Clinical Trial – Metformin in Li Fraumeni Syndrome


A clinical trial of the antidiabetic drug metformin is being developed for adults with Li Fraumeni Syndrome (LFS). This is the MILI trial – which we’ve discussed before. The trial aims to test whether metformin can reduce the incidence of cancer in people with LFS over a 5-year period. To do this rigorously, the study has to be randomised, in other words participants within the trial will be split into two groups for comparison: one group receiving metformin and one group not receiving it. Importantly, in the MILI trial, all participants will receive yearly cancer screening (annual whole body and brain MRI and routine blood tests for early cancer detection), regardless of whether they are allocated to the metformin or non-metformin treatment groups.

The team designing the trial have now to decide what the non-metformin arm of the study should be. If it is run as a placebo-controlled trial, participants will receive tablets that could either be metformin or a “dummy” (or inert) drug to take every day for 5 years. Neither the participant nor the study team will know whether they were on the real metformin until the trial is finished. An alternative trial design is called an open-label design where participants in the non-metformin arm will simply have screening alone, will not receive placebo tablets but will have to agree not to take metformin (or metformin-related agents) as a cancer preventative during the 5 years of the study.

Important: This survey is ONLY for people diagnosed with Li Fraumeni Syndrome! Please do NOT complete it if you do not have LFS! Thank You!

Survey is now closed – Thank You to all who took part. Results will be published soon!.