December 2018

• 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. Read more about repurposing here.

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.

 

 

ENDS   -

  

NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease. 

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

 

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCH10 £10 to 70070

 

For more information and interview requests: Visit www.duchenneuk.org

Molly Hunt – Communications Manger, Duchenne UK  E: [email protected]