View clinical trials related to Muscular Atrophy.
Filter by:The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
Single-centre observational study over one year investigating the mechanisms of muscle homeostasis in patients with acute skeletal muscle atrophy following major aortic surgery
IO-SMA-Registry is a prospective, longitudinal and observational study which objective is to collect prospectively information on longevity, psychomotor development and respiratory function of patients with infantile-onset spinal muscular atrophy.
Rotator cuff disease is one of the most prevalent musculoskeletal conditions across the world. Patients with chronic rotator cuff tears often have substantial muscle atrophy and fatty infiltration. Surgical repair of the tear does not reverse the atrophy, and many patients continue to experience weakness, pain, and a persistent reduction in the quality of life. An important limitation in our ability to successfully rehabilitate these injuries postoperatively and return patients to normal function has to do with the poor quality of the muscle and tendon after rotator cuff repair. The stromal vascular fraction (SVF) of subcutaneous adipose tissue is highly enriched with cells (SVFCs) that can both directly participate in tissue regeneration by differentiating into myogenic and tenogenic cells, and indirectly by secreting growth factors and small molecules which activate pathways associated with healthy tissue regeneration. High numbers of autologous SVFCs can be isolated using the cost-effective, intraoperative Icellator (Tissue Genesis, Honolulu, HI) point-of-care system. This clinical trial will be determine if the use of SVFCs can enhance outcomes for patients who are undergoing surgical repair of a torn supraspinatus rotator cuff.
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
The primary objective of this study is to investigate the natural history of spinal muscular atrophy (SMA) types 2 and 3 patients in Taiwan. This study will provide further insights into the clinical course SMA. Several analyses will be conducted regarding overall survival, demographic characteristics, motor function, respiratory and nutritional support, and genotype and phenotype correlation.
The aim of this randomised, double-blind, placebo-controlled, parallel groups trial is to investigate the effects of 12 weeks resistance exercise and whey protein supplementation on energy metabolism, markers of appetite, inflammation and hormonal response and body composition and strength and functional performance. Generally healthy, retired men aged 60-80 years will be recruited (n = 52 in total, n = 13 per group). Participants will be randomised to either: a) control group, b) whey protein supplement group, c) resistance exercise + control group or d) resistance exercise + whey protein supplementation group.
This is a retrospective, observational study and will investigate the clinical predictive value of and change in muscle quantity and quality in critically ill patients with severe respiratory failure requiring veno-venous extracorporeal membrane oxygenation (VV-ECMO).
This is a safety and tolerability study investigating the effect of an amino acid formulation in healthy volunteers during and after limb immobilization.
Several studies have shown that lean mass, in particular muscle mass, is an excellent predictive survival factor in many diseases. A better knowledge of the mechanisms responsible for muscle atrophy and the identification of atrophic process markers are deeply needed for the development of new anti-atrophic therapies. Either as drugs used to treat several medical conditions or as endocrine hormones released in response to many stress situations (e.g., sepsis, cancer, insulinopenia…), glucocorticoids (GC) are recognized to play a major role in skeletal muscle atrophy. Indeed, the inhibition of GC action by a receptor antagonist (RU486) or by muscle-specific invalidation of the GC receptor inhibits the muscle atrophy in these stress situations. Therefore, all these data clearly indicate that GC play a major role in skeletal muscle atrophy observed in several conditions. Emerging evidence has revealed that the skeletal muscle has a secretory function. Human skeletal muscle secretome was first estimated at about 300 proteins by computational analysis and proteomic analysis have recently confirmed these results. Some of these secreted proteins, conceptualized as myokines, can act locally on muscle cells through autocrine/paracrine loops and on surrounding tissues such as muscle blood vessels or can be released into the blood stream to produce systemic effects. One prominent example is interleukin (IL)-6 which is released into circulation by contracting skeletal muscle and can regulate metabolic and inflammatory processes. As IL-6, several other potential myokines have been identified including IL-8, IL-15, insulin-growth factor I (IGF-I), follistatin-like 1 (FSTL1) or fibroblast-growth factor (FGF)-21. Moreover, secreted proteins may also reflected metabolic changes which take place in muscle cells. Indeed, myoblast differentiation is accompanied by dramatic changes in the secreted proteins profile as increased expression of Semaphorins, IGF-I, matrix metalloproteinase (MMP)-2 or Collagens. Thereby, the investigators hypothesized that skeletal muscle atrophy induced by GC is associated with specific alterations of the muscle secretome. The aim of this project is to identify the GC-induced changes in the secretome of human skeletal muscle cells in culture (in vitro approach) and to determine how these changes translate into the circulation of subjects exposed to high concentrations of GC (Cushing's syndrome) (in vivo approach). Characterization of these changes in human subjects should allow to better understand the cellular mechanisms involved in muscle atrophy and might lead to identify circulating biomarkers associated with skeletal muscle atrophy, as telopeptides are for bone tissue.