Muscle tissue disorder

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the dru…

This Phase 1 trial tests the safety and tolerability of a new drug called AZD5492 in 72 adults with lupus, myositis, or rheumatoid arthritis. Participants receive one or two doses under the skin and are monitored for side effects and how the drug moves through the body. The study…

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years …

This early-phase trial is testing a new biologic drug called XmAb657 in 60 people, including healthy volunteers and those with autoimmune diseases like scleroderma, Sjögren's disease, and idiopathic inflammatory myopathies. The main goal is to check safety and how the body handle…

This study tests whether two dietary supplements, HMB and 2-HOBA, can improve muscle mass, physical function, and overall health in adults aged 65 and older. Over 90 days, 120 participants will take one or both supplements or a placebo. Researchers will measure changes in muscle,…

This study is testing the long-term safety of an experimental drug called PGN-EDODM1 in 48 adults with myotonic dystrophy type 1 (DM1) who have already taken the drug in a previous study. Participants will receive the drug by IV infusion and be monitored for side effects. The goa…

This early-phase study tests a new treatment called anitocabtagene autoleucel (anito-cel) for people with generalized myasthenia gravis, a condition that causes muscle weakness. Anito-cel is a type of CAR T-cell therapy that targets BCMA, a protein on certain immune cells. The tr…

Researchers at Johns Hopkins University are studying whether neuromuscular ultrasound and MRI can serve as reliable markers of swallowing muscle problems in people with inclusion body myositis (IBM). The study compares imaging and clinical findings in 30 participants, including p…

This study is testing a handheld device called mScan that uses a tiny, painless electrical current to measure muscle health. Researchers want to see if it can give similar results to an MRI, but faster and more conveniently. The study involves 150 adults with and without muscle d…

This study tests whether a supplement called geranylgeraniol (GG), derived from annatto seeds, can reduce muscle pain and weakness caused by statin medications. Researchers will give 95 adults either a low or high dose of GG or a placebo for three months. The goal is to see if GG…

This Phase 2 trial tests whether KL1333 can reduce fatigue and improve leg strength in adults with primary mitochondrial disease, a genetic condition that affects energy production. About 180 participants will receive either KL1333 or a placebo twice daily for 48 weeks. The study…

This study tests whether online or group physiotherapy can be as effective as individual in-person sessions for treating jaw muscle pain (TMD). About 90 adults with jaw muscle pain will be randomly assigned to one of three treatment formats. The goal is to find more accessible wa…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study tracks how quickly the diaphragm weakens in 15 ICU patients on mechanical ventilation. Researchers use advanced muscle tests and brain stimulation to measure changes in diaphragm function and control. The goal is to better understand ventilator-induced diaphragm dysfun…

This study gathers medical information from 200 adults with possible nerve, muscle, or autonomic nervous system disorders who are not already in other NIH studies. Participants undergo standard tests like nerve conduction studies, EMG, and physical exams. The data is stored anony…

This study follows 150 people aged 7 and older with RYR1-related muscle disorders for 3 to 5 years. Researchers will collect medical history, perform physical exams, and use questionnaires, activity trackers, and tests of heart, lung, and muscle function. The goal is to better un…

This study looks at how energy production inside cells (mitochondria) works in people with cystinosis, a rare disease that causes cystine buildup and early aging. Researchers will measure cell energy activity in 25 patients and compare it to healthy people. The goal is to underst…

This study follows children and adults worldwide who have a rare genetic muscle disease called TNNT1 myopathy. Researchers aim to document how the disease progresses over time, including survival and motor milestones. The goal is to identify reliable measures that can be used in …

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

This study follows 100 people in Spain with nemaline myopathy, a rare muscle disease, to see how their symptoms change over time. Researchers will use ultrasound, movement tests, and breathing checks to map the disease's natural course. The goal is to build a detailed patient dat…

This study investigates whether serious muscle weakness, known to affect ICU patients, also occurs in people hospitalized outside the ICU. Researchers will follow about 600 adults in Italian hospitals, half with organ failure and half without, to see how often weakness develops a…

This pilot study is looking at why some patients in the intensive care unit (ICU) develop severe muscle weakness after surgery. Researchers will take blood and small muscle samples from 20 high-risk patients to look for chemical changes that might cause or signal this weakness. T…

This study aims to find better ways to measure fatigue and walking problems in people with neuromuscular diseases like muscular dystrophy and spinal muscular atrophy. Researchers will use a wearable sensor to track physical activity for one week in daily life and during a walking…

This study follows 100 adults who needed ECMO support for severe heart or lung failure. Researchers will track muscle size, strength, and quality of life over time to understand why some patients develop lasting weakness. No new treatments are being tested; the goal is to gather …