The DMD Hub’s Clinical Trial Finder brings together trustworthy and reliable information on all existing and upcoming trials for Duchenne Muscular Dystrophy in the UK.
Our Clinical Trial Finder has been designed for patients and caregivers, to be as accessible and comprehensive as possible. Every trial has information on outcome measures, inclusion criteria and an easy to understand lay summary. You can use search filters to find trials that are relevant to you and download a fact sheet for each trial. The information on each trial has been sourced directly from industry and hospitals and is verified by Duchenne UK and the DMD Hub management team.
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Patients should contact sites directly for additional information on studies they believe they would be eligible to participate in.
= Fully recruited
= Not yet recruiting
= Enrolling by invitation
This study is designed to study a number of genes considered to be modifiers for DMD. This translational research will identify and obtain DNA samples and clinical information from 400 cases with DMD. This data will then be grouped into clinically and genetically defined groups. The DNA of the participants will be analysed and correlated to motor performance, age at loss of ambulation, severity of respiratory failure and severity of cardiac impairment.
This phase 1 study is designed to determine the safety and tolerability of Wave Life Science’s Exon 51 skipping therapy.
PolarisDMD is a global, placebo controlled, Phase 3 trial for edasolonexent (CAT-1004). Edasalonexent is an NF-kB inhibitor, which could provide an alternative to steroids. Edasalonexent has been shown to preserve muscle function and substantially slow Duchenne disease progression in the MoveDMD trial.
This trial will evaluate the efficacy and safety of edasolonexent in patients with DMD, and is intended to support an application for commercial licencing of edasolonexent.
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This phase 4 clinical study is designed to assess the safety of Translarna, also known at Ataluren. This study will follow patients who are receiving Translarna as part of their usual care for 5 years. At the patients usual visits, data will be collected to determine the safety and effectiveness of Translarna.
This is an open label extension trial for patients who completed the POLARIS-DMD trial, and their siblings who meet the inclusion criteria between the ages of 4-12yrs (up to their 13th birthday).
This trial is looking at the safety, tolerability and durability of taking edasalonexent over a long period of time. Edasalonexent has been shown to delay the progression of DMD and could provide an alternative to steroids. It is in tablet form and taken orally (by mouth) three times a day.
Stay informed about the latest DMD clinical trials and keep up-to-date with the latest news from the DMD Hub.