A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Muscular DystrophiesMuscular Dystrophy10 moreHOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.
Efficacy of Low Level LASER Therapy in Treating Temporomandibular Muscle Disorders
Temporomandibular Joint Dysfunction SyndromeThis study aimed to evaluate the sustainability of the efficacy of using low level laser therapy and CAD/CAM Michigan splint in improving the range of mandibular movements, muscle activity and reducing the pain.
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers...
Healthy VolunteerMuscular Dystrophies8 moreThis is a Phase 1 2-part, single-center, open-label study in healthy male volunteers. Part A will assess the absorption, metabolism, excretion, and pharmacokinetics of one oral dose of radiolabeled EDG-5506. Part B will assess bioavailability of EDG-5506 with a single oral dose of EDG-5506 and a single intravenous dose of radiolabeled EDG-5506.
Effect of Spinal Manipulation on Electromyography of the Masseter Muscle
BruxismMuscle DisorderResearch Problem: To know the immediate effects of high speed and low amplitude cervical manipulation on the electromyographic activity of the masseter muscles of physiotherapy students at the University of the Americas Course objective: To compare the immediate effects of high-speed, low-amplitude cervical manipulation on the electromyographic activity of the masseter muscles versus a placebo intervention in kinesiology students at the University of the Americas. Specific objectives: To describe the changes in the electromyographic activity of the masseter muscles of the high-speed cervical manipulation group in students of the physiotherapy school of the University of the Americas of the Santiago Centro campus. To describe the changes in the electromyographic activity of the masseter muscles of the placebo group in students of the physiotherapy school of the University of the Americas of the Santiago Centro campus. Methodology: Single-blind, randomized clinical trial. Expected results: Significant differences are expected between the intervention group and the control group. This is reflected in a decrease in electromyographic activity in the masseter muscles after high-speed cervical manipulation.
A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients...
FibromyalgiaMyofascial Pain Syndromes5 moreThe use of low-dose CBP dosed nightly at bedtime for FM was supported by the results of Tonix' TNX-CY-F202 Phase 2b study (also referred to as the BESTFIT Study). The TNX-CY-F202 study provided strong evidence that TNX-102 SL 2.8 mg dosed nightly results in beneficial effects upon pain, sleep and other FM symptomatology. The present trial is designed to assess the safety and efficacy of TNX-102 SL 2.8 mg tablets, taken daily at bedtime over 12 weeks to treat fibromyalgia.
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Mitochondrial MyopathyPhase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this patient population.
Pharmacokinetic Study on N-acetylneuraminic Acid
Nonaka MyopathyHereditary Inclusion Body MyopathyThe aim of this study is to investigate pharmacokinetics and safety of N-acetylneuraminic acid in patients with Distal myopathy with rimmed vacuoles (DMRV) - hereditary inclusion body myopathy (hIBM). Dosages are 800mg single and three times a day and 800mg 3 times a day for 5 consecutive days. N-acetylneuraminic acid and N-glycolylneuraminic acid in serum and urine are measured before and after oral administration of N-acetylneuraminic acid.
Qigong vs. Aerobic Exercise in the Treatment of Childhood Chronic Musculoskeletal Pain
Muscular DiseaseFibromyalgiaThis study is comparing two different exercise programs, Qi Gong (gentle stretching, and breathing) with an aerobics program (boxing and dancing) to see if children with chronic pain or fibromyalgia are able to take part in the program. We would also like to know if it is easy to take part in an exercise program. We hypothesize that children with chronic musculoskeletal pain and FM, who engage in Qigong fitness training, will be more compliant, and have an equivalent or possibly superior improvement on measures of motor function (peak power, endurance, and metabolic efficiency) compared to children given an aerobic fitness exercise program.
Effects of Statins on Pro- and Antioxidant Status : Link With Statin-Associated Myopathy.
HealthyTo evaluate the effects of a 8-week treatment with 80 mg/day simvastatin on prooxidant and antioxidant status at rest and after exercise, on mitochondrial respiration and calcium release in skeletal muscle, and on aérobic aptitude in 24 healthy male subjects.
Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
Hereditary Inclusion Body MyopathyThis study will evaluate patients with Hereditary Inclusion Body Myopathy (HIBM) and examine the effects of immune globulin (IG) treatment on muscle and muscle function. HIBM is a progressive neuromuscular disease that begins in early adulthood, primarily affecting limb muscles. It results from mutations of the gene that is responsible for producing sialic acid, a sugar normally found on the surface of certain proteins, including alpha-dystroglycan, which is involved in muscle function. Some patients with HIBM have decreased sialic acid on the alpha-dystroglycan protein, which may be the cause of their muscle weakness. IG is a protein in the blood that carries a large amount of sialic acid. This study will administer IG to patients with HIBM and determine if the sialic acid in IG is taken up by muscle cells in these patients and if it can restore some of their muscle function. Four patients with HIBM will be admitted to this study at the NIH Clinical Center for evaluation and IG treatment. The evaluation lasts about 1 month. After completing baseline studies (see below), patients receive two intravenous doses of immune globulin (on days 6 and 7), followed by measurement of muscle strength 2 days later (day 9). They receive additional IG infusions on days 13, 20, and 27. A final set of tests is performed on day 29. Patients may leave the hospital on pass when no studies are being done. A patient's initial evaluation includes: History and physical examination, neurological examination, eye examination 24-hour urine collection Blood tests on two separate days Photographs showing the extent of muscle affected Chest x-ray, electrocardiogram (EKG), and echocardiogram Two muscle biopsies, one before and one after the IG treatments. For this procedure, a small sample of muscle tissue is surgically removed for examination under the microscope. Muscle strength and endurance testing, including the following: The patient uses pulleys attached to machines that measure the strength of 24 different muscle groups The patient walks for 6 minutes and performs exercises To evaluate swallowing, the patient swallows a thick substance called barium The patient's tongue strength is measured using a specialized instrument. -Magnetic resonance imaging (MRI) of the muscles of the thigh or calf: MRI uses a magnetic field and radio waves to produce detailed pictures of organs and tissues. During the scan, the subject lies on a table in a narrow cylinder containing a magnetic field, wearing ear plugs to muffle loud noises that occur with electrical switching of the magnetic fields. He or she can speak with a staff member via an intercom system at all times during the procedure. The neurological and muscle strength and endurance evaluations are repeated on study days 9 and 29.