Clinical Trial Finder

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

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

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.

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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

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

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

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

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

Hydrotherapy in DMD

Hydrotherapy for health in boys and adolescents with Duchenne muscular dystrophy

Hub Summary

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.  

We are running a study to try to better understand the impact that hydrotherapy might have on young people with DMD, and whether or not it offers any benefits to their physical and mental wellbeing. We will use the findings from the study to develop simple guides to advise on activities and exercise while in the water.