OHSU

Clinical Trials at the OHSU ALS and Neuromuscular Disease Center

Clinical trials are the final step in a long process of research. They allow scientists to test the value of their research to diagnose, treat and prevent diseases and disabilities. Each clinical trial is an opportunity to test a promising new invention or treatment in humans for the first time .

Clinical trials for people with ALS and neuromuscular disorders include:

IRB00006175 A Multicenter Study for the Validation of ALS Biomarkers 

The primary objective of this study is to identify factors that contribute to the pathogenesis of amyotrophic lateral sclerosis (ALS). Development of disease biomarkers and diagnostic laboratory tests would facilitate earlier treatment intervention, help monitor treatment efficacy, and ultimately, lead to the identification of targets that could be used in therapy development. The ALS Center at OHSU is one of the 35 ALS centers of the Northeast ALS (NEALS) Consortium in the US and Canada currently participating in this trial. People diagnosed with ALS or other neuromuscular diseases may be eligible to participate.

 IRB00006696 Trial of high-fat / high-calorie diet versus optimal nutrition in Amyotrophic Lateral Sclerosis 

Progressive weight loss is a common symptom of ALS and correlates with disease progression and time to death. People with ALS tend to eat fewer than the recommended daily amount of calories. This study will determine if a high-fat diet, which slows the ALS progression in animals, has the same effect in humans with ALS.  

IRB00005176 Lipoic acid for Chronic Inflammatory Demyelinating Polyneuropathy -- a randomized, double-blind, placebo controlled pilot study 

The purpose of this pilot study is to examine if alpha lipoic acid (LA) is an effective treatment for chronic inflammatory demyelinating polyneuropathy (CIDP). Lipoic acid has anti-oxidant and anti-inflammatory properties. It is naturally present in the body and often used as a dietary supplement. For more information about the study, please contact Diana Dimitrova at 503 494-7269 or .

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 .

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 .

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 .

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 .

IRB00006543 A Phase 2 Open-label Study to Evaluate the Long-term Safety of Sifalimumab in Adult subjects with Systemic Lupus Erythematosus or Myositis

This is an open-label extension study of the MEDI-545 study, and two other studies which were not performed at OHSU. Eligibility is limited to subjects who participated in one of the three previous trials of the study drug. For more information please contact the Neuromuscular Disease Research Center at 503 418-2124 or .

IRB0006457 MGAWN1 in West Nile Virus

(This study is currently closed to enrollment but may re-open in the spring of 2011)
West Nile virus is a disease spreadpredominantly after a bite from an infected mosquito but has also occurred during transfusion of blood products, organ transplants from infected donors, from pregnant mothers to a fetus and through work exposure.  About 20 percent of infected people experience West Nile fever with symptoms including fever, headache, body aches, skin rash and swollen glands.  West Nile virus can enter the brain and cause severe, life-threatening neuroinvasive disease. The purpose of this study is to determine the safety and efficacy of the drug MGAWN1 in West Nile virus and West Nile fever. For more information please contact the Neuromuscular Disease Research Center at 503 418-2124 or .

IRB00004416 IVIG in Multifocal Motor Neuropathy (MMN) – closed to 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 .

Other Clinical Trials

In addition to OHSU clinical trials, you can search for clinical trials throughout the United States and around the world at www.clinicaltrials.gov.