An Extension Study of MOM-M281-004 to Evaluate the Safety, Tolerability, and Efficacy of M281 Administered...
Generalized Myasthenia GravisThe purpose of this study is to evaluate the long-term safety and tolerability of M281 in participants with generalized myasthenia gravis (gMG)
Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia...
Myasthenia GravisThis is a multicenter, randomized, double blind, placebo-controlled, Phase 3 study to evaluate the efficacy and safety of tolebrutinib compared with placebo in adult participants aged 18 to 85 years old with moderate-to-severe gMG, who are receiving Standard of Care (SoC). The double-blind (DB) treatment period of 26 weeks will comprise of 7 site visits followed by a 2-year open label extension (OLE) period with quarterly visits. The efficacy of tolebrutinib versus placebo during DB period will be assessed by clinical evaluations, that include scales based on physician examination or direct participant feedback i.e., patient reported outcomes (PROs). These evaluations will continue during the OLE in order to measure long term efficacy and safety.
Trial of Orencia in Patients With Myasthenia Gravis
Myasthenia GravisThis pilot research study is being done to see if the drug abatacept (Orencia ®) will be helpful in treating patients with myasthenia gravis (MG) who do not respond satisfactorily to other drugs that are used to suppress the immune system. Abatacept has been successful in treating experimental MG in laboratory animals, and this study is to determine its effectiveness in patients with MG.
Evaluating Muscle Weakness Improvement With Lorcaserin in ICU
Muscle WeaknessSepsisICU acquired muscle weakness is a significant problem in patients recovering from critical illness. This trial will evaluate the safety and efficacy of a drug in improving muscle weakness in critically ill patients.
SARCOPEDIA - Sarcopenia Diagnostics in Aging Medicine
SarcopeniaMuscle Weakness1 moreMusculoskeletal aging is one of the major responsibilities and challenges for public health. In particular, sarcopenia correlates with an increased risk of falls and increased morbidity and mortality. With regard to screening for sarcopenia, the guidelines of the European Working Group on Sarcopenia in Older People (EWGSOP2) refer to algorithmic case finding, diagnosis, and quantification of the severity of sarcopenia in clinical. While functional measurements are more difficult to standardize and associated with higher variability, Dual-energy X-ray absorptiometry (DXA) is considered a highly accurate method, even referred to as the "gold standard" to determine muscle mass in the scientific literature. Nevertheless, DXA, which is routinely used, shows inconsistent correlation with functional decline in muscle strength. Another method of muscle quantification is bioelectrical impedance analysis (BIA), a simple, portable instrument that is more readily available and applicable due to its lower cost. However, it tends to overestimate muscle mass and is also more susceptible to a person's hydration status. Because of these difficulties, recent research has focused on the potential of using shear wave elastography. This method indirectly serves to quantify the rapid type II muscle fibers in order to make statements about the muscle quality because an age-related decrease in type II muscle fibers is associated with a more frequent fall frequency. First, the investigators will define three different categories according to the EWGSOP 2 guidelines based on the muscle strength (grip strength, assessed by pneumatic hand dynamometer) and muscle mass (Appendicular skeletal muscle mass, assessed by BIA): "No Sarcopenia", "Probable Sarcopenia" and "Confirmed Sarcopenia". Within these categories, the Investigators would like to establish a multivariate data analysis of different functional measurements with quantitative imaging results. This exploratory trial design is intended to improve understanding within the three categories and to test proxy measurements of different patients who are ruled out for common routine measurements due to, for example, cognitive impairment or pre-existing rheumatic disease. This is essential to consider the heterogeneity of the aging society proportionally.
PB125, Osteoarthritis, Pain, Mobility, and Energetics
OsteoarthritisKnee3 moreNuclear factor erythroid 2-related factor 2 (Nrf2) is an important regulator in the body. It controls how well cells protect themselves against stress. PB125 (Pathways Bioscience) is a plant based activator of this important regulator Nrf2. PB125 is made up of three plant extracts (rosemary, ashwagandha, and Sophora japonica) so that it contains these things; 1. Carnosol, 2. Withaferin A, and 3. Luteolin. Carnosol comes from rosemary leaves. Rosemary is a spice often used in Italian foods and grown in many herb gardens all around Fort Collins. Withaferin A comes from the medicinal plant Withania somnifera, also called ashwagandha. Ashwaganda is commonly known as "Indian Winter cherry" or "Indian Ginseng" and it is one of the most important herbs of Ayurveda (the traditional system of medicine in India) used for millennia. Finally, luteolin is found widely in plants including those present in the diet (peppers, onions, celery, herbs/spices). Some people purchase these herbs commercially, and take them on their own for a variety of purposes. Typically, when you buy them, they will be in much higher doses than they are in PB125. What makes PB125 different is that very low doses of each of the 3 components work together-synergistically-to activate Nrf2 and increase the ability of cells to respond to stress. It is unknown if there are any benefits to taking PB125 and the risks are currently unknown. The purpose of this study is to examine changes in muscle, in joint pain, in mobility (standing and walking) and in leg strength that occur after consuming PB125 every day for 3 months. We want to make these measurements in people who have been diagnosed with mild to moderate osteoarthritis-a degenerative joint disease-in their knees.
Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis
Myasthenia GravisMG may be neonatal, congenital, or autoimmune. Neonatal MG arises from transplacental transfer of ACh receptor antibodies from a mother with autoimmune MG to the fetus. Neonatal MG resolves with post delivery clearance of maternal antibodies. Congenital MG results from a genetic defect in the ACh receptor. Patients with congenital MG do not have ACh receptor antibodies. Both neonatal and congenital MG are excluded from this study. Autoimmune MG, which is the most common form of MG, affects approximately 25,000 Americans. Like most autoimmune diseases, it is associated with particular HLA genotypes, has a female predominance, and environmental factors involved in breaking tolerance to the ACh receptor are unknown. Patients with refractory and severe autoimmune MG will be considered candidates for this study. The purpose of this study is to assess the toxicity/feasibility (phase I) of autologous hematopoietic stem cell transplantation for refractory myasthenia gravis.
Nordic Hamstring Exercise After ACL Reconstruction Reconstruction
ACL InjuryMuscle Weakness1 moreAnterior cruciate ligament (ACL) injuries constitute a significant portion of major knee joint injuries sustained by young, active individuals, and significantly increase risk for long-term disability. Yet the recommended solution to restore joint stability following injury--ACL reconstruction (ACLR)--does not prevent post-traumatic knee osteoarthritis (PTOA). Post-traumatic quadriceps (dys)function is a hallmark characteristic following ACLR, reported to accelerate the onset of PTOA after ACL injury, making the recovery of muscle function a primary concern to clinicians. However, hamstrings muscle function is drastically underrepresented relative to the quadriceps in the context of recovery from ACLR, which impedes the ability to develop targeted treatment approaches. Persistent hamstrings weakness is widely reported in patients who undergo ACLR with a hamstring tendon (HT) autograft, which increases ACL strain, and may contribute to higher graft failure rates in this population. To effectively treat muscular impairments, underlying neuromuscular adaptations known to occur in response to ACLR must be targeted. Eccentric exercise is uniquely suited to enhance neuromuscular function. The Nordic hamstring exercise (NHE) is a specific form of eccentric exercise that is clinically relevant and easy to implement, but has not been explored as an intervention for hamstrings neuromuscular dysfunction in patients who undergo ACLR with HT. To establish an evidence-based treatment model, the investigators will use a single-blind, randomized controlled clinical trial to establish the feasibility and efficacy of a 4-week NHE protocol in patients who undergo ACLR with HT. Separate factorial ANOVAs will be used to assess the effects of group (NHE, control) and time (baseline, 4 weeks) on selected outcomes. Effect sizes will be calculated for within- and between-group comparisons. The investigators expect to observe improvements in hamstrings neuromuscular function following the NHE protocol, and that those improvements will be greater than the control group. Additionally, the investigators expect the protocol to be feasible in terms of intervention adherence and patient retention. This study will identify specific barriers to the implementation of NHE in patients who undergo ACLR with HT, and will provide support for the application of an easy to implement clinical intervention able to address a complex neurophysiological problem.
Benefits of Upper Extremity Exercises
Muscle WeaknessPt will be provided with a upper extremity exercise program that will be completed daily during their session with occupational therapy. During the session the Activity Measure-Post Acute Care and an activity tolerance chart will be completed. The data from the outcome measures will be used to determine if there is a benefit to completing a daily exercise program.
Cholecalciferol Supplementation in Strengthening Inspiratory Muscles in Cholecalciferol-Deficient...
Chronic Obstructive Pulmonary DiseaseMuscle WeaknessThis phase IIb randomized trial studies how well cholecalciferol (vitamin D3) supplementation works in strengthening inspiratory muscles in cholecalciferol-deficient patients with chronic obstructive pulmonary disease (COPD). Cholecalciferol supplementation may help reduce the risk of developing lung cancer and strengthen the diaphragm in cholecalciferol-deficient patients with COPD.