View clinical trials related to Muscular Diseases.
Filter by:The purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with IIM who previously participated in ARGX-113-2007. Secondary objectives include efficacy measures of efgartigimod PH20 SC in participants with IIM.
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases.
The purpose of this research study is to test the feasibility and response of a new exercise protocol on improving physical activity in adolescents with cerebral palsy.
This is a Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Froniglutide in Patients With Idiopathic Inflammatory Myopathy ("FROniGlutide Study")
In patients with myositis early immunomodulation by intensive treatment ("hit-early/hit-hard" principle) may induce faster reduction of disease activity and prevent chronic disability. Intravenous immunoglobulin (IVIg) in addition to standard treatment with glucocorticoids may be beneficial for this purpose: add-on IVIg improved symptoms in steroid-resistant myositis, and first-line monotherapy IVIg led to a fast and clinically relevant response in a pilot study in nearly 50% of patients with myositis.
The objective of this study is to evaluate acute changes of cardiac troponin (and other cardiac biomarkers) and mid-term biovariability in patients with cardiomyopathy associated with chronic skeletal muscle disease. The specific aims of the study are: Firstly, to evaluate the feasibility of the ESC 0/1 hour protocol for rule-in and rule-out of a non-ST-segment elevation acute coronary syndrome (NSTE-ACS). Secondly, a) to determine reference change values (RCV) to characterize physiological biovariability, b) to differentiate acute from chronic high-sensitivity cardiac troponin T (hs-cTnT) elevations.
Objective: To collect information and biospecimens (such as blood, muscle, and skin samples) that will be used to research causes and treatments of inflammatory muscle diseases. Eligibility: People aged 12 years and older with suspected or confirmed inflammatory muscle disease. Healthy volunteers aged 18 years and older are also needed. Design: Participants will have at least 1 clinic visit. Each visit will last 4 to 8 hours. Some may return for additional visits. All participants will undergo these procedures (unless they are unable to): - Physical exam, including blood and urine tests. - Magnetic resonance imaging (MRI) scan of the thigh. Participants will lie still on a table with padding around 1 thigh. The table will slide into a tube. The scan will last for approximately 40 minutes. Some procedures are optional: - Muscle biopsy. An area of skin will be numbed. A quarter-inch cut will be made. Several pieces of muscle tissue, about the size of grains of rice, will be removed. - Skin biopsy. An area of skin will be numbed. A piece of skin about a quarter inch in diameter will be removed. - Genetic testing. Some of the samples collected may be used for genetic testing.
The goal of this clinical trial is to test ketone bodies in healthy elderly and young individuals. The main question it aims to answer are: • Do ketone bodies improve skeletal muscle function? Participants will ingest a ketone monoester and skeletal muscle function will then be evaluated by: - Special magnetic imaging techniques - Intravenous infusion of tracer-marked nutrients - Performance tests on a ergometer bike and in a dynamometer Researchers will compare the outcomes between within the young and elderly groups and between the young and the elderly group to investigate if age has an effect on the outcomes.
The goal of this observational study is to assess if diabetes and obesity are independently related to functional and structural muscle deficits, and how muscular deficits relate to metabolic properties of diabetes and obesity. All studies will include clinical muscle strength and contractile examinations, functional tests, and MR imaging and spectroscopy techniques. The main questions this project aims to answer are: 1. Is chronic hyperglycemia in type 1 and 2 diabetes associated with functional and structural deficits of skeletal muscles unrelated to the presence of neuropathy? 2. Is obesity associated with functional and structural impairments of skeletal muscles unrelated to the presence of type 2 diabetes ? 3. Does weight loss improve muscle metabolic flexibility and economy and modify skeletal muscle function and structure in obese subjects with and without type 2 diabetes? The project will include three studies, intended to answer the hypotheses listed above: Study 1: Evaluation of functional and structural muscular deficits of diabetic myopathy in relation to prolonged hyperglycemia prior to and 6 months following glycemic improvement in patients with type 1 and 2 diabetes Study 2: Functional and structural muscular deficits in severely obese subjects with and without type 2 diabetes prior to assisted weight loss. Study 3: Changes in functional and structural muscle properties following assisted weight loss in severely obese subjects with and without type 2 diabetes - a 1-year follow-up study.
This is a longitudinal study in a cohort of patients with a genetic diagnosis of Primary Mitochondrial Myopathy to describe the natural history of the disease and identify clinical, biochemical, molecular, and radiological variables that allow evaluation of the severity and progression of the disease and may be useful in future clinical trials.