DMD research

Update on the clinical trial to investigate the effectiveness of Tamoxifen in Duchenne muscular dystrophy

Duchenne UK has now received preliminary data on the TAM-DMD clinical trial, which was investigating the safety and efficacy of tamoxifen, a breast cancer drug, as an effective treatment for DMD.

Duchenne UK, along with our charity partners, funded a trial called TAMDMD to assess the safety and efficacy of tamoxifen, a drug which is currently used to treat breast cancer and showed promise for treating DMD. We have now received preliminary data from the trial.

The data, which compared disease progression in a group of boys receiving tamoxifen to that in a group receiving a placebo, has now been analysed following completion of the initial stages of the trial. This analysis found that over the 48 week trial period, patients in both the tamoxifen and placebo group showed mild disease progression against all clinical and MRI endpoints. While there was a trend for less disease progression in the tamoxifen group, the differences between the two groups were not large enough to determine if tamoxifen was effective in delaying disease progression.

Further analysis of the data is ongoing, with investigators specifically looking to try to understand why the group of boys receiving placebo did not progress as much as would have been expected – a result which contributed to the small difference between the two groups.

The data has however shown that across this trial and the follow-on open label extension, there have been no safety concerns so far and the drug has been generally well tolerated.

Based on these initial data though, the clinical team have concluded that the trial did not meet its primary goals. These preliminary results have now been communicated to all trial sites and participants  and further discussion around the data and next steps for those boys who are still enrolled in the open label extension study are ongoing. Clinical decisions for this group will be made in conjunction with participants and their families based on review of all available clinical information.

While these are not the results we had been hoping for, we are incredibly grateful for the support of all clinicians and sponsors, and of course to all of those who have participated in this trial. We would also like to thank all the funders: E-Rare; Eureka Foundation; Alex’s Wish; Joining Jack; Duchenne Parent Project Netherlands; Monaco Association Against Muscular Dystrophy; Duchenne Now; Duchenne Switzerland; Swiss Foundation for Research into muscle diseases; Caring for Connor; Brandon’s Fund; and all our Family Funds – For Felix, Team Felix, Help Harry, Archie’s March, Jacobi’s Wish, Jack’s Mission, Chasing Connor’s Cure, Smile with Shiv, Team Oscar and Jack’s Aim.

As we continue to analyse the data we will maintain transparent communications, and we anticipate being able to publish the full data in a peer-reviewed journal in 2022.

Additional information on the trial found here, as a Q&A.

Trial participants and their families should contact their trial sites directly with any questions for further information.

Visit our research glossary to find out more about the terminology used in DMD research

Published on 30 September 2021

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