1. SUB-TYPE OF MDS: All patients with MDS (but see basic exclusion criteria below)
2. SEVERITY OF MDS: Intermediate or High Risk MDS and Acute myeloid leukaemia.
3. NAME OF DRUG: PDR001, MBG453. PDR001 is an antibody that makes cancer cells recognizable to the immune system thus resulting in reduction of tumour growth and size. MBG453 is an antibody that blocks a marker on leukaemia cells (TIM-3) thus making them more vulnerable to the effect of drugs used in leukaemia treatment.
4. Aims and benefits: This Phase I study investigates different doses of the drugs PDR001 and/or MBG453 to determine the dose which is safe, well tolerated and effective. It investigates the drugs when used on their own as well as in combination with each other and in combination with Decitabine or Azacitidine.
5. Primary outcome: To identify toxicities that occur due to dosage of the study drugs. To determine the safety of the study drugs when used on their own and in combination.

• Basic inclusion criteria:
  • Relapsed/refractory AML patients deemed unsuitable for standard therapy.
  • Newly diagnosed AML suitable for treatment with Decitabine or Azacitidine.
  • Intermediate or high risk MDS or MDS/MPN including CMML unsuitable for standard chemotherapy. This includes patients who have failed hypomethylating agent therapy.
  • ECOG performance status of ≤ 2.
• Basic exclusion criteria:
  • Patients who have received prior hypomethylating agent treatment for AML or MDS.
  • Patients with active, known or suspected autoimmune disease.
  • Interstitial lung disease or pneumonitis.
• Trial sites/locations and name of physician in charge of trial (none are currently recruiting):
  • Novartis Investigative Site, Cardiff, United Kingdom, CF4 4XN

If you think this information is relevant to you, please discuss it with your own physician.