For more information about this study, please contact:
Sharon L. Halton, MSW, LCSW
Senior Research Coordinator
Luis F. Lay, Jr., MD
Senior Research Coordinator
To be involved in the neuromuscular drug trials, you must be evaluated and followed at the Methodist Neurological Institute Neuromuscular Clinical Center.
The primary objective of this study is to determine the safety and efficacy of Belimumab to treat MG. Belimumab is not approved by the Food and Drug Administration (FDA) for the treatment of MG. However, it has been studied and approved by the FDA for treating other autoimmune disorders including lupus. You will be able to receive standard treatment for your MG during the study. Your study doctor will manage these treatments. This study will last for 6 months.
The primary objective of this study is to determine the safety and tolerability of oral administration of 0.5mg fingolimod daily vs. matched oral placebo administered daily. The primary outcome will be safety and tolerability defined as the ability of subjects to complete the entire 4-week study. The secondary objective of the study is to quantify the effect of the treatment on circulating lymphocyte populations in the blood of patients with ALS. A lymphocyte is a small white blood cell that plays a large role in defending the body and is of fundamental importance in the immune system. Status: Active
The purpose of this study is to determine efficacy of dexamethasone on disease progression in ALS. Neuroinflammation is a prominent pathologic feature of sporadic ALS and is characterized by activated microglia and infiltrating T lymphocytes at sites of spinal cord motor neuron injury. Microglia are cells that make up the innate immune system of the central nervous system and are key cellular mediators of neuroinflammatory processes. T lymphocytes belong to a group of white blood cells that contribute to immune defenses. Glucocorticosteroids have numerous clinical treatment applications based upon its diverse and broad effects upon a variety of physiologic and pathologic states and conditions, from edema, oxidative stress, apoptosis, immune inflammation, stress responses, healing, and metabolism. Glucocorticoids (GCs) exert their potential inflammatory and immunosuppressive effects through an intricate combination of mechanisms. Status: Active
The primary objective 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 aim is to develop a laboratory test that can be used by primary care physicians, neurologists and other clinicians to provide guidance with regard to “something to worry about” (possible ALS). It would provide benefit in generating appropriate referrals to neurological specialists early in disease progression.
The test will identify people with ALS and distinguish ALS from disorders that present with similar symptoms (e.g. carpal tunnel, radiculopathies, myopathies). A test with high sensitivity for ALS can be very useful for specialty neurologist as well for early and accurate detection of ALS. Blood (plasma and DNA) with/without CSF (cerebrospinal fluid) samples will be collected from age and gender matched healthy individuals, disease mimics, and ALS patients. Status: Active
Inhibiting expression of the SOD1 gene prevents animals from developing the disease. Increasing or decreasing the number of mutated genes proportionately speeds or slows the progression of the disease. Therefore, reducing SOD1 levels in patients with SOD1 associated Familial ALS (FALS) may be a promising therapeutic approach. Through an extensive in vitro screening program for medications having the ability to reduce SOD1 levels, several molecules that reduce SOD1 protein levels are known. One of the most potent molecules is pyrimethamine, an FDA approved medication used for the treatment of malaria and toxoplasmosis. Pyrimethamine dramatically reduces SOD1 levels in mice and our preliminary studies show similar findings in humans. Our study's primary objective is to determine if familial ALS patients taking pyrimethamine will show a decline in SOD1 levels in the CSF by 15% or more. We will also determine if SOD1 and pyrimethamine are present in the blood and if the SOD-1 levels decline over the course of the study. We will also evaluate the safety and tolerability of pyrimethamine in patients with FALS. Secondary objectives will be to determine dose optimization for maximal SOD1 level reduction. We will also assess the feasibility of proceeding to phase II/III studies using pyrimethamine. Status: Active
The purpose of this study is to evaluate the effectiveness of two doses of subcutaneous IgPro20 (with a portable pump) compared with in the treatment of CIDP and also to look at the safety and tolerability of these doses. IVIG is probably the first-line treatment for CIDP, but is extremely time consuming requiring hospital or home health IVIG infusion. The convenience of portable pumps have been used successfully in other disease processes. Status: Active
This study will look at cells in the blood called T regulatory cells to see if they reflect disease severity or progression and whether improvement of disease during standard immune therapy is associated with increases and/or normalization of these cells in patients with CIDP. The impact of this study may improve how patients are diagnosed and treated. This study will also serve to provide a basis for further study of disease initiation, progression, and even response to therapy. Status: Active
The purpose of this study is to find out if the drug FTY720/fingolimod is safe and has beneficial effects in people who have chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Fingolimod is an oral (capsule), once-daily study drug that offers a new approach to the treatment of multiple sclerosis (MS) that may also apply to CIDP. Fingolimod acts on certain types of white blood cells (lymphocytes) responsible for immune reactions. It makes a proportion of these cells move away from areas of inflammation and redirects them towards lymph nodes and other places in the body where they rest. These cells are believed to play an important role in the inflammation process associated with MS and CIDP. Status: Active