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Tamoxifen clinical trial is fully recruited

Joining Jack 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.

Joining Jack has championed this project from the start investing £300,000 as part of £1.5m investment over four years. Additional trial sites are also being funded 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, Joining Jack working with Duchenne UK was instrumental in helping to reach the recruitment target by working with three UK centres – Leeds, Glasgow and Alder Hey.  A total of nine 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/

Alex Johnson, co-founder of Joining Jack said:

“We are incredibly grateful to Prof Fischer and his team for reaching this important milestone. Joining Jack 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 Duchenne UK 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.” 

 

As well as £300,000 funding from Joining Jack, the trial is being jointly funded by Duchenne UK, E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy.

 

A huge thank you to you, our loyal and dedicated supporters, and also our partner charities and funds for their support of our ongoing work on repurposing: Duchenne UK, 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.

 

Joining Jack would also like to thank Marshall Wace for supporting this project.

 

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