Active clinical trials for "Muscular Dystrophies"

The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).

While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age. The primary reason for the delay is that daily corticosteroid use has many side effects including short stature, obesity, and osteoporosis. A recent randomized blinded study of weekend oral corticosteroid use over one year showed equal improvement in strength with fewer side effects, particularly as related to growth and cushingoid changes. The investigators will test the efficacy of oral weekend corticosteroid use in infants and young boys with DMD who are under age 30 months. The investigators have demonstrated that the Bayley-III Scales of Infant development shows that infants and young boys in this age group who are untreated decline in abilities when compared to their peers. Here, in this Phase 2 historically controlled trial, the investigators will use these two measures and treat boys at five Muscular Dystrophy Association-DMD centers

The primary objective of this investigation is to assess the effectiveness of transcutaneous electrical nerve stimulation applied using VECTTOR to reduce the symptoms of Duchenne Muscular Dystrophy and reduce the impact of DMD upon the participants' quality of life. The primary outcome measures will include: increased muscle strength, increased range of joint motions and improved sleep parameters of ASI, N3 and REM.

In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime. Sodium/proton type 1 exchanger (NHE-1) inhibition is an innovative pathway that has proved to efficiently prevent the accumulation of muscle damage (inflammation and fibrosis) in animal models of muscular dystrophies and heart failure. Based on prior safety and efficacy results in animal and humans, NHE-1 inhibition with Rimeporide represents a new therapeutic approach with no restriction on age and on genetic subtypes which could be combined to other treatments that restore or augment dystrophin.This study examines the safety and tolerability and effects on the muscles of rimeporide, in patients aged 6 to 14 years with Duchenne Muscular Dystrophy (DMD).

The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.

Primary Objective The primary objective of the study was to establish the effects of givinostat versus placebo administered chronically over 18 months to slow disease progression in ambulant DMD subjects. Secondary Objectives The secondary objectives of this study were: To assess the safety and tolerability of givinostat versus placebo administered chronically in DMD subjects To evaluate the PK profile of givinostat administered chronically in DMD subjects To evaluate the impact on quality of life (QoL) and activities of daily living of givinostat versus placebo administered chronically.

This is an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study will consist of 2 Cohorts. Cohort A will include participants ages 3 months to 3 years, and Cohort B will include participants ages 4 to 7 years old. All participants in the study will receive IV delandistrogene moxeparvovec.

A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of care with glucocorticoids. Furthermore, the investigators plan to include 6-20 non-ambulant patients who do not receive glucocorticoids (as parallel group B), 10 to 16 years old, to obtain efficacy and safety data in a broader DMD population. All patients will receive 20 mg of tamoxifen (TAM) or placebo once daily during 48 weeks. An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE.

Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes. Emerging treatment approaches aimed at improving activity and participation have shifted from focusing only on impaired body functions towards the performance of functionally meaningful activities within the youth's natural environment. It is unclear, however, whether targeting intervention at the activity/participation level can, at the same time, result in improvement of personal functional skills (e.g., reaching) and body functions (e.g., range of motion) -components also important to address and maintain within the rehabilitation process. Together with key community-based stakeholders including youth/parents, clinicians, and policy-makers, the investigators plan, therefore, to examine whether engaging in a 6-week community based activity (e.g., joining a sledge hockey team, boccia) can lead to a significant improvement in three key body functions: motor, cognitive and affective functions. Eight participants with physical disabilities will take part in the study and engage in an activity program of their own choice. Changes in their body functions (e.g., movement-related functions, attention, behavior, mood) will be measured multiple times before, during and after the engagement in an individualized activity/program. Findings of this pilot study analyzed with input from key stakeholders can advance the investigators understanding about methods for testing complex and unique individual-based interventions. This can guide clinicians, families and policy-makers to select effective approaches that not only promote participation but can also facilitate additional (motor and mental) benefits from one single intervention. Such findings may also reduce the burdens on the healthcare system as well as on the youth and families.

The study aimed to assess the safety and, partially, the efficacy of dietary supplementation of a flavonoids-, DHA- and EPA-based natural supplement in non-ambulant DMD boys and in a cohort of LGMD and FSHD patients to compare its effect in MDs of different aetiology and to eventually highlight any differences in inflammatory involved pathways. To assess safety, patient's laboratory parameters were monitored and adverse events recorded, while efficacy was evaluated through performance scale questionnaire and strength measurement (6 minute walking test and Biodex System 4 Dynamometer parameter evaluation). This study was conceived as proof of principle for the safe use of flavonoids/omega3s-based compound as an adjuvant in the management of neuromuscular disorders; besides, its efficacy in alleviating symptoms linked to secondary effects of genetic mutation as inflammation, muscular pain and weakness was assessed.