OHSU

Clinical Trials

On-going clinical studies

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 503 494-7269 or dimitrov@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 503 494-7269 or dimitrov@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 503 494-7269 or dimitrov@ohsu.edu.



IRB00010000  Rasagiline for ALS
The purpose of this study is to learn if rasagiline is safe in a larger number of ALS patients, and if the drug has an effect on mitochondria (energy-producing parts in cells) that could potentially slow down the disease. Study participants will receive the study drug or placebo for 12 months. Study procedures include physical exam, pulmonary function tests, vital signs, blood and urine sample collection, and questionnaires. The study drug is approved for the treatment of Parkinson's disease. The study drug is not approved by the U.S. Food and Drug Administration (FDA) for treatment of ALS.

For more information, please contact the Neuromuscular Disease Research Center at 503 494-7269 or dimitrov@ohsu.edu.