View clinical trials related to Muscular Disorders, Atrophic.
Filter by:Rationale: Situations such as fractures of the lower extremity can necessitate a prolonged period of immobilization in otherwise healthy individuals. Long-term immobilization of the lower extremity has shown to cause significant reductions in skeletal muscle mass, already occurring during the early stages of disuse. Accordingly, feasible strategies for attenuating this loss of muscle during disuse need to be pursued. Local neuromuscular electrical stimulation (NMES) offers such a potential strategy but, as yet, remains untested during prolonged muscle disuse in a clinical setting. Objective: To investigate whether twice daily local (gastrocnemius/soleus) NMES attenuates muscle loss during 2 weeks of unilateral ankle immobilization. Study design: Randomized, parallel (two groups) study design. Study population: 30 adults (18-65 y) with any form of closed ankle fractures needing surgical treatment. Intervention: Twice daily neuromuscular electrical stimulation (NMES) or no intervention. Main study parameters/endpoints: Primary: Calf muscle (gastrocnemius) cross sectional area (CSA) as determined by CT scan. Secondary: type I and II muscle fiber CSA and SC content, intramuscular triglyceride content and mRNA and protein expression of anabolic signaling proteins.
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study. This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.