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 download a fact sheet for each trial, and use search filters to find trials that are relevant to you. 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.

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 clinicaltrials.gov updated with accurate information.

Showing all 26 trials

Catabasis- POLARIS DMD

Phase 3 POLARIS DMD Trial

Hub Summary

POLARIS DMD is a global, placebo controlled, Phase 3 trial for edasolonexent (CAT-1004). Edasalonexent is an NF-kB inhibitor, which could provide an alterntive 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.

Study Number: not on clinicaltrials.gov.uk

Learn More
Trial Status
Not yet recruiting
Trial Sponsor
Catabasis Pharmaceuticals
Age
4-7 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

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

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Trial Status
Recruiting
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

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
Trial Status
Recruiting
UK Locations
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

Brain study

Brain imaging and Cognition in patients with DMD

Hub Summary

This 2-year natural history study is designed to study the progression of pathological changes in the brain associated with the absence of dystrophin. The study will focus on cognitive impairment as well as looking at the relationship between the outcome measures and behavioural functioning.

Study Number: NOT ON clinicaltrials.gov

Learn More
Trial Status
Ongoing (fully recruited)
UK Locations
Trial Sponsor
Newcastle-upon-Tyne Hospitals NHS trust
Age
8 and over
Mutation Specific
All treatment types
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

Ataluren long-term

Study of Ataluren for previously treated patients with nmDBMD in Europe, Israel, Australia and Canada

Hub Summary

DMD is caused by a mutation in the gene which produces dystrophin. Dystrophin functions to maintain muscle structure and function. The loss of dystrophin in DMD leads to muscle weakness and loss of ambulation. A nonsense mutation is a specific type of mutation which is the cause of DMD in 10-15% of patients.

Ataluren is a drug designed to make the body's machinery less sensitive to nonsense mutations. This phase 3 trial is designed to assess the long-term safety and tolerability of Ataluren.

Study Number: NCT01557400

Learn More
Trial Status
Trial complete
Trial Sponsor
PTC Therapeutics
Age
All
Mutation Specific
Mutation specific therapies, Nonsense mutations only
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Non-ambulant

Santhera (SIDEROS)

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
Trial Status
Recruiting
Trial Sponsor
Santhera Pharmaceuticals
Age
10 years +
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

PreU7-53

Observational study of patients with DMD theoretically treatable with exon skipping 53

Hub Summary

PreU7-53 is an observational cohort study. This natural history study is designed to monitor upper limb muscle impairment in patients potentially treatable with AAV-mediated exon skipping.

Study Number: NCT01385917

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Trial Status
Ongoing (fully recruited)
UK Locations
Trial Sponsor
Genethon
Age
12-20
Mutation Specific
Mutation specific therapies, Amenable to treatments with Exon 53 skipping
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Non-ambulant

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
Trial Status
Not yet recruiting
Trial Sponsor
ReveraGen BioPharma
Age
4 to 7 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

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.

Please note that this trial is no longer recruiting at GOSH. 

Study Number: NCT02500381

Learn More
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

Testosterone for DMD

Testosterone therapy for Pubertal delay in DMD

Hub Summary

This observational study is looking at the outcomes of testosterone treatment in boys with DMD. The study is following the progression of adolescent males with DMD and delayed puberty. These patients are treated with testosterone to induce puberty. The participants are treated with the standard regiment of testosterone and this study is collecting data to review the effectiveness and tolerability of the current treatment regimen.

The researchers will use the results to explore what effect testosterone treatment has on pubertal development, growth, muscle strength and function, bone mineral density and body composition. The study also aims to define and understand side effects testosterone treatment may have.

Study Number: NCT02571205

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Trial Status
Ongoing (fully recruited)
UK Locations
Trial Sponsor
Newcastle-upon-Tyne Hospitals NHS Trust
Age
12-17
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Non-ambulant

Domagrozumab extension

An Open-label extension Study to Evaluate Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy

Hub Summary

This study is designed to evaluate the safety and efficacy of Domagrozumaub, a myostatin inhibitor. Myostatin is a protein in the body which inhibits muscle growth and it is required to stop muscles from growing too large. It it thought that inhibiting myostatin may help preserve or improve muscle function in patients with DMD. 

