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.
Entrada - ELEVATE-44
Entrada Therapeutics is developing an exon 44 skipping therapy (called ENTR-601-44) for people living with Duchenne. Its goal is to help the body make a shorter, but still potentially functional dystrophin protein. Dystrophin is important because it helps keep muscles strong and stable.The ENTR-601-44-201 study (also called ELEVATE-44) is a global, two-part, randomized, double-blind placebo-controlled, Phase 1/2b study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in people living with Duchenne who are amenable to exon 44 skipping.
Age
4
-
20
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Santhera GUARDIAN
This Phase 4 study aims to assess safety and effectivness of long-term treatment with vamoralone in boys with Duchenne Muscular Dystrophy (DMD) who have completed prior studies with vamoralone.
Age
Any
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Italfarmaco - Givinostat in Younger DMD patients
The purpose of the study is to find out about the safety and tolerability of givinostat for the treatment of Duchenne muscular dystrophy (DMD) in patients aged from at least 2 years old to less than 6 years old. The study will also evaluate how the body absorbs, distributes, breaks down and eliminates givinostat.
Age
2
-
6
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
NutrInD: Nutrition In Duchenne Study
The purpose of this study is to collect information about the nutritional needs of people with Duchenne Muscular Dystrophy (DMD).
Age
5
-
21
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
PepGen - CONNECT2-EDO51
The CONNECT2-EDO51 Phase 2 clinical trial is a multinational, randomized, double- blind, placebo-controlled, multiple ascending dose (MAD) study, that will enroll ambulatory and non-ambulatory boys and young men living with DMD amenable to exon 51-skipping, who are at least six years of age. Participants will receive seven doses of either PGN-EDO51 or placebo at approximately four-week intervals for 24 weeks. Participants will provide a muscle biopsy at baseline and then at week 25. The trial will evaluate the safety and tolerability of PGN-EDO51 and the levels of dystrophin in skeletal muscle following repeat dosing. All participants will have the opportunity to participate in an open-label extension for 108 weeks after completing the MAD period where all participants will receive only the investigational study drug PGN-EDO51.
Age
6
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
Sarepta ENVISION
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Age
8
-
17
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
Roche - ENVOL
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants (Aged up to 3 years of age) will be in the study for approximately 264 weeks.
Age
Any
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
BioMarin - BMN 351
The purpose of this study is to test the safety and tolerability of BMN 351 in participants aged 4-10 with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping. BioMarin Pharmaceutical Inc (BioMarin), the sponsor of this study, wants to find out what effects, good and/or bad, BMN 351 has on your child and their DMD. BMN 351 is an experimental study medication that is given intravenously (through a needle or tube inserted into a vein); each infusion lasts about an hour.
Age
4
-
10
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Italfarmaco - ULYSSES
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.
Age
9
-
17
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
nonAmbulant
DMDhome
This decentralised study aims to evaluate whether new video-based electronic clinical outcome assessments (eCOAs) captured with the Atom5TMplatform DMDhome are able to detect disease progression from the late ambulatory to transfer stage and early non-ambulatory stage, compared with standard validated clinical assessments.
Age
8
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
BIND 2
The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.
Age
5
-
17
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
NCT04668716
Great Ormond Street Hospital for Children NHS Foundation Trust
Genethon- Microdystrophin Gene Therapy (GNT-016-MYDF)
GNT0004 is a recombinant adenovirus-associated viral (AAV) vector gene therapy, composed of an AAV8 serotype capsid containing a sequence-optimised gene for a human microdystrophin.
Age
6
-
10
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Antisense - ATL1102
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).
Age
10
-
17
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
nonAmbulant
Dyne Therapeutics - DYNE-251
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy, aged 4 to 16 years, Amenable to Exon 51 Skipping.
Age
4
-
16
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
NS Pharma - RACER53-X
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.
Age
5
-
8
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Hydrotherapy in DMD
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.
Age
6
-
25
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
D-Brain
Research has shown that in a proportion of individuals with DMD and BMD, there might be some involvement of how the brain works. This can result in some individuals having a degree of learning difficulties, behavioural or psychological difficulties.
Age
7
-
17
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Sarepta - MIS51ON
This phase 3 study is designed to evaluate the safety and tolerability of two doses of eteplirsen. Part 1 (now closed for recruitment) will investigate two doses with Part 2 comparing the most effective dose from Part 1 with a 30mg/kg dose of eteplirsen.
