Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
Status:
Not yet recruiting
Trial end date:
2025-06-30
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Prof. Janet Dunn
Collaborators:
Blood Cancer UK Dudley Group NHS Foundation Trust King's College Hospital NHS Trust University of Birmingham University of Manchester