Symptoms and General Pathology

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Medroxyprogesterone

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Combination of 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)

Sodium Valproate, Bezafibrate, Medroxyprogesterone

Danazol 1 x 200mg capsules tds, (starting 1 x 200mg od)

Bethany Foster, BSc

Helen Higgins, MSc

Research and Development Manager

Dr Manoj Raghavan

Head of Research & Development

Dr Ruth Witherall

Head of Research

Dr Catherine Hockings

Dr Mohamed Ifraz Hamid

Dr Helen Mccarthy

Research and Development Department

Dr Stephen Jenkins

Dr Jeff Neilson

Dr Shereef Elmoanly

Dr Ovini Gamage

Contracts Manager

Dr Elspeth Payne

Research & Innovation Governance Manager

Dr Austin Kulasekararaj

Dr Pramila Krishnamurthy

Dr Victoria Potter

Research Development

Dr Pavel Kotoucek

Dr Mike Hamblin

Dr Gavin Campbell

Dr Mahalakshmi Mohan

Dr Joseph Padayatty

Dr Khalid Saja

Dr Francis Anyanwu

Research Department

Dr Noel Ryman

Dr Katherine Smith

Dr Hussan Janan

Dr Henna Wong

Dr Kanchana De Abrew

Dr Jennifer Arnold

Dr Marianna Koperdanova

Dr Katharine Lowndes

R&I/CTU Manager

Dr Sreetharan Munisamy

Dr Iresha Dharmasena

Dr Caroline Grist

Dr Jindriska Lindsay

Dr Jayne Osborne

Dr Sharath Dr Sharath

Dr Samih Salih

Dr Moya Young

Research and Development Office

Dr Thomas Mckerrell

R&I Administrator

Dr Yadanar Lwin

Dr Tom Taylor

Dr Gerardo Errico

Dr Malik Saeed

Dr Jennifer Byrne

Research and Development

Dr Dominic Culligan

Mrs Mariella Lamacchia

Dr Stephanie Bruce

Dr Paraskevi Untiveros

Dr Sophie Lee

Dr Harshini Alwis

R&D Manager

Dr Simone Green

R&I Offices

Dr Katherine Hodgson

Research Manager

Dr Matthew Horran

R&D Assistant Director

Jeanette Wells

Dr Ali Mahdi

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.

Repurposed Drugs to Improve Haematological Responses in 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

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.

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.

Clinically meaningful haematological responses that persist for 16 weeks or longer.

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.

Overall survival at close of trial will be reported separately for each trial arm

Spots Global Cancer Trial Database for sodium valproate, bezafibrate, medroxyprogesterone