MDS Current Trials UK

We aim to list an easily understandable description of any MDS clinical trials currently opened to recruitment in the UK.

Patients will be able to read up on the details of these trials and use this information to discuss with their consultants or nurses.

Not all patients can be enrolled in drug trials - as not every patient will fit the necessary inclusion criteria, which need to be followed very strictly. However, if a particular clinical trial drug is considered an option by the haematologist, it is sometimes possible to obtain the drug outside the trial, on the basis of compassionate use.

All the trials listed in this page have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust 

  1. SUB-TYPE OF MDS:High risk MDS and AML, not suitable for induction chemotherapy or haemopoietic stem cell transplant
  3. NAME OF DRUG: PDR001, MBG453, Decitabine
  4. Aims and benefits: PDR001 is a monoclonal antibody that functions to block the activity of PD-1 (programmed cell death protein 1) thus allowing T-cells to recognise and target cancer cells. MBG453 is a monoclonal antibody that binds to a specific protein found on the surface of T-cells thus allowing the T-cell to function normally against cancer cells by reducing tumour growth. This is a phase 1b study aimed at determining the safety and tolerability of the study drugs alone and in combination with decitabine. The study also aims to determine the recommended doses of each of these drugs.
  1. Primary outcome measures: 
    To establish the safety for the study drugs and determine the doses of the drugs that result in toxicities.
  2. Secondary outcome measures:
    To determine more information about how the drug works in the body including the rate at which the drug is spread throughout and removed from the bloodstream and also to establish response rates.
  3. Main basic exclusion criteria:
    The study has various treatment arms, each of which has specific inclusion criteria.
    • AML (de novo or relapsed/refractory) patients who are deemed unsuitable for standard induction chemotherapy or haemopoietic stem cell transplant.
    • AML patients who are suitable for decitabine therapy.
    • High risk MDS patients who are deemed unsuitable for standard induction chemotherapy or haemopoietic stem cell transplant.
    • High risk MDS patients who have failed hypomethylating agent therapy.
    • ECOG performance status of 0-2
  4. Basic exclusion criteria:
    • Patients who are suitable for standard induction chemotherapy or haemopoietic stem cell transplant and are willing to receive it.
  5. Trial sites/locations and name of the physician in charge of trial:
    • - University Hospital of Wales, Cardiff

Please read information and always discuss trial information with your own physician.

Paper of the Month

Venetoclax and hypomethylating agents (HMAs) induce high response rates in MDS, including patients after HMA therapy failure

Visit our archives

IPSS-R MDS Risk Assessment Calculator

Revised International Prognostic Scoring System (IPSS-R) for Myelodysplastic Syndromes Risk Assessment Calculator


Click here

MDS Fact Sheet

MDS Factsheet for GPs General Practicioners

MDS Fact Sheet

Click here

Varicella Vaccine in MDS

Urgent Clarification on varicella vaccine policy

Current advice from the UK MDS Forum is not to give the Live Varicella Vaccine to patients with MDS.

Please read below correspondence between Professor Salisbury and Dr George Follows, the latter on behalf of the CLL Forum, on the varicella vaccine policy.
This is an external link to the British Society for Haematology  Click here

NICE Guideline

Lenalidomide for treating myelodysplastic syndromes (MDS) associated with an isolated deletion 5q cytogenetic abnormality.

NICE logo

Click here

BSH Guidelines

This is an external link to the home of the British Society for Haematology (BSH). 

BSH Logo Strapline RGB

Click here