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.

Join The DMD Hub, to stay up-to-date with the latest DMD clinical trials updates.

IMPORTANT NOTICE REGARDING THE IMPACT OF COVID-19 ON CLINICAL TRIALS IN THE UK

The DMD Hub sites have re-started to screen patients for ongoing trials and are working together to move ahead with setting up new trials that were not open to recruitment before March 2020

They are also taking learning from the COVID-19 situation and applying it where they can, such as more home visits. 

Patients should contact sites directly for additional information on studies they believe they would be eligible to participate in. 

The DMD Hub is not promoting any particular trial or therapy.  You should always consult your neuromuscular consultant before joining a trial. Please note that the trial status may vary from site to site and you should contact the individual site for details.
Please remember that this information relies on the sponsors keeping the DMD Hub updated with accurate information.

Location Trial Status:

= Fully recruited

= Recruiting

= Not yet recruiting

= Enrolling by invitation


Showing 5

DMD Hub logo

Wave Life Sciences Exon 51

A Multicentre, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy

Hub Summary

This phase 1 study is designed to determine the safety and tolerability of Wave Life Science’s Exon 51 skipping therapy.

Study Number: NCT03508947

Learn More
Overall Trial Status
Trial complete
Trial Sponsor
Wave Life Sciences
Age
5 to 18 years
Mutation Specific
Mutation specific therapies, must be amenable to exon 51 skipping
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

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
DMD Hub logo

Catabasis - Galaxy DMD [TERMINATED]

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Hub Summary

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. 

Study Number: NCT03917719

Learn More
Overall Trial Status
Trial terminated
Trial Sponsor
Catabasis Pharmaceuticals
Age
4-12
Mutation Specific
All treatment types
Muscle Biopsy
Ambulation
Ambulant
DMD Hub logo

Phase 3 PolarisDMD Trial [TERMINATED]

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

Hub Summary

 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.

To receive up-to-date information about this trial, please sign up to the Catabasis newsletter

Study Number: NCT03703882

Learn More
Overall Trial Status
Trial terminated
Trial Sponsor
Catabasis Pharmaceuticals
Age
4-7 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD.

Disease translation in DMD

Hub Summary

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. 

Study Number: Not on clinicaltrials.gov

Learn More
Overall Trial Status
Recruiting
Trial Sponsor
Great Ormond Street Hospital NHS Foundation Trust
Age
>5 years old
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation

We have placed cookies on your computer to help make this website better. For more information please click here

By continuing to use this site or closing this panel, we'll assume you're OK to continue. You can view our full privacy policy here