This is an open-label extension study of the Phase 2 study of Domagrozumaub - subjects are only eligible for this study if they were enrolled into and completed the phase 2 study. 

Study Number: NCT02907619

Learn More
Trial Status
Trial terminated
Trial Sponsor
Pfizer
Age
6 - 18
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Non-ambulant

PTC Ataluren Phase 3

Phase 3 Extension study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Hub Summary

DMD is caused by a mutation in the gene which produces dystrophin. Dystrophin functions to maintain muscle structure and function. The loss of dystrophin in DMD leads to muscle weakness and loss of ambulation. A nonsense mutation is a specific type of mutation which is the cause of DMD in 10-15% of patients.

Ataluren is a drug designed to make the body's machinery less sensitive to nonsense mutations. This phase 3 trial is designed to assess the long-term safety of Ataluren in boys with nonsense dystrophinopathies. The study will also assess changes in clinical measures such as muscle function and pulmonary function.

Study Number: NCT02090959

Learn More
Trial Status
Trial complete
Trial Sponsor
PTC Therapeutics
Age
7-18
Mutation Specific
Mutation specific therapies, Nonsense mutations only
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

Domagrozumab phase 2

A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

Hub Summary

This phase 2 trial is designed to evaluate the safety and efficacy of Domagrozumaub, a myostatin inhibitor. Myostatin is a protein in the body which inhibits muscle growth and it is required to stop muscles from growing too large. It is thought that inhibiting myostatin may help preserve or improve muscle function in patients with DMD. 

Study Number: NCT02310763

Learn More
Trial Status
Trial terminated
Trial Sponsor
Pfizer
Age
6-16
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

RIM4DMD

Rimeporide in patients with Duchenne muscular dystrophy

Hub Summary

Patients with DMD have imbalanced levels of calcium and sodium in their muscle cells, this is thought to play a key part in the damage which occurs to muscles. This study is evaluating the safety and tolerability of rimeporide. Rimeporide is a drug which works by inhibiting the movement of sodium and calcium from muscle cells. Inhibition of this mechanism has been proven to be efficient in preventing inflammation and fibrosis (muscle damage) in animal models. In addition to the preventative effect on muscle damage, rimeporide was also shown to be cardioprotective. 

This investigational treatment could have no restriction on age and is not mutation specific, meaning it could treat all patients with DMD. This phase 1b study will examine Rimeporide in patients aged 6 to 14 years.

Study Number: NCT02710591

Learn More
Trial Status
Trial complete
UK Locations
Trial Sponsor
EspeRare Foundation
Age
6-14
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

Summit- Ezutromid (PhaseOut DMD)

PoC Study to Assess Activty and Safety of SMT C1100 (Ezutromid) in Boys with DMD

Hub Summary

Please note this trial is currently only open to recruitment for patients who have participated in a prior Summit trial.

This phase 2 clinical trial is designed to assess the activity and safety of Utrophin modulation in patients with DMD with SMT C1100 (Ezutromid). Ezutromid is an orally administered small molecule utrophin modulator.

Utrophin is a bodily protein which is structurally and functionally similar to dystrophin. Utrophin is naturally produced in the early stages of muscle development but is turned off as muscle fibres mature and dystrophin increases to perform the same role. Utrophin modulation aims to maintain the production of utrophin to compensate for the absence of dystrophin in patients with DMD.

This 1 year study has an optional extension phase. 

Study Number: NCT02858362

Learn More
Trial Status
Trial terminated
Trial Sponsor
Summit Therapeutics
Age
5-10
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant

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

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Trial Status
Recruiting
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

KINEDMD

KINEDMD

Hub Summary

This study is for proof of concept, designed to use artificial intelligence to identify kinematic biomarkers (fingerprints of movement) of DMD progression. This could speed up drug development for new therapies and repurposed drugs, in order to deliver treatments to children as fast as possible.

Once fingerprints of movement specific for DMD boys are identified, the results will be published and made available to the whole community. This pilot study also wishes to lay the foundation for future validation of these novel biomarkers. This is an opportunity to take part in innovative natural history research that could improve clinical trial design for future trials.

