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
UK Locations
London - GOSH
Leeds
Newcastle
Oswestry
Oxford
Queens Square
Temple Street
Trial Sponsor
Santhera Pharmaceuticals
Age
10 years +
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Santhera SIDEROS Open Label Extension [TERMINATED]

Phase III Study with Idebenone in Patients with Duchenne Muscular Dystrophy

Hub Summary

This study is a stage 3 trial of Sanethera's Idebenone drug and it's long term effects in delaying the loss of lung function in patients with DMD, receiving glucocorticoid steroids. This trial is only open to those patients who were part of the SIDEROS trial and are currently taking steroids. 

Study Number: NCT03603288

Learn More
Overall Trial Status
Trial terminated
UK Locations
London - GOSH
Leeds
Newcastle
Oswestry
Trial Sponsor
Santhera Pharmaceuticals
Age
11 +
Mutation Specific
All treatment types
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Italfarmaco- Givinostat (EPIDYS)

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients with Duchenne Muscular Dystrophy (EPIDYS)

Hub Summary

This study will compare the change in stair climb test and other functional tests in patients taking givinostat and patients taking a placebo. Givinostat has potential anti-inflammatory, antifibrotic and proregenerative effects.

Please note this protocol was amended early 2019.

Study Number: NCT02851797

Learn More
Overall Trial Status
Trial complete
UK Locations
London - GOSH
Alder Hey
Newcastle
Oswestry
Trial Sponsor
Italfarmaco
Age
6-17
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
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Italfarmaco - Givinostat Extension

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Hub Summary

This study is an open label extension, looking at the long-term safety and tolerability of GIVINOSTAT in patients who have already taken part in and completed any of the previous studies. 

GIVINOSTAT is a drug that may help to promote muscle regeneration and reduce inflammation and fibrosis in DMD patients. 

This extension is expected to last until the drug receives the necessary approvals and is available on the market or the study needs to be stopped due to safety and/or efficacy reasons.

Study Number: NCT03373968

Learn More
Overall Trial Status
Fully recruited
UK Locations
London - GOSH
Alder Hey
Newcastle
Oswestry
Trial Sponsor
Italfarmaco
Age
7+
Mutation Specific
All treatment types
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-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
Fully recruited
UK Locations
London - GOSH
Alder Hey
Birmingham
Bristol
Leeds
Newcastle
Oswestry
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
DMD Hub logo

Hydrotherapy in DMD

Hydrotherapy for health in boys and adolescents with Duchenne muscular dystrophy

Hub Summary

Exercise is very important for young people to help keep them healthy. It could be as important for children and young people who have a muscle disease, like Duchenne muscular dystrophy (DMD). Hydrotherapy is a form of exercise that involves doing exercises with a physiotherapist in a heated swimming pool.  

We are running a study to try to better understand the impact that hydrotherapy might have on young people with DMD, and whether or not it offers any benefits to their physical and mental wellbeing. We will use the findings from the study to develop simple guides to advise on activities and exercise while in the water. 

Learn More
Overall Trial Status
Recruiting
UK Locations
Manchester
Oswestry
Preston
Trial Sponsor
Lancashire Teaching Hospitals NHS Trust
Age
6-25
Mutation Specific
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Antisense - ATL1102

A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of ATL1102 in Non-ambulatory Participants With Duchenne Muscular Dystrophy

Hub Summary

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to 17 years old. The study includes a randomised, double-blind, placebo-controlled treatment period (Part A), followed by an open labelled treatment period (Part B).

Study Number: NCT05938023

Learn More
Overall Trial Status
Trial terminated
UK Locations
London - GOSH
Alder Hey
Birmingham
Glasgow
Leeds
Manchester
Oswestry
Trial Sponsor
Antisense Therapeutics Limited
Age
10 - 17
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Non-ambulant

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