AMMO

• Research type

  Research Study

• Full title

  A randomised phase 2 study of ASTX727 versus best supportive care in MDS/MPN Overlap Syndromes

• IRAS ID

  1004656

• Contact name

  Birgit Whitman

• Contact email

  Researchgovernance@contacts.bham.ac.uk

• Sponsor organisation

  Research Strategy and Services Central

• Eudract number

  2021-004585-35

• ISRCTN Number

  ISRCTN30808508

• Research summary

  MDS/MPN overlap syndromes are rare bone marrow cancers. Patients commonly experience fatigue, infections and bleeding. Due to the age of onset, for many patients there are few effective treatments available.

  AMMO will test a drug called ASTX727 to see how effective it is at treating these diseases and the impact it has on quality of life. ASTX727 is a hypomethylating drug in tablet form.

  Extensive previous research has established that hypomethylating drugs have benefit in MDS/MPN; some have already been approved for use following successful trials. However, the current approved treatments can only be given intravenously or subcutaneously (an injection under the skin). There is also not yet enough evidence for using these drugs to treat many types of MDS/MPN overlap syndromes.

  ASTX727 was developed by a drug company in the USA and has already undergone some early phase clinical trials in similar diseases. Results have been promising, with the drug seeming to work as well as the intravenous form of decitabine (on which it is based) and with no unexpected side effects; most patients tolerated ASTX727 well. This will be the first time ASTX727 has been tested in the UK but a complementary international clinical trial is currently ongoing.

  The AMMO study is for patients with MDS/MPN overlap syndromes and will compare:
  1)Best supportive care: the treatment that patients would normally receive as standard which may include transfusions and hydroxycarbamide capsules
  2)A drug called ASTX727: this alters how genes are switched on and off by blocking a protein whose job it is to “mark” which genes should be switched on in any particular cell. In this way we hope it can improve blood counts and symptoms

  The aim of the AMMO study is to gather more information about how effective and safe ASTX727 is compared with best supportive care.

• REC name

  West of Scotland REC 1

• REC reference

  22/WS/0011

• Date of REC Opinion

  22 Feb 2022

• REC opinion

  Further Information Favourable Opinion