Clinical Trial Finder

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

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

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

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

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

A Feasibility study of an Online Mindfulness intervention for Duchenne Muscular Dystrophy

Pilot Study of an Online Mindfulness Intervention for Duchenne Muscular Dystrophy: Exploring Feasibility, Engagement, and Outcomes

Hub Summary

This study is led by Manchester Metropolitan University and Duchenne UK and approved by the Manchester Metropolitan University ethics committee.

Scientific Rationale
Almost all men with DMD experience pain, which is a contributing factor to lower quality of life (QoL), particularly within the mental health domain. Psychosocial health issues are significantly more prevalent in DMD, with anxiety prevalence reaching 33%, depression prevalence at 19%, and the prescription of associated medication to 28% of men with DMD. Although poor mental health is multifactorial in nature, pain has been associated with both anxiety and depression in DMD. Beyond the behavioral support for higher prevalence of autism spectrum disorders, mental health support and pain management for those adult transitioners and men with DMD lacks emphasis, with currently only cursory mentions to mental health and pharmacological solutions to pain being advocated in the most recent standards of care.

Given the effectiveness of Mindfulness Based Interventions (MBI) within other conditions, MBI may represent a potential intervention for anxiety, depression, pain and QoL in DMD. In conditions with neurological impairment, interventions to support mental health are considered fundamental to improving quality of life. Relevant to DMD particularly, MBI have been shown to:

•    Improve mental health and quality of life in people with spinal cord injury
•    Reduce psychological distress
•    increase self-protective awareness of early physical symptoms and increase proactive condition self-management and autonomy

The current research aims to assess the feasibility of an eight week online mindfulness intervention for people living with DMD (co produced using PPIE and informed by evidence on the efficacy of mindfulness in other chronic health conditions).

Study Number: N/A