1. SUB-TYPE OF MDS: Low risk MDS patients
2. SEVERITY OF MDS: IPSS Low or Intermediate-1 risk MDS
3. NAME OF DRUG: Imetelstat
4. Aims and benefits: This phase 2/3 study aims to determine the effectiveness of Imetelstat in transfusion dependent low risk MDS patients who have either lost their response or not responded to erythropoiesis-stimulating agent (ESA) treatment. The study has 2 parts. Part 1 seeks to determine the effectiveness and safety of Imetelstat, looking more closely at improvement in blood counts and reduction in transfusion requirements. Part 2 is double-blind and randomised to compare the effectiveness of Imetelstat with placebo.
5. Primary outcome:To determine the percentage of patients who have not required a red cell transfusion in an 8 week period.
6. Secondary outcome:To determine adverse effects, red cell transfusion independence, improvement in blood counts, progression to acute myeloid leukaemia and overall survival.

1. Basic inclusion criteria:
   • Diagnosis of MDS with IPSS Low or Intermediate-1 risk
   • Red blood cell transfusion dependent
   • ECOG performance status 0, 1 or 2
2. Basic exclusion criteria:
   • Received and investigational drug or device within 30 days of study entry or enrolled in an investigational study
   • Prior treatment with Imetelstat
   • Prior treatment with a hypomethylating agent, Lenalidomide, an erythropoiesis-stimulating agent (ESA) or any chemotherapy, immunomodulatory, or immunosuppressive therapy within 4 weeks prior to study entry
3. Trial sites/locations and name of physician in charge of trial (none are currently recruiting):
   • Nottingham University Hospitals NHS Trust
   • Aberdeen Royal Infirmary
   • The Leeds Teaching Hospitals NHS Trust
   • Guy's Hospital, London
   • Southampton University Hospital

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