View clinical trials related to Muscular Diseases.
Filter by:This study is planning to evaluate the safety and clinical efficacy of medium-chain fatty acid capsules (food-grade CNT-02) in subjects with primary triglyceride deposit cardiomyovasculopathy (TGCV) and neutral lipid storage disease with myopathy (NLSD-M) associated with adipose triglyceride lipase (ATGL) genetic defects.
The present trial is designed to assess the safety and efficacy of TNX-102 SL 2.8 mg tablets, taken daily at bedtime after 12 weeks of treatment in patients with fibromyalgia. The use of low-dose sublingual formulation of cyclobenzaprine (TNX-102 SL) dosed nightly for fibromyalgia is supported by the results of TNX-CY-F202 Phase 2b study -- the results provide strong evidence that TNX-102 SL 2.8 mg dosed nightly results in beneficial effects upon pain, sleep and other FM symptomatology.
In adults, muscle diseases are usually chronic long-term conditions that do not have a definitive cure. Supportive care has been shown to reduce complications from muscle disease and improved survival in some cases. However, there has been limited research to evaluate interventions that may improve quality of life (QoL) with this patient group. The QoL of those with MD is not just affected by the severity of their MD but also a variety of psychological variables. Based upon the knowledge of these psychological variables the investigators feel that a particular type of psychological intervention known as "acceptance and commitment therapy" (ACT) could potentially improve QoL in those with MD. The investigators therefore propose to test whether ACT does in fact improve QoL in those with MD by randomising 154 patients to receive either standard medical care plus a guided self-help ACT programme, or standard medical care only.
The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.
The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.
The aim of the project is to develop new Magnetic Resonance (MR) imaging techniques for better diagnosis and monitoring of patients with muscular disorders. Muscle quality in patients with Late Onset Pompe Disease (Acid Maltase Deficiency type 2) and in patients with Myotonica Dystrophy will be evaluated, by determining muscle strength in relation to muscle size and muscle strength in relations to fat-muscle ratio.
This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.
The objective of this study is to determine whether the administration of N-acetylcysteine (NAC) improves oxidative stress. To determine this, the study will assess the impact of oral treatment on the balance between reduced and oxidized form of glutathione in erythrocytes of peripheral blood.
Patients will be randomly assigned to two separate groups. Both patient groups will be evaluated using the WHOQOL-BREF questionaire. Evaluations will be carried out just before the study begins and at intervals of six months during a period of one year for a total of three evaluations. Group one will be made up of patients who will begin homeopathy or acupuncture or anthroposophic medicine treatment and patients from group two will start homeopathy or acupuncture or anthroposophic medicine six months after the beginning of the study. Each one of these three research branches will be done independently (Homeopathy versus control; Acupuncture versus control; Anthroposophic Medicine versus control). Conventional medical treatment will be available for all patients in both groups. A data sheet will be filled out on each patient with data as to age, education level, marital status and the patient's own perception of their health status. During the interviews the interviewer will have no influence whatsoever over the respondents' answers. Integrative/complementary or traditional medicine doctors do not participate in the research and will not be informed about which patients are participating in this project, and as such the research will be blinded for these doctors. Minitab software, version 16, was used for sample calculation through the module Power and Sample Size. Sample calculation option for paired t-tests before and after intervention demonstrated the need to randomize 906 patients: 450 patients for homeopathy branch (225 patients in the homeopathy group and 225 patients in the control group), 228 patients for acupuncture branch (114 patients in the acupuncture group and 114 patients in the control group), and 228 patients for anthroposophic medicine branch (114 patients in the anthroposophic medicine group and 114 patients in the control group).
Adult patients with suspected or confirmed idiopathic inflammatory myopathy (IIM) will be recruited. Patients will be approached, consented, have baseline demographics, diagnostics and disease activity measures recorded, and blood taken. The collection of data and biological material will mirror usual clinical practice as far as possible. Subjects will ideally attend further visits at 3, 6 and 12 months to have bloods taken, outcome measures recorded and questionnaires completed. In addition, blood, muscle biopsies and imaging undertaken as part of usual care will also be collected for research purposes to measure a number of biomarkers for the assessment of diagnostic accuracy and clinical utility evaluation. As per usual practice, a muscle biopsy will be performed at baseline, and a further biopsy offered at 6 months to assess treatment response. A magnetic resonance (MR) muscle protocol will also be performed as per usual clinical practice, and a gadolinium-enhanced MR heart scan offered. Both these scans will be repeated at 6 months. An existing electronic database entry system will be used for data entry and capture on an anonymised basis. The study will thus be based around diagnostic evaluations and outcome measures to improve quality of care in IIM.