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. If you'd like more information about what a clinical trial is and how it is conducted, please go to this section.

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 - and to the UK MDS Forum  for their continuous work in updating this listing.

Learn more about the latest MDS research

MDS Clinical Trials

MONOCLE

NAME OF DRUG: Tefinostat, a histone deacetylase inhibitor that targets monocytes. Tefinostat will be administered orally on a continuous basis for at least 6 cycles each 28 days in length. WHAT SUBTYPE OF MDS: Chronic myelomonocytc leukaemia (CMML) FOR WHAT LEVEL OF MDS SEVERITY RISK: CMML specific prognostic score (CPSS) Intermediate-2 or high risk Read […]

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Durvalumab following failure of Azacitidine or Decitabine

Name of trial drug: Oral Azacitidine plus Durvalumab, which activates the immune system to create a response against cancer/tumor cells For what level of MDS severity risk: Intermediate –2 or High Risk What subtype of MDS: Patients who failed to respond to azacitidine or decitabine(SCT) Read More

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Durvalumab in Previously Untreated Patients

Investigational drugs: Subcutaneous Azacitidine and Durvalumab, which activates the immune system to have a response against tumor cells. For what level of MDS severity risk: High risk; also elderly patients (age ≥ 65 years) with Acute Myeloid Leukaemia What subtype of MDS: Intermediate –2 or High Risk Read More

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Venetoclax in Combination With Azacitidine

Investigational drugs: Venetoclax (a drug that encourages the death of cancer cells by blocking a marker on the cells called BCL-2) plus Azacitidine. For what level of MDS severity risk: High risk What subtype of MDS: Intermediate –2 or High Risk patients who have never had treatment for MDS Read More

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CATAPULT

Investigational drugs: Gene modified WT1 (Wilms’ Tumor 1) TCR therapy – Patient’s white blood cells (T cells) will be modified by transferring a gene which enables them to make a new T cell receptor (TCR) that can recognize fragments of a protein called WT1 which is present at abnormally high levels on the surface of myelodysplastic […]

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CAMELLIA

Investigational drugs: Anti-CD47 monoclonal antibody, Hu5F9-G4 (target therapy that blocks the growth of cancer cells) For what level of MDS severity risk: What subtype of MDS: Patients with Acute Myeloid Leukemia that has returned after a period of improvement (relapsed) or has not responded to previous treatment (refractory). Read More

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REDDS

Investigational intervention: Blood transfusion support For what level of MDS severity risk: All What subtype of MDS: All Read More

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LI-1

Name of trial drug: AC220 (Quizartinib), Ganetespib, Tosedostat, Selinexor, Lenalidomide and other novel therapies that will become available throughout the course of the study. These are all target treatments that block various pathways in the proliferation or growth of leukaemia cells. For what level of MDS severity risk: High (> 10% bone marrow blasts); also acute […]

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AML 19

Name of trial drug: Mylotarg, CPX-351 (a novel drug which combines two anti-cancer drugs – cytarabine and daunorubicin in a new formulation) For what level of MDS severity risk: High (> 10% bone marrow blasts); also acute myeloid leukaemia What subtype of MDS: Intermediate – 2 or High Risk Read More

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