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


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DMD Hub logo

Sarepta- ESSENCE

Study of SRP-4045 and SRP-4053 in DMD Patients

Hub Summary

This study is a stage 3 trial of Sarepta's exon 45 and exon 53 skipping drugs. Exon skipping drugs use a small piece of genetic material to skip over the part of the dystrophin gene with a mutation. The part of the dystrophin gene with a mutation varies between patients. Therefore, exon skipping trials are mutation specific. This trial requires you to be amenable to the skipping of exon 45 or 53.

The main objective of this study is to determine the efficacy of the drugs compared to a placebo in DMD patients.

Study Number: NCT02500381

Learn More
Overall Trial Status
Recruiting
Trial Sponsor
Sarepta Therapeutics, Inc.
Age
7-13
Mutation Specific
Mutation specific therapies, Must be amenable to exon 45 or 53 skipping
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

FOR-DMD

Finding the optimum regimen for Duchenne Muscular Dystrophy

Hub Summary

FOR-DMD study is designed to compare three different ways of giving corticosterioids to boys with DMD. The aim of this study is to see which method increases muscle strength the most and which produces the fewest side effects. The results of this study should provide patients and caregivers clearer information and guidelines about the best ways to take corticosteroids. The study will look at the following administration of corticosteroids:

  • Prednisone 0.75mg/kg/day
  • Prednisone 0.75mg/kg/day with 10 days on/10 days off treatment
  • Deflazacort 0.9mg/kg/day

Study Number: NCT01603407

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
University of Rochester
Age
4-7
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

Tamoxifen (TAMDMD)

Tamoxifen in Duchenne Muscular Dystrophy

Hub Summary

PLEASE NOTE: Recruitment for Group A (ambulant DMD patients) has been completed but recruitment for Group B (non-ambulant, steroid-naive DMD patients) is still open. Please contact the sites if you would like to take part. 

This placebo control, 48-week clinical trial will look at the treatment with Tamoxifen for both ambulant and non-ambulant patients with DMD. Tamoxifen has been used to treat breast cancer since the 1980s and is also used for hormonal disorders in pre-pubescent boys. Preliminary data in the DMD mouse model demonstrated that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.

Study Number: NCT03354039

Learn More
Overall Trial Status
Recruiting
UK Locations
Trial Sponsor
University Children's Hospital Basel
Age
78 months to 16 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Vamorolone Phase 2b (VISION-DMD)

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Hub Summary

This Phase 2b study is designed to evaluate the efficacy, safety pharmacodynamics and pharmacokinetics of vamorolone in comparison to corticosteroids and placebo treatments over a 24 week period. The study will also evaluate the persistence of the effect of vamorolone over a period of 48 weeks. 

The study is designed to compare 2 different doses of Vamorolone to a standard dose of corticosteroids (prednisone at 0.75 mg/kg/day) and to a placebo. Across all sites, this trial will be recruiting a total of 120 ambulant DMD patients ages 4 to <7 years. 

Study Number: NCT03439670

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
ReveraGen BioPharma
Age
4 to 7 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

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