professor of Clinical Trials
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Related Links
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REPAIR-MDS Trial Website
Other Identifiers
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2020-005446-42
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
SOC.11/20-21
Identifier Type: -
Identifier Source: org_study_id
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