Age
4
-
13
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Sarepta - EMBARK
This study will evaluate the safety and efficacy of gene transfer therapy in boys aged between 4 and 7 with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
Age
4
-
7
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
D3 Creatine
This study will use a new, non-invasive method of measuring muscle mass in patients with Duchenne muscular dystrophy. It is jointly funded by Duchenne UK, Muscular Dystrophy Association and Parent Project Muscular Dystrophy.
Age
5
-
25
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
WVE - N531
This is a phase 1b/2a looking at the safety of a new exon skipping investigational therapy, WVE-N531. Wave will also be looking at the pharmacokinetics (how the drug is absorbed and metabolised in the body) and pharmacodynamics (how the drug affects the body).
Age
5
-
18
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
Genethon - Natural History of Duchenne Muscular Dystrophy
This natural history study is looking to collect data on the natural disease course in a cohort in young male subjects aged from 5 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.
Age
5
-
9
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Pilot Study Comparing night time AFOs with CCDs in the management of ankle contractures
This pilot study aims to identify the sample size required to power a larger scale study to compare AFOs and CCDs in managing ankle range of movement and function in boys with Duchenne muscular dystrophy. It will also explore adherence and patient satisfaction for the two devices.
Age
4
-
10
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
FibroGen - LELANTOS 2
This phase 3 study is looking at the efficacy and safety of pamrevlumab versus a placebo, in combination with corticosteroids (deflazacourt or prednisone). The trial is open to patients who are able to complete the 6 Minute Walk test (6MWD) with a distance of at least 270m but no more than 450m on two occasions 3 months before starting on the trial, as well as being able to rise from the floor (TTSTAND) in less than 10 seconds at the screening visit. Patients must also be on a stable dose of corticosteroids for a minimum of 6 months.
Age
6
-
12
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
FibroGen - LELANTOS
This phase 3 study is looking at the efficacy and safety of pamrevlumab versus a placebo, in combination with corticosteroids (deflazacourt or prednisone). It is only open to non-ambulant patients. There will be a placebo arm of the trial and 50% of the patients will be randomly allocated to each arm. Once all patients have completed the 52-week study, they may be eligible for rollover into an open-label extension (OLE) with pamrevlumab + corticosteroids.
Age
12
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
nonAmbulant
BIND Study
This study is looking at the connection between the behavioural aspects of DMD and a patient's DMD gene mutation. Participants will be asked to complete an online questionnaire, which will take approximately 70mins and can be completed at multiple sittings. This is open to male DMD patients between 5 and 17yrs old.
Age
5
-
17
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Interactive Virtual Reality System on Physiotherapy
This study is looking at the use of Immersive Virtual Reality (IVR) to improve the uptake of physiotherapy amongst young people with DMD. The first phase will involve a design workshop to explore different scenarios on the IVR to mirror current DMD physiotherapy recommendations, with the second phase looking at testing of these scenarios by young people with DMD. The study is currently open to those patients currently being seen at Leeds Teaching Hospital and Sheffield Children's NHS Foundation Trust, and is being funded by The Children's Hospital Charity.
Age
5
-
10
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
NS Pharma - RACER53
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.
Age
4
-
7
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
ambulant
Pfizer - CIFFREO
This study is a phase 3 trial testing the safety and efficacy of Pfizer's gene therapy construct, PF-06939926. It is delivered using an adeno-associated virus, AAV, and carries a shortened version of the dystrophin gene (mini-dystrophin). The treatment will be given by an intravenous infusion.
Age
4
-
7
years
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
ambulant
Italfarmaco - Givinostat Extension
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.
Age
7
Mutation Specific
Non-mutation Specific Therapies
Muscle Biopsy
Not Required
Muscle Biopsy Not Required
Ambulation
both
Sarepta - MOMENTUM
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.
Age
7
-
21
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
Testosterone in DMD follow up study
This study will be investigating the effects of testosterone being used over the course of 2 years to bring on puberty in boys with DMD. Puberty is often delayed in boys with DMD, and is a side effect of taking steroids, which are part of the Standards of Care. This study will be looking not only at the physical effects of taking testosterone, but also the emotional and psychological effects.
Age
12
-
17
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
Sarepta Extension Study for Casimersen or Golodirsen
This is an open-label, non-randomized extension trial for patients who have already taken part in the trials testing the drugs, casimersen or golodirsen, to evaluate the effects of their long term use.
Age
7
-
23
years
Mutation Specific
Mutation specific therapies
Muscle Biopsy
Required
Muscle Biopsy Required
Ambulation
both
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
