ASTX727-03LD

• Research type

  Research Study

• Full title

  A Randomized, Open-Label, Phase 1 2 Study of ASTX727 Low Dose (ASTX727 LD) Extended Schedule in Subjects with Lower Risk (IPSS Low or Intermediate-1) Myelodysplastic Syndromes (MDS)

• IRAS ID

  300922

• Contact name

  Paresh Vyas

• Contact email

  paresh.vyas@imm.ox.ac.uk

• Sponsor organisation

  Astex Pharmaceuticals, Inc.

• Eudract number

  2019-003281-40

• Clinicaltrials.gov Identifier

  NCT03502668

• Duration of Study in the UK

  1 years, 9 months, 30 days

• Research summary

  Myelodysplastic syndromes (MDS) are a type of rare blood cancer. There are many different types of MDS. Some types can stay mild for years whilst others are more serious. Patients often present with complications related to fatigue, infections or bleeding.

  Currently available therapies depend on the type of MDS the patient has, with the aim of treatment to get the number and type of blood cells in the bloodstream back to normal and manage symptoms. Treatment is typically given via time consuming intravenous (IV) infusion or large volume injections. The study sponsor, Astex Pharmaceuticals (Astex), have developed a new oral treatment which if successful will greatly reduce the inconvenience associated with current standard of care treatment, especially for those patients with lower or intermediate risk disease who may require and remain stable on treatment for several months or even years.
  
  The study drug being tested (ASTX727) is made up of decitabine, which blocks cancer cells from growing, and a new chemical entity called cedazuridine. Cedazuridine slows the rate at which decitabine is broken down in the body, allowing oral rather than intravenous decitabine delivery to be effective. The purpose of the present study is to evaluate the effect of various dose regimens and schedules of ASTX727 on safety and response in a lower-risk MDS population.

  In Phase 1, a total of 48 participants were enrolled and this part of the study is now closed to recruitment. No UK patients will be invited to take part in this phase.
  In Phase 2, 40 participants per regimen will be enrolled, up to a total of approximately 128 in the study.

  Participants will be recruited from approximately 40 study centres in approximately 6 countries in North America and Europe.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  21/YH/0258

• Date of REC Opinion

  17 Dec 2021

• REC opinion

  Further Information Favourable Opinion