12th February 2020

Duchenne UK is pleased to announce that TAMDMD, the Tamoxifen trial, led by Professor Dirk Fischer, has now been fully recruited for the main group of patients.

This means that in 12 months, these patients will have completed their 48 weeks of treatment and results of the trial can be expected in Spring 2021.

Duchenne UK has championed this project from the beginning, and we have committed £1.5m over four years. We are funding additional trial sites as well as the open label extension once the trial has been completed to allow boys who have been dosed as part of the trial to continue taking Tamoxifen.

Through the DMD Hub, DUK was instrumental in helping to reach the recruitment target by working with three UK centres – Leeds, Glasgow and Alder Hey.  A total of 9 sites across Europe have recruited 79 patients to the trial. Other sites are in Switzerland, Germany, Netherlands and Spain.  

Tamoxifen is a breast cancer drug that has the potential to be re-purposed for DMD.  Early data in the DMD mouse model showed that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.  There is also some encouraging clinical data from an Investigator in Israel who has been treating a small number of her DMD patients with tamoxifen. 

The TAMDMD trial is important as it will give a definitive answer to the question of whether Tamoxifen is a useful and safe treatment for DMD.

We are exceptionally proud to work with such a committed group of people, from clinicians to researchers, from fundraisers to partner charities, who are united in our ambition to bring effective treatments to this generation of patients.

There is also a second group that is still recruiting for non-ambulant, steroid naïve boys, and the UK trial sites are all still recruiting for this group only. If you are interested in taking part in this trial, please visit the DMD Hub website to see contact details for these sites: https://dmdhub.org/trials/tamdmd/

Emily Crossley and Alex Johnson, Co-founders of Duchenne UK said:

“We are incredibly grateful to Prof Fischer and his team for reaching this important milestone. Duchene UK has committed vital funds and expertise to help get this trial underway. We have helped with regulatory support and recruitment for the study through the DMD Hub. We want to thank all the families who have enrolled in the study.”  

Professor Dirk Fischer, from UKBB, the University Children’s Hospital in Basel, Switzerland in Switzerland who is running the study, said:

 “We are very excited to announce that we have reached our goal of 79 recruited patients in group A (ambulant patients) of the TAMDMD study. This moves us closer to our target and we hope that we will be able to complete overall recruitment by the end of March 2020. Furthermore, 14 group B patient (non-ambulant) need to be included.” 

The trial is being jointly funded by Duchenne UK, E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy.

In this endeavour, we have not only our loyal and dedicated supporters to thank, but also our partner charities and funds for their support of our ongoing work on repurposing: Joining Jack, Alex’s Wish, Duchenne Now, For Felix, Jack’s Mission, Jack’s Aim, Archie’s March, Caring for Connor, Brandon’s Fund, Team Felix, Smile with Shiv, Help Harry, Hope for Harry, Jacobi’s Wish, Jayden's Army, The Smedley Family and Chasing Connor’s Cure.

Duchenne UK would also like to thank Marshall Wace for supporting this project.

NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) 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.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

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 DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org