Alex’s Wish invests £30,000 to Tamoxifen Open Label Extension Study

So pleased to let you know that we have just invested the £30,000 raised at the Mardi Gras Ball!

In 2017 we funded £25,000 into a €675,000 funding towards TAMDMD, a phase 3 clinical trial to test the breast cancer drug tamoxifen for use in patients with Duchenne muscular dystrophy. This funding provides the research team with a project manager as well as funding a site in Europe and two sites in the UK, Alder Hey and Leeds.

In June 2018 the first patients were dosed in this trial. So far, 12 patients have been dosed from a total of 99 participants.

But what happens when the trial is over? It is important that all trial participants (especially those being on the placebo arm) have access to the treatment once they have completed the trial. This is the case in most clinical trials and is known as the Open Label Extension Study.

So after raising £30,000 at our Mardi Gras Ball we decided to commit that amount towards an additional investment of £780,000 to the TAMDMD trial, for an open label extension study.

The open label extension study will allow participants to continue in the study after they have completed the main trial.  During this extension study all participants will be taking tamoxifen, whereas in the main trial half of the participants will have been taking a placebo. Efficacy data will still be collected and participants and safety monitoring will continue throughout.

The open-label extension study will help gather long term data on the effects of tamoxifen on patients with DMD.

The full funding amount of £780,000 was made up of Duchenne UK and their collaboration with Alex’s Wish and all these wonderful organisations and family funds – Duchenne Now, Joining Jack, Archie’s March, Help Harry, Jacobi’s Wish, Jack’s Aim, Team Felix and The Smedley Family.

This project is a great example of the commitment to developing existing medicines as potential treatments for DMD, an approach known as repurposing. Read more about repurposing here.

Posted in Collaboration, Funding