Clinical Trial Finder

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

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

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

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

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

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

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. 

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