Rhabdomyolysis

Researchers are evaluating anitocabtagene autoleucel (anito-cel), a BCMA-directed CAR-T cell therapy, in adults with generalized myasthenia gravis (GMG) classified as MGFA Grade 2 to 4a. This Phase 1 open-label, multi-center study aims to assess the safety, tolerability, and preliminary efficacy of anito-cel in patients who require immunosuppressive therapy as determined by their neurologist. The study focuses on identifying dose-limiting toxicities (DLTs), the maximum tolerated dose (MTD), and selecting the recommended Phase 2 dose (RP2D) for treatment. The study includes sequential phases: screening, enrollment with leukapheresis to collect cells, pretreatment with a standard lymphodepletion chemotherapy regimen for 5 days, and a single intravenous infusion of anito-cel cells. Optional bridging therapy may be given while the CAR-T cells are prepared. Following infusion, participants will be closely monitored for safety and efficacy outcomes over time. Participants will undergo safety monitoring for at least 28 days after infusion, with safety data collected throughout the study duration of up to 24 months. Clinical effects will be evaluated using measures including the Myasthenia Gravis Activities of Daily Living (MG ADL) score, Quantitative Myasthenia Gravis (QMG) score, and Myasthenia Gravis Composite (MGC) scale. Additionally, blood tests will monitor autoantibody levels and pharmacokinetics of the therapy. This comprehensive follow-up ensures careful assessment of treatment impact and participant health.

Muscle mass loss is a common and serious problem in people with cancer, especially in those with metastatic lung cancer. This condition, called cancer cachexia, involves involuntary weight loss and affects up to 80% of such patients. Researchers are investigating the molecular causes of muscle and fat loss, hormone sensitivity, and the role of exercise in potentially restoring muscle function and improving quality of life for both patients and their caregivers. The study aims to fill important gaps in knowledge about cancer cachexia and its impact on health and survival. The study includes three parts: first, examining hormone sensitivity and physical performance in patients with lung cancer undergoing their first type of cancer treatment; second, analyzing changes in muscle, fat, and blood samples to understand disease progression; and third, testing an exercise program involving supervised single-leg kicking training for 8 weeks to see if it can improve muscle signaling and function. Control subjects matched for age, weight, gender, and activity level will also participate in the exercise intervention. Participants will undergo assessments including muscle mass and disease outcome measurements after 12 weeks, insulin sensitivity testing at 8 weeks, and surveys on quality of life. Muscle and tissue samples will be collected to study molecular changes. The study will monitor physical function, hormone sensitivity, and patient-reported outcomes to understand the effects of cancer and exercise over time. Total participation duration varies with study parts but includes multiple visits for testing and training sessions.

This research aims to compare a new method of weight training with typical weight training to see if it causes more exercise-induced muscle damage, which may help build bigger muscles. It also explores how muscle soreness affects daily activities in young adults aged 18 to 35 who have prior resistance training experience. The study is conducted by the New York Institute of Technology and involves healthy volunteers. Participants will be randomly assigned to one of two groups: fast eccentric resistance training or slow eccentric resistance training. The study includes three lab sessions, each lasting 45 minutes. Sessions involve warm-up treadmill walking, maximal voluntary contraction tests on the chest press, and sets of eccentric (lowering) bench press exercises at 75% of maximum effort. After sessions two and three, participants rate muscle soreness for three days and answer questions about how the soreness affects their activities. During participation, individuals complete demographic questionnaires, muscle soreness self-assessments, and strength testing. The primary outcomes measured are muscle soreness over three days following training and changes in maximal voluntary isometric contraction before and immediately after interventions. Follow-up is conducted via two email questionnaires after sessions two and three. The total commitment includes three lab visits plus follow-up assessments. Safety is monitored by excluding those with injuries or cardiovascular issues that would prevent pain-free training.

This observational registry studies the use of the CytoSorb device, a sorbent hemoperfusion system, in real-world critical care settings for patients with serious conditions such as septic shock, acute respiratory distress syndrome, trauma, and other life-threatening illnesses. The goal is to create a comprehensive data repository and reporting system to monitor and improve patient care quality when using this device in intensive care units. Participants will be those who have planned or actual use of the CytoSorb 300 mL device. The study collects data on the device's use without assigning specific treatments, serving as a scientific resource to evaluate outcomes like ICU and hospital mortality. This registry includes patients receiving the device in various critical care scenarios, excluding certain uses like antithrombotic removal or intraoperative cardiac surgery applications. During the study, data about patient outcomes through ICU discharge or hospital discharge will be collected and analyzed, typically over an average of 7 to 14 days. This includes monitoring ICU mortality and in-hospital mortality. The registry aims to support ongoing assessment of the device's use by gathering real-world patient information over time to help improve care practices in critical care environments.

Researchers are collecting data from adults with neuromuscular disorders who are not currently enrolled in other NIH studies. This observational study aims to build a repository of information to help generate future research hypotheses and improve understanding of various neuromuscular conditions. It also supports training programs for clinical neurophysiology fellows and keeps medical staff updated on a wide range of neuromuscular diseases. Participants will visit the NIH EMG Lab for evaluation using standard neurophysiological tests such as electromyography (EMG), autonomic nervous system testing, and neuromuscular ultrasound as needed. Additional assessments may include nerve conduction studies, sweat function tests, heart rate and blood pressure monitoring, and breathing or body position changes. These procedures are part of standard care and diagnostic evaluation. Each participant will have one or two visits lasting less than four hours each. During visits, physical exams and various assessments will be performed to evaluate nerve and muscle function. Data from participants will be stored securely with unique identifiers for future research use. Participants may be contacted for follow-up visits, and the primary goal is to create a valuable information repository over a 10-year period.

