• Open Label Extension Study will give all patients on the TAMDMD trial access to Tamoxifen after completing trial
• Total funding commitment to the TAMDMD trial from Duchenne UK now stands at £1.4million
In July 2017, we announced €675,000 of 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? We think 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.
That is why we are committing an additional £780,000 to the TAMDMD trial, for an open label extension study. The additional investment brings Duchenne UK’s total commitment to the Tamoxifen trial to just under £1.4million.
As it will take approximately 1 year to recruit all of the participants to the trial and each participant will be in the trial for 48 weeks, there will be a gap of approximately 1 year in which the first patients have finished the study but the results are not known.
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.
Emily Crossley, Co-founder of Duchenne UK said:
“Duchenne UK is committed to ensuring patients are at the heart of everything we do, so we are very pleased to be able to fund the extension study to ensure that all the patients taking part in the trial will be given the opportunity to take Tamoxifen once they have completed the main trial. The extension study will also help us gather important longer term data on the effect of Tamoxifen on patients.”
Professor Dirk Fischer, from UKBB, the University Children’s Hospital in Basel, Switzerland, who is running the study, said:
“We are very grateful for the ongoing support provided by Duchenne UK, which includes not only financial help, but also support when negotiating with regulatory authorities and other involved parties.”
For more information about the Tamoxifen clinical trial visit the DMD Hub clinical trial finder: https://dmdhub.org/trials/tamdmd/. The main trial is being jointly funded by Duchenne UK, E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy.
Duchenne UK’s commitment to developing existing medicines as potential treatments for DMD, an approach known as repurposing.
Duchenne UK would like to thank our partner charities and funds for their support of this project: Alex’s Wish, Duchenne Now, Joining Jack, Archie’s March, Help Harry, Jacobi’s Wish, Jack’s Aim, Team Felix and The Smedley Family.