Metformin in LFS (MILI) Trial

• Research type

  Research Study

• Full title

  Metformin in Li Fraumeni Syndrome (MILI) Trial: A phase II randomised open-label cancer prevention trial of metformin in adults with Li Fraumeni Syndrome

• IRAS ID

  1006131

• Contact name

  MILI Trial Manager

• Contact email

  octo-mili@oncology.ox.ac.uk

• Sponsor organisation

  University of Oxford

• Eudract number

  2022-000165-41

• ISRCTN Number

  ISRCTN16699730

• Research summary

  This clinical study is investigating the use of a drug called metformin as a way of reducing the cancer risk in people with Li Fraumeni Syndrome (LFS). LFS is a rare genetic condition that predisposes people to develop one or more cancers. It is caused by an inherited mutation in a gene called TP53. TP53 is the most important anticancer gene in the body. For most people with cancer the gene is only mutated in their cancer cells but for people with LFS it is mutated in all the cells in the body. This means there is a very high risk of developing cancer for people with LFS – a 90% risk for women and about 70% for men.
  Currently there are no treatments to reduce this high risk of cancer. People with LFS have regular health checks and whole-body MRI imaging to catch any new cancers early. Research shows that for people with LFS, spotting cancer early improves survival. Many LFS people are still not getting the care and scans they need.
  Metformin is a well-known and safe drug used to treat diabetes. Laboratory experiments have shown that in mice with LFS, metformin can reduce the risk of them developing cancer. Metformin alters the metabolism of cells with mutated TP53 and makes them act more like normal cells. This is not enough evidence to conclude that metformin will reduce the risks of cancer in people with LFS. A larger clinical trial is needed. The MILI study aims to enroll 224 people with LFS in the UK. Half the participants will take metformin everyday for 5 years, the other half will not. All participants will have regular check-ups including whole body MRI scans at selected NHS hospitals. The sample collection will help researchers understand more how the cells increase the risk of cancer in people with LFS. The study has been designed with members from the George Pantziarka TP53 Trust (GPTT) the charity for LFS. Fore more information please go to the GPTT charity website http://www.tp53.co.uk or contact the MILI trial office: octo-MILI@oncology.ox.ac.uk

• REC name

  West of Scotland REC 1

• REC reference

  23/WS/0051

• Date of REC Opinion

  12 Sep 2023

• REC opinion

  Further Information Favourable Opinion