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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Santhera (SIDEROS) [TERMINATED]

A Phase III Double-blind Study with Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) taking Glucocorticoid Steroids

Hub Summary

The SIDEROS trial is designed to determine the effect of idebenone at delaying the loss of lung function in patients with DMD, receiving glucocorticoid steroids. This is a placebo-controlled trial.

Study Number: NCT02814019

Learn More
Overall Trial Status
Trial terminated
Trial Sponsor
Santhera Pharmaceuticals
Age
10 years +
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
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PepGen - CONNECT2-EDO51

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDO51 with a Long-Term Extension in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)

Hub Summary

The CONNECT2-EDO51 Phase 2 clinical trial is a multinational, randomized, double- blind, placebo-controlled, multiple ascending dose (MAD) study, that will enroll ambulatory and non-ambulatory boys and young men living with DMD amenable to exon 51-skipping, who are at least six years of age. Participants will receive seven doses of either PGN-EDO51 or placebo at approximately four-week intervals for 24 weeks. Participants will provide a muscle biopsy at baseline and then at week 25. The trial will evaluate the safety and tolerability of PGN-EDO51 and the levels of dystrophin in skeletal muscle following repeat dosing. All participants will have the opportunity to participate in an open-label extension for 108 weeks after completing the MAD period where all participants will receive only the investigational study drug PGN-EDO51.

Study Number: EU Clinical Trial Number: 2023-508383-29

Learn More
Overall Trial Status
Recruiting
UK Locations
Trial Sponsor
PepGen
Age
6 +
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

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