Duchenne Research Fund is the funding body for this study. 

Learn More
Trial Status
Recruiting
UK Locations
Trial Sponsor
Imperial College London
Age
6 to 17 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

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
Trial Status
Not yet 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

Sarepta 51 (Young)

Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping

Hub Summary

This phase 2 open-label study is designed to determine the safety and efficacy of eteplirsen in young patients with DMD. Etepliresen (EXONDYS 51®) is an exon-skipping drug designed to treat patients with DMD amenable to exon 51 skipping. This study will enroll young males between the ages of 6 months and 4 years.

Study Number: NCT03218995

Learn More
Trial Status
Recruiting
UK Locations
Trial Sponsor
Sarepta Therapeutics, Inc.
Age
6 months to 4 years
Mutation Specific
Mutation specific therapies, Must be amenable to exon 51 skipping
Muscle Biopsy
No Muscle Biopsy Required
Ambulation

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.

Study Number: NCT02851797

Learn More
Trial Status
Ongoing (fully recruited)
Trial Sponsor
Italfarmaco
Age
6-17
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

Roche- BMS-986089

Clinical trial to Evaluate the Efficacy, Safety and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy

Hub Summary

This placebo-controlled study is designed to assess the efficacy, safety and tolerability of two different doses of BMS-986089 in ambulatory males with DMD. BMS-986089 is a subcutaneously delivered treatment which inhibits myostatin.

Myostatin is a protein in the body which inhibits muscle growth and it is required to stop muscles from growing too large. Myostatin production increases naturally with age. It is thought that inhibiting myostatin function could lead to increased muscle size and strength in patients with DMD.

Study Number: NCT03039686

Learn More
Trial Status
Recruiting
Trial Sponsor
Hoffmann-La Roche
Age
6-11
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

Vamorolone Phase 2 Extension

Long-term extension Study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)

Hub Summary

This extension study is designed to assess the safety of using vamorolone long term in children with DMD. The study will also compare muscle function to boys with DMD in other studies who did not take steroids as well as comparing weight gain with boys taking vamorolone and boys taking traditional steroids (prednisone).

Vamorolone is a steroid alternative which is designed to be an alternative to corticosteroids with reduced side effects.

Study Number: NCT03038399

Learn More
Trial Status
Enrolling by invitation
UK Locations
Trial Sponsor
ReveraGen BioPharma, Inc.
Age
4-7
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

Sarepta 53

Phase I/II Study of SRP-4053 in DMD Patients

Hub Summary

This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics of Sarepta's exon skipping drug SRP-4053. SRP-4053 is designed to treat patients with DMD with deletions amenable to exon 53 skipping.

Study Number: NCT02310906

Learn More
Trial Status
Ongoing (fully recruited)
Trial Sponsor
Sarepta Therapeutics, Inc.
Age
6-15
Mutation Specific
Mutation specific therapies, Amenable to exon 53 skipping
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant

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
Trial Status
Ongoing (fully recruited)
Trial Sponsor
University of Rochester
Age
4-7
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant

Outcome measures

Outcome measures in Duchenne Muscular Dystrophy: A natural history study

Hub Summary

With so many new therapies emerging for DMD, it is important we understand the natural history of the disease. This natural history study will assess the natural history of DMD sing a selection of assessment tools. The aim is to obtain natural history data to capture disease progression linking the ambulant and non-ambulant phases of DMD. This study will also provide an insight into the relationship between different assessment tools which are used in the clinic. 

Study Number: NCT02780492

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Trial Status
Ongoing (fully recruited)
Trial Sponsor
University College, London
Age
5-18
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

MCID DMD

Minimal Clinically Important Difference (MCID) in Duchenne Muscular Dystrophy (DMD) tests

Hub Summary

This study is looking at whether we can quantify the smallest change in two outcome measures that is meaningful for families, patients and clinicians. Participation in this study includes just a one-off questionnaire which takes around 30-45 minutes to complete. 

Learn More
Trial Status
Recruiting
UK Locations
Trial Sponsor
Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health
Age
7 to 18 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant