Clinical Trial Finder

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.

Please note that the DMD Hub is not responsible for the direct recruitment of patients to trials. Although we work closely with sites to ensure the recruitment status for every trial is accurate and up to date, there may be a delay in updating the Clinical Trial Finder while the patient screening process takes place.

We recommend that UK patients/parent and caregivers register with the Central Recruitment Pilot Project, which will enable trial sites to contact you directly if you are eligible for a study. 

The DMD Hub is not promoting any particular trial or therapy.  You should always consult your neuromuscular consultant before joining a trial. 

Sign up to the DMD Hub mailing list to be notified of new clinical trials and research studies and DMD Hub news.

Please remember that this information relies on the sponsors and trial sites keeping the DMD Hub updated with accurate information.

Location Trial Status:

= Fully recruited

= Recruiting

= Not yet recruiting

= Enrolling by invitation


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Patient Registry Translarna (Ataluren)

Long-term observational study of Translarna safety and effectiveness in usual care

Hub Summary

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. 

Study Number: NCT02369731

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
PTC Therapeutics
Age
Child, adolescent and adult
Mutation Specific
Mutation specific therapies, Nonsense mutation
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

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