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Using existing drugs to improve blood production in patients with Myelodysplastic Syndromes

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

Phase 2 Interventional University of Warwick · NCT04997811

This study is testing whether a combination of existing medications can help improve blood production and quality of life for people with Myelodysplastic Syndromes.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment120 (estimated)
Ages18 Years and up
SexAll
SponsorUniversity of Warwick Academic / other
Drugs / interventionschemotherapy
Locations19 sites (Birmingham, Bordesley Green East and 18 other locations)
Trial IDNCT04997811 on ClinicalTrials.gov

What this trial studies

This clinical trial, known as REPAIR-MDS, aims to repurpose existing medications to enhance the health and quality of life for individuals suffering from Myelodysplastic Syndromes (MDS). The study will compare the effects of a combination treatment called VBaP (sodium valproate, bezafibrate, medroxyprogesterone) against danazol in a multicenter, open-label, phase 2 randomized controlled format. By improving the production of healthy blood and immune cells, the trial seeks to combat the complications associated with MDS, including chronic fatigue and recurrent infections. The ultimate goal is to reduce the transformation of MDS into acute myeloid leukaemia (AML), which has a poor prognosis.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with Myelodysplastic Syndrome who have specific hematological deficiencies and have not responded to erythroid stimulating agents.

Not a fit: Patients with Myelodysplastic Syndromes who do not meet the eligibility criteria or have a very poor prognosis may not benefit from this study.

Why it matters

Potential benefit: If successful, this trial could significantly improve the health outcomes and quality of life for patients with Myelodysplastic Syndromes.

How similar studies have performed: Other studies have explored the repurposing of drugs for hematological conditions, showing promise, but this specific approach is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria (for Randomisation):

1. Provision of written informed consent
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 (For Randomisation):

Abnormal liver function (if patient has Gilbert's syndrome, then abnormal direct Bilirubin is an exclusion) 2. Cockcroft Gault CrCl \< 20ml/min 3. Current systemic treatment for low risk MDS 4. History of Allogeneic Bone Marrow Transplant 5. History of having received ESAs and/or G-CSF in the past 16 weeks 6. Currently receiving statin medication for Secondary Prophylaxis of Cardiovascular Disease, Cerebrovascular Disease or Peripheral Vascular Disease (Please note patients receiving statin medication for Primary Prophylaxis of Cardiovascular Disease - i.e. the patient has no prior history of Ischaemic Heart Disease nor Cerebrovascular Disease - can still be entered, please see section 1.4 Statin use) 7. Currently receiving fibrate medications 8. Currently receiving sodium valproate, carbamazepine or phenytoin for treatment of epilepsy 9. Prior cytotoxic chemotherapy or hypomethylating agents for AML/MDS (eg Azacitidine) 10. Concurrent active malignancy requiring treatment 11. History of any Androgen Dependent Tumour (patients with Prostate Cancer are Excluded when a biopsy proven diagnosis of Prostate Cancer has been made OR their PSA is known to be elevated OR they are on active treatment for Prostate Cancer, including hormonal therapy).

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

Where this trial is running

Birmingham, Bordesley Green East and 18 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Myelodysplastic Syndromes
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.