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


Showing 9

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Tamoxifen (TAMDMD)

Tamoxifen in Duchenne Muscular Dystrophy

Hub Summary

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
Trial complete
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
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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
Trial complete
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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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
Trial complete
Trial Sponsor
University of Rochester
Age
4-7
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
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NS Pharma - RACER53-X

Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

Hub Summary

This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.

The dystrophin gene has 79 pieces called exons. These link together to form a code which instructs the body to make dystrophin. If there is a fault, as in DMD, the sequence is broken and the code cannot be read. Exon skipping drugs complete the sequence and leads to a shortened dystrophin being produced that still contains the important pieces of this molecule.

Study Number: NCT04768062

Learn More
Overall Trial Status
Enrolling by invitation
Trial Sponsor
NS Pharma, Inc.
Age
5-8
Mutation Specific
Mutation specific therapies, Exon 53
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
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NS Pharma - RACER53

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

Hub Summary

This is a placebo-controlled phase 3 study, designed to investigate the efficacy and safety of NS Pharma's exon skipping drug, Viltolarsen. It will be focusing on patients with mutations amenable to exon 53 skipping and will involve a weekly intravenous infusion over 48 weeks.

The dystrophin gene has 79 pieces called exons. These link together to form a code which instructs the body to make dystrophin. If there is a fault, as in DMD, the sequence is broken and the code cannot be read. Exon skipping drugs complete the sequence and leads to a shortened dystrophin being produced that still contains the important pieces of this molecule.

Study Number: NCT04060199

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
NS Pharma
Age
4-7
Mutation Specific
Mutation specific therapies, Exon 53
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
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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
Fully recruited
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
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Sarepta - MOMENTUM

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Hub Summary

This phase 2 study is designed to determine the maximum dose for Sarepta Therapeutics Exon 51 skipping therapy, as well as its safety and tolerability.

There will be two arms to the study - in Part A, patients will receive 1 of 5 doses of SRP-5051 monthly by intravenous infusion. Once the maximum dose has be has been determined, all patients will then roll over into Part B and will receive the maximum dose by intravenous infusion for 24 weeks. In Part B, an additional 15 patients will also be enrolled at the beginning of the study. 

Part A recruitment has now been completed and Part B will be beginning soon, involving the original patients from Part A as well as some additional patients. 

The UK sites have not yet been finalised, we will provide an update once we have these details.

Study Number: NCT04004065

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
Sarepta Therapeutics
Age
7-21 years old
Mutation Specific
Mutation specific therapies, must be amenable to exon 51 skipping
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
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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
Recruiting
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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Italfarmaco - ULYSSES

Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)

Hub Summary

The main objective of this study is to demonstrate the efficacy of givinostat in reducing muscle decline in non-ambulant patients aged 9-17 with Duchenne Muscular Dystrophy.  Additional objectives are the evaluation of safety, tolerability of the drug and further exploration of efficacy of givinostat in non-ambulant DMD population.

Study Number: NCT05933057

Learn More
Overall Trial Status
Recruiting
UK Locations
Trial Sponsor
Italfarmaco
Age
9-17
Mutation Specific
All treatment types
Muscle Biopsy
Ambulation
Non-ambulant

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