View clinical trials related to Muscular Atrophy.
Filter by:The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.
Objectives: This study aims to examine the use of low frequency (2Hz), low amplitude (intensity just produce visible muscle contraction), and long duration (2x3 hrs/day) neuromuscular electrical simulation (NMES) in attenuating the effects of muscle atrophy resulted from disuse. Design and subjects: The study is a randomized, double-blind, controlled, and parallel group study. Subjects with stable chronic obstructive pulmonary disease (COPD) will be included. Intervention: Subjects will be randomized to 3 groups to receive different NMES program over the quadriceps and calf muscles: (i) the proposed NMES program; (ii) conventional NMES program (50Hz, 30 min/day), or sham group for a period of 8 weeks. Outcome measures:The effectiveness of the NMES will be evaluated by the improvement in muscle cross-sectional area (CSA), muscle performance (muscle strength, muscle shortening velocity and muscle activation testing), functional performance (6 min walk) and subjects' rating of the perceived acceptability of the stimulation protocol. Data analysis: Baseline characteristics of the intervention and sham groups will be compared using one way ANOVA. Two-way mixed repeated measures analysis of variance will be performed to examine the differences between groups over time for all the outcome variables. The significance level is set at p < 0.05. Expected results: The investigators hypothesize that the proposed new paradigm of NMES would be more effective in improving muscle cross-sectional area (CSA), strength, endurance, and exercise tolerance.
Neuromuscular electrical stimulation in pectoral muscles (fibres of the pectoralis major muscle bilaterally) and rectus abdominis muscles (bilaterally) preserves / decreases the loss of muscle mass.
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.
Limb muscle dysfunction, characterized by atrophy and weakness, is amongst the most troublesome systemic consequences of chronic obstructive pulmonary disease (COPD) leading to poor functional status and premature mortality. One prevailing hypothesis stipulates that the deterioration in muscle structure and function during COPD results from a spillover of inflammatory mediators from the lungs to the systemic circulation and then to the muscles.
The primary aim of the study is to evaluate consequences of frailty in critically ill patients. We hypothesize that a higher frailty index (based on published questionnaires) predicts a longer surgical intensive care unit and hospital length of stay, less ventilator-free days and a higher likelihood of an adverse discharge disposition. Our secondary aim is to identify muscle-size derived variables that can be used to predict frailty. We hypothesize that a low skeletal muscle mass measured by ultrasound can be used to quantify frailty, and to also predict the outcome of SICU patients, expressed as longer stay in the surgical intensive care unit and longer stay in the hospital, less ventilator-free days and a higher likelihood of an adverse discharge disposition. Our third aim is to examine potential triggers of muscle wasting in critically ill patients. Muscle wasting will be assessed by repetitive ultrasound measurements of muscle mass. We hypothesize that a significant decrease in skeletal muscle mass predicts longer stay at the surgical intensive care unit and longer hospital length of stay, less ventilator-free days and adverse discharge disposition.
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy after 13 weeks; and to estimate the Maximum Tolerated Dose (MTD) of orally administered branaplam; and to identify the dose that is safe for long term use as well as that can provide durable efficacy optimal dosing regimen in patients with Type 1 SMA.
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them. Moreover a lot of patients with NMD have so poor condition that even short transportation is able to worse it. Such situation forces researchers to limit a region of recruitment for clinical trials and to exclude from trials more severe subgroup of patients, which cause additional issues especially for rare diseases. The purpose of this study is to prove hypothesis about possibility to reliably monitor patient condition remotely, without trial site visiting. Visit-free study design is potentially able to widen eligible patient population and to decrease patient dropout rate as well as burden of numerous assessments. Meanwhile assessment frequency could be increased enabling monitoring of short fluctuations in patients' condition. Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above issues are completely applicable. Direct current stimulation (DCS) of neural structures is well studied and safe intervention, however, its effects on SMA patients' strength and durability has not been reported for today. The investigators suppose that investigation of DCS action in SMA patient population is an adequate model for visit-free design feasibility, reliability and sensitivity evaluation.
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).