Researchers are evaluating the use of an n-of-1 trial to help patients with prior statin intolerance tolerate rosuvastatin, a cholesterol-lowering medication. The trial focuses on individuals aged 21 years and older who are eligible for statin therapy but have stopped using at least two different statins due to side effects. The study aims to better identify true statin intolerance, which could improve treatment decisions and cost-effectiveness of lipid-lowering therapies, especially for those at risk of cardiovascular disease. Participants will be randomly assigned to receive either placebo or rosuvastatin first, followed by the other treatment, in a double-blinded manner. They will take three 28-day packs of placebo and two 28-day packs of rosuvastatin during the study period. This design helps determine whether patients can tolerate rosuvastatin when unblinded after the initial blinded phase. During the 9-month study, researchers will monitor the presence or absence of musculoskeletal symptoms, statin tolerance, and factors associated with statin intolerance. Participants may continue their other lipid-lowering treatments if stable for at least one month. The study involves regular assessments to track symptoms and response, with safety and tolerance carefully evaluated throughout the trial.

Researchers are studying the use of dexamethasone, a steroid, in addition to standard care for patients aged six months to 25 years diagnosed with rhabdomyolysis, a condition involving muscle breakdown. This study aims to evaluate whether adding dexamethasone improves pain, reduces hospital stay length, and lowers kidney complications. The trial is a blinded, randomized study conducted at a single center with up to 50 participants. Participants will be randomly assigned in a 2:1 ratio to receive either dexamethasone or a placebo once daily for five days, along with standard care. The dexamethasone dose is 0.6 mg/kg per day, up to a maximum of 16 mg. Both groups will follow the same treatment duration and schedules, with no additional interventions beyond the study medication and standard care. During the study, patients and their caregivers may complete surveys assessing pain before and after treatment. Researchers will review medical records throughout and after the five-day treatment period to monitor muscle breakdown, kidney issues, and length of hospital stay. The primary outcomes include measuring these factors from five days up to one year, with pain outcomes assessed up to 14 days. The trial involves careful monitoring and no extra procedures beyond standard care and study medication.

This trial investigates the use of battlefield acupuncture for managing pain in patients admitted with exertional rhabdomyolysis. Researchers aim to compare pain levels and pain medication needs between patients who receive this acupuncture treatment and those who do not. The study is a pilot, prospective observational case series conducted at Tripler Army Medical Center without blinding of patients or providers. Patients who meet the criteria for rhabdomyolysis and choose to undergo battlefield acupuncture will receive the treatment within 12 hours of hospital admission alongside standard pain care. There is also a comparison group of patients admitted with rhabdomyolysis who decline the acupuncture treatment and receive only standard care. The study follows participants for up to 12 months to monitor pain and medication use. Participants will have their pain levels measured on a scale from 0 to 10 throughout the study period. Researchers will also track the amount of pain medication used from enrollment until the end of treatment at 12 months. Secondary outcomes include the length of hospital stay, recorded from admission to discharge over a maximum of 7 days. The study does not use randomization or blinding and involves ongoing monitoring of pain responses.

Researchers are evaluating how different types and intensities of exercise affect muscle recovery in people taking statin medications compared to healthy individuals not using statins. The study aims to understand muscle-related side effects linked to statins, especially when combined with physical activity, to help create safer exercise guidelines for statin users. This is a non-randomized, dose-escalation trial comparing statin users with matched controls based on age, sex, and body mass index. Participants will undergo a stepwise exercise program starting with aerobic cycling for 40 minutes at 60% heart rate reserve, followed by resistance training at increasing intensities of 40%, 60%, and 80% of an estimated one-repetition maximum. Resistance training includes eight machine-based exercises with repetitions decreasing as intensity rises. Each intensity phase consists of two sessions separated by a 5 to 21-day rest period, with all sessions supervised for safety and proper technique. During the study, participants will have medical screening and baseline tests including blood samples and pain assessments. Before and after each exercise session, muscle damage markers, pain thresholds, heart rate, blood lactate, and perceived exertion will be recorded. Participants will also complete daily questionnaires on muscle symptoms and recovery needs. The main measure is the change in blood creatine kinase levels 24 hours after exercise, with study duration per participant under four months.

Researchers are investigating the liver safety of statin use among inpatients in the neurology department in China. This study uses both a cross-sectional design and a retrospective cohort study to analyze statin effects from different perspectives. The goal is to provide additional evidence-based guidance for clinical use of statins in patients with neurological conditions, including cerebrovascular disease, headache, dizziness, and vertigo. The research consists of two phases. First, a cross-sectional study examines the relationship between statin use before hospital admission and the initial liver enzyme levels of patients. Next, a retrospective cohort study divides patients who were not on statins prior to admission into two groups based on whether they started statins after admission. Liver enzymes such as aspartate aminotransferase (AST) and alanine aminotransferase (ALT) are measured four weeks after admission to assess liver safety. Participants are hospitalized adults aged 18 to 85 years with specific neurological diagnoses. Researchers review medical histories and liver enzyme tests, focusing on AST and ALT levels four weeks after admission. The study excludes patients with incomplete medical records, short prior statin use, malignant tumors, or blood diseases. The total participation involves data analysis from hospital records during the defined study period from June 2020 to February 2023.