Clinical Trials
The following is a list of the research activities of the Neuromuscular Disease Center:
On-going clinical studies
IRB00004416 IVIG in Multifocal Motor Neuropathy (MMN)- CLOSED ENROLLMENT
MMN is a medical condition that can cause weakness in one or more of your arms and legs. The condition slowly gets worse over time and may cause you to lose muscle strength and experience muscle cramping. The condition is due to the nerve signal being blocked from your brain to the affected muscle(s). The purpose of this study is to find out if intravenous immunoglobulins are safe and effective in reducing the muscle weakness caused by MMN. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00003813 MEDI-545 in Adult Patients with Dermatomyositis or Polymyositis-
Now closed to enrollment. The purpose of this study is to determine if an experimental drug called MEDI-545 will help treat dermatomyositis and polymyositis. The study drug is one of a class of drugs that suppress your immune system. In laboratory studies, the study drug has been shown to inactivate or neutralize a protein that is thought to be related to myositis. Based on these studies, the study drug may reduce the muscle and skin symptoms in people with myositis. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00003646 Myozyme Temporary Access Program (MTAP)-
Now closed to enrollment. Pompe Disease is a metabolic condition in which your body does not produce enough of an enzyme used to help muscle function. Subjects in this study have already been receiving the enzyme replacement therapy (ERT) Myozyme (alglucosidase alfa) which is intended to replace the missing enzyme in your body. Myozyme has been approved by the Food and Drug administration for Pompe disease. The purpose of this program is to provide people with Pompe disease in the United States (US) access to Myozyme produced from a large-scale (faster) manufacturing process for a limited time until the product is commercially available again in 2009. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00002811 Etanercept in Dermatomyositis (DM)-
Now closed to enrollment. DM is a disorder in which your immune system is overactive and attacks your own body's cells and organs. In the case of DM, the immune system attacks the skin and muscles leading to a rash and weakness. The purpose of this research study is to determine whether the medication etanercept is safe and tolerated when it is taken by people with DM. Right now, this drug is not approved for use for DM in the United States because we do not know enough about it. It is currently used in the treatment of diseases like Rheumatoid Arthritis. The study drug works by blocking a chemical in the body called tumor necrosis factor –a (TNF-a). TNF-a is involved in inflammation and may play a role in the symptoms of DM. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00001965 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome-
Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive weakness that affects the upper parts of the legs and arms but does not usually involve the respiratory muscles and the muscles of the face. This treatment trial is considered "compassionate use". Compassionate use is mostly obtained for patients with advanced diseases who have tried all of the available treatment options and whose disease has not responded or for patients with diseases that have no approved treatment options and no clinical trials meet the patients needs. 3,4-Diaminopyridine (DAP), although not yet approved for use by the U.S. Food and Drug Administration, has improved strength in some subjects with these disorders. Since there are no other accepted treatments for LEMS and since DAP has relatively few side effects, the FDA allows for its use in controlled circumstances like this study. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00001825 OHSU Neuromuscular Diseases Registry
-Doctors often study problems by looking at groups of people with the same condition. By combining the information of these people, they see patterns in the disease. One way to do this is by following people with similar conditions in a registry. If you agree, we would like to place your medical records into a Neuromuscular Disease Registry. Your information will be used by researchers in future studies. Your participation will help increase our knowledge about problems with muscles and nerves. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00001292 Pompe Disease Registry
- The purpose of the Pompe Registry is to gather information on people with Pompe disease to help the medical community better understand Pompe disease and its medical management. The Pompe Registry is an ongoing, observational database that tracks the natural history and outcomes of patients with Pompe disease. Information from normal standard of care for patients with Pompe disease, such as blood and urine laboratory results, muscle function assessments, respiratory evaluations, heart evaluations, quality of life assessments and information regarding enzyme replacement therapy (ERT), will comprise the Pompe Registry database. Information about pregnancy and lactation will be collected from all female subjects in the registry and information about ventilator status will be collected at entry to the registry and as changes occur. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
IRB00004753 THAOS Registry
A Global, Multi-Center Survey of Patients with Documented Transthyretin (TTR) Mutations or Wild-Type TTR Amyloidosis.
Amyloid is a type of protein that can aggregate in different areas of the body leading to a multitude of different symptoms. The purpose of the THAOS Registry is to gather information on people with TTR Mutations or Amyloidosis to help the medical community better understand the condition and improve medical management. The THAOS Registry is an ongoing, observational database that tracks the natural history and outcomes of patients with TTR mutations or Amyloidosis. Information from normal standard of care for patients such as blood and urine laboratory results, muscle function assessments, neurological assessments, respiratory evaluations, heart evaluations, and quality of life assessments will be recorded in the registry. For more information, please contact the Neuromuscular Disease Research Center at 503 418-2124 or neuromus@ohsu.edu.
