Clinical Trial Finder
The Clinical Trial Finder brings together trustworthy and reliable information on all existing and upcoming trials for Duchenne muscular dystrophy in the UK.
MCID DMD
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.
Age
7
-
18
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Description
Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health
Sarepta- ESSENCE
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.
Age
7
-
13
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Wave Life Sciences Exon 51
This phase 1 study is designed to determine the safety and tolerability of Wave Life Science’s Exon 51 skipping therapy.
Age
5
-
18
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
Brain study
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.
Age
8
-
8
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Vamorolone Phase 2b (VISION-DMD)
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.
Age
4
-
7
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Tamoxifen (TAMDMD)
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.
Age
78
-
78
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD.
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.
Age
5
-
5
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Vamorolone Phase II Extension
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).
Age
4
-
7
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Testosterone for DMD
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.
Age
12
-
17
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Italfarmaco- Givinostat (EPIDYS)
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.
Age
6
-
17
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
KINEDMD
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.
Age
6
-
17
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Outcome measures
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.
Age
5
-
18
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
RIM4DMD
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.
Age
6
-
14
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
PTC Ataluren Phase 3
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.
Age
7
-
18
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
FOR-DMD
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:
Age
4
-
7
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Patient Registry Translarna (Ataluren)
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.
Age
Any
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Sarepta 53
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.
Age
6
-
15
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Ataluren long-term
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.
Age
Any
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
nonAmbulant
PreU7-53
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.
Age
12
-
20
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
nonAmbulant
DYSTANCE 51 [TERMINATED]
Please note that Wave have stopped the development of this drug after the Phase 1 Open-label extension failed to meet its primary endpoint.
Age
5
-
12
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Phase 3 PolarisDMD Trial [TERMINATED]
PolarisDMD is a global, placebo controlled, Phase 3 trial for edasolonexent (CAT-1004). Edasalonexent is an NF-kB inhibitor, which could provide an alternative to steroids. Edasalonexent has been shown to preserve muscle function and substantially slow Duchenne disease progression in the MoveDMD trial.
Age
4
-
7
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Catabasis - Galaxy DMD [TERMINATED]
This is an open label extension trial for patients who completed the POLARIS-DMD trial, and their siblings who meet the inclusion criteria between the ages of 4-12yrs (up to their 13th birthday).
Age
4
-
12
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Domagrozumab extension [TERMINATED]
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.
Age
6
-
18
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
nonAmbulant
Domagrozumab phase 2 [TERMINATED]
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.
Age
6
-
16
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Santhera SIDEROS Open Label Extension [TERMINATED]
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.
Age
11
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Roche- RO7239361 [TERMINATED]
Please note the development of this drug was stopped after this trial failed to reach its objectives.
Age
6
-
11
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Santhera (SIDEROS) [TERMINATED]
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.
Age
10
-
10
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Summit- Ezutromid (PhaseOut DMD) [TERMINATED]
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.
Age
5
-
10
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
