Clinical Trial Finder

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

Santhera (SIDEROS) [TERMINATED]

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

BioMarin - BMN 351

A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy

Hub Summary

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. 

Study Number: NCT06280209

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

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

DYSTANCE 51 [TERMINATED]

A Randomised, Double-blind, Placebo-controlled, Efficacy and Safety Study of Suvodirsen (WVE-210201) With Open Label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy

Hub Summary

Please note that Wave have stopped the development of this drug after the Phase 1 Open-label extension failed to meet its primary endpoint. 

DYSTANCE 51 is a phase 2/3 clinical trial designed to evaluate the efficacy and safety of WVE-210201 (suvodirsen) in ambulant boys with DMD mutations amenable to exon 51 skipping.

DYSTANCE 51 is comprised of two phases, a placebo-controlled phase and an open-label phase. In the placebo-controlled phase, patients will be randomized to receive suvodirsen 3 mg/kg, suvodirsen 4.5 mg/kg or placebo for 48 weeks. Dystrophin protein levels and functional outcomes will be assessed for each patient throughout the initial 48-week treatment period.

Each participant will have two biopsies in total, one at baseline, and one at either week 12, 22, or 46.

Following completion of the placebo-controlled phase of the study, patients will enter the open-label phase to receive ongoing treatment with suvodirsen. There will be no placebo or biopsies in the open label phase. However, functional assessments will continue.

Study Number: NCT03907072

Sarepta Extension Study for Casimersen or Golodirsen

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Hub Summary

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. 

This trial focuses on patients with Exon 45 and Exon 53 mutations, and will involve weekly intravenous infusions of 30mg/kg for up to 2 years. 

Study Number: NCT03532542

Genethon - Natural History of Duchenne Muscular Dystrophy

A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 5 to 9 Years

Hub Summary

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.

Study Number: NCT03882827

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

Sarepta - EMBARK

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy (EMBARK)

Hub Summary

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.

Study Number: NCT05096221

BIND Study

Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5) Part 1: a Multicentre Online Phenotyping and Neurobehavioural Data Collection Study

Hub Summary

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. 

Study Number: NCT04583917

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

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

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

Roche - ENVOL

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Hub Summary

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.

Study Number: NCT06128564

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. 

Genethon- Microdystrophin Gene Therapy (GNT-016-MYDF)

GNT-016-MYDF: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation of selected dose part and then by a long-term follow-up part in ambulant boys aged 6 to 10 years with DMD

Hub Summary

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.

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leading to absence (almost absence) of functional dystrophin, a key protein that prevents muscle cell damage during physiological contractions. Conceptually, adding a functional copy of the gene to muscle cells would, in principle, treat the underlying cause of the disease. However, there are two main obstacles to overcome to achieve this. First, a vehicle (vector) is needed to transport the functioning gene to target muscle cells. AAV capsids are frequently used as the vehicle to transport genes in other gene therapy products. The specific type of AAV capsid in GNT0004 is called AAV8. The second obstacle to overcome is the size of the dystrophin gene. It is one of the largest genes in our bodies and because it is so large it cannot fit inside the AAV capsid. To address this obstacle, the Genethon research group has developed a miniaturized but functional version of dystrophin (Microdystrophin).

The principle of GNT0004 is to deliver an optimized microdystrophin protein to target tissues (heart and skeletal muscles) in DMD subjects. It is expected to significantly delay or slow down the progression of the disease in humans in a similar manner to how it has shown to be able to do this in animal models with the disease.

In order to help GNT0004 reach muscles throughout the body, GNT0004 will be given as a one-time intravenous (IV) infusion.

Study Number: EUDRACT N° 2020-002093-27

Santhera SIDEROS Open Label Extension [TERMINATED]

Phase III Study with Idebenone in Patients with Duchenne Muscular Dystrophy

Hub Summary

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. 

Study Number: NCT03603288

D-Brain

Defining outcome measures for behavioural and emotional problems in dystrophinopathies

Hub Summary

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.

To investigate this, we are conducting a large cohort study to identify which part of the DMD gene is responsible for the development of these complications and help us further define and develop robust clinical assessments which will outline psychological profiles for DMD and BMD patients.

The study will involve two consecutive visits, which will include assessment of cognition (thinking and problem solving) and behaviour (inattention, anxiety, hyperactivity, etc.) . These two visits will be repeated after 6 months. For those interested there is also the possibility of participating in the MRI component of the study.

This study will cover your travel expenses and will provide you with detailed feedback on your son’s potential difficulties that can be used to receive educational support, to get a referral for local authorities or for your own personal use as it might give you some useful insights about your son’s behaviour.

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

Sarepta - MIS51ON

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients WITH DUCHENNE MUSCULAR DYSTROPHY With Deletion Mutations Amenable to Exon 51 Skip

Hub Summary

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. 

Study Number: NCT03992430

Pfizer - CIFFREO

A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Hub Summary

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. 

Two-thirds of the participants will receive the treatment. One-third will be randomly allocated to the placebo arm, but will be able to receive the treatment in the second year, so long as it remains safe to do so.

Please note that patients will need to be on daily steroids for 3 months before screening, to be eligible. They will also be able to be recruited to the trial up until their 8th birthday. For more information about the recruitment process for gene therapy trials, please click here. 

EU Clinical Trial Register number: 2019-002921-31

Study Number: NCT04281485

Phase 3 PolarisDMD Trial [TERMINATED]

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

Hub Summary

 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.

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.

To receive up-to-date information about this trial, please sign up to the Catabasis newsletter. 

Study Number: NCT03703882

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

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.

Please note this protocol was amended early 2019.

Study Number: NCT02851797

Catabasis - Galaxy DMD [TERMINATED]

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Hub Summary

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). 

This trial is looking at the safety, tolerability and durability of taking edasalonexent over a long period of time. Edasalonexent has been shown to delay the progression of DMD and could provide an alternative to steroids. It is in tablet form and taken orally (by mouth) three times a day. 

Study Number: NCT03917719

Italfarmaco - Givinostat Extension

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Hub Summary

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. 

GIVINOSTAT is a drug that may help to promote muscle regeneration and reduce inflammation and fibrosis in DMD patients. 

This extension is expected to last until the drug receives the necessary approvals and is available on the market or the study needs to be stopped due to safety and/or efficacy reasons.

Study Number: NCT03373968

Roche- RO7239361 [TERMINATED]

Clinical trial to Evaluate the Efficacy, Safety and Tolerability of RO7239361 in Ambulatory Boys with Duchenne muscular dystrophy

Hub Summary

Please note the development of this drug was stopped after this trial failed to reach its objectives.

This placebo-controlled study is designed to assess the efficacy, safety and tolerability of two different doses of RO7239361 in ambulatory males with DMD. RO7239361 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

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

Vamorolone Phase II 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

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

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

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

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. 

BIND 2

Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5 and WP6) Part 2: a Neurobehavioural and MRI Study

Hub Summary

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.

The investigators will recruit both patients who have completed a related online study (ClinicalTrials.gov Identifier NCT04583917) and patients directly recruited from participating clinics or research settings. These patients will undergo a structured cognitive and neurobehavioural assessment. A subgroup of patients assessed in the research setting will be invited to also attend a second visit involving a magnetic resonance imaging (MRI) scan of the brain.

The investigators aim to recruit 80 participants in the UK and the number of participants in the remaining countries will be 190 patients.

Study Number: NCT04668716

Dyne Therapeutics - DYNE-251

Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Hub Summary

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.

Study Number: NCT05524883

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

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

Sarepta ENVISION

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

Hub Summary

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.

Study Number: NCT05881408

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