AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Duchenne Muscular DystrophyOpen-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular...
Duchenne Muscular DystrophyThe study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.
Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device...
Scoliosis NeuromuscularDuchenne Muscular Dystrophy1 moreThis study will be conducted without blind method. The portable seat device devised to maintain lumbar lordosis will be made within 1 year after the loss of ambulation in the participants with Duchenne muscular dystrophy with prospective design. In the control group, the presence of scoliosis will be calculated 5 years after the loss of ambulation in participants with Duchenne muscular dystrophy through analysis of retrospective medical records who had not been applied the portable seat device.
A Phase 1/2 of NS-050/NCNP-03 in Boys With DMD (Meteor50)
Duchenne Muscular DystrophyThis is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD mutation amenable to exon 50 skipping.
CRT-P or CRT-D in Dilated Cardiomyopathy
CardiomyopathyDilated1 moreThe ICD-Reality study is a non-commercial, investigator-led, multicenter, prospective, randomized, controlled trial. We aim to determine the effect of CRT-D or CRT-P implantation in non-ischemic cardiomyopathy and heart failure patients. The reason why we initiated this trial is the lack of evidence-based treatment for the significant number of these patients. In these patients, 5-year mortality remains as high as 20% despite recent therapeutic advances. Based on currently available evidence, because of a significant decrease in mortality due to modern pharmacotherapy, it is not certain which of these patients should receive a CRT-P and who should receive a CRT-D. No dedicated and adequately powered trial has addressed this important question. We hypothesize that patients with symptomatic HF, LVEF ≤35%, without left ventricular mid-wall fibrosis on LGE-CMR, will not benefit from CRT-D implantation compared with CRT-P only implantation. If our hypothesis is confirmed, this could provide evidence for the management of these patients with a significant impact on common daily praxis and health care expenditures. We aim to enroll 600 patients in the trial. 924 patients are needed to be screened for these 600 patients to be randomized. Patients with non-ischemic HF visiting an out-patient department and possibly eligible for the trial will have their pharmacotherapy optimized. Patients with a significant amount of fibrosis will be excluded from the study and treated according to local practice with an emphasis on ICD implantation to prevent SCD. After fulfilling all eligibility criteria, including maximally tolerated pharmacotherapy, subjects will be randomized by the physicians who enrolled them in a 1:1 ratio to receive CRT-D or CRT-P implantation. All patients will be followed-up for at least 3 years after the implantation.
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular DystrophyThis is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
Duchenne Muscular DystrophyDuchenne Muscular Dystrophy is a genetic disease that causes progressive muscle weakness. There is now substantial evidence that boys with this disease do not demonstrate age-related gains in their cognitive skills. The goals of this study are (i) to use a technology-enabled neurobehavioral assessment called National Institutes of Health Toolbox Cognition Battery (NIHTB-CB) to assess brain development over time; (ii) engage with key-stakeholders to understand how neurodevelopmental problems like attention-deficit hyperactivity, autism spectrum affects individuals (and/or) families, so that we can understand meaningful effects of a potential treatment at an individual level, and (iii) to investigate using brain magnetic resonance imaging (MRI) changes in brain connectivity.
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously...
Duchenne Muscular DystrophyThe FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.
A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping...
Duchenne Muscular DystrophyThe primary purpose of the study is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). The study consists of 2 periods: A Screening Period (up to 45 days) and a Treatment and Observation Period (16 weeks).
Bicycle Training on Ventilatory Functions in Duchenne Muscular Dystrophy
Duchenne Muscular DystrophyThe purpose of the study is to investigate the effect of bicycle ergometry training on ventilatory functions in boys with Duchenne muscular dystrophy.