Clinical Trial Finder

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

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

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

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

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

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

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

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