Repair-MDS

• Research type

  Research Study

• Full title

  Repurposed drugs to improve haematological responses in Myelodysplastic Syndromes (REPAIR-MDS)

• IRAS ID

  1003603

• Contact name

  Stephen Jenkins

• Contact email

  stephen.jenkins2@nhs.net

• Sponsor organisation

  University of Warwick

• Eudract number

  2020-005446-42

• Research summary

  Our proposed clinical trial – REPAIR-MDS – seeks to repurpose existing drugs in order to dramatically improve the outlook, health and quality of life of people with a debilitating and progressive blood cancer called myelodysplastic syndromes (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 will be a significant breakthrough in UK medical research as the first randomised trial delivered in this neglected patient group, establishing the precedent for future trials.
  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 the even more aggressive blood cancer: acute myeloid leukaemia (AML). The general outlook for AML is poor, but when AML arises from MDS it’s even worse. At diagnosis, MDS patients are categorised according to the likelihood of developing AML or dying very early for other reasons. This determines their treatment plan. Groups are termed: low risk, intermediate risk or high risk. High risk patients are often eligible for AML trials. However, very few trials are available for ‘low’ risk patients. Low risk patients have a 20-35% risk of developing AML within five years of diagnosis. Meanwhile their quality of life is poor, due to low blood counts. Most die prematurely from infections, or complications related to MDS irrespective of whether they progress to AML. Our proposed trial will be in low risk, and some intermediate risk patients, and will test the ability of already existing drugs to improve bloo dcell production in these patients.

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  21/ES/0037

• Date of REC Opinion

  13 May 2021

• REC opinion

  Further Information Favourable Opinion