Current Research

POSTED MAY 2012

This is to inform you that the Neuromuscular Section of the Department of Neurology at the University of Kansas Medical Center is conducting a study, “Efficacy of Prednisone in the treatment of Ocular Myasthenia” (EPITOME Study). The study was developed at the University of Miami, and there are only 5 centers in United States participating in this trial. These include the University of Kansas Medical Center, University of Miami (Miami, FL), University of Rochester (Rochester, NY), Duke University (Durham, NC) and University of Virginia (Charlottesville, VA). Information on the trial is on clinicaltrials.gov identifier

# NCT00995722, the weblink being http://clinicaltrials.gov/ct2/show/NCT00995722.

 As you know, Myasthenia Gravis (MG) is a rare disorder and eye muscle weakness (droopy lid or double vision) is one of most common symptoms. The purpose of this controlled study is to determine if Prednisone is an effective therapy for newly diagnosed Ocular MG patients, in addition to Pyridostigmine, also known as Mestinon. The hypothesis is that adding Prednisone therapy will improve the Ocular MG manifestations and prevent the development or limit the severity of generalized MG. Our goal is to find an effective therapy for Ocular MG and possibly prevent the development of generalized MG.

 If you have Ocular MG patients and are interested in participating in this study, please contact our study coordinator Yodit Teklu at 913.588.0678 or yteklu@kumc.edu. We will inform you and talk to you about the study and the screening process to see if you are a potential study candidate.

 

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This is to inform you that the neuromuscular section of the Department of Neurology at the University of Kansas Medical Center is conducting a study, “Phase II Trial of Methotrexate in Myasthenia Gravis”. The study was developed at KUMC, and there are only 8 other centers in United States participating in this trial. These include the University of Texas Southwestern Medical Center (Dallas), University of California-Irvine, Ohio State University, University of Texas Science Center in San Antonio, University of Virginia, Emory University (Atlanta), University of California in San Francisco in Fresno. For potential subjects in your area we would be the closest site. Information of the trial is on clinicaltrials.gov identifier # NCT00814138

 The purpose of this study is to determine if Methotrexate 20 mg weekly oral dose is a safe and effective therapy for Myasthenia Gravis (MG) patients who are Prednisone dependent. This is a randomized, double-blind, placebo controlled study. MG patients who will enter this trial will have symptoms and signs of the disease while on Prednisone therapy. The hypothesis is that adding Methotrexate therapy in these patients will improve the MG manifestations so that Prednisone dose can be reduced and clinical measures of MG severity will improve.

 As you know, MG is a rare disorder. Clinical researchers in MG were recently disappointed because two recent large clinical trials of Mycophenolate Mofetil (Cellcept) in MG were negative. Intravenous immunoglobulin (IVIg) has recently been shown to be effective in MG as a short term therapy, but it is extremely expensive, at times unavailable, and its benefit after eight weeks is unknown. The long-term potential side effects of Prednisone can be significant. Our goal is to find an effective, available and reasonably inexpensive oral agent with a tolerable side effect profile that can decrease dependency on Prednisone. We believe Methotrexate may be able to satisfy this goal. Methotrexate has shown to be beneficial in treatment of MG symptoms as seen in a few retrospective case studies, including our study presented at AAN in 2009.

 If you have any MG patients who are interested in participating in this study, please contact our study coordinator Laura Herbelin at 913.588.5095 or lherbelin@kumc.edu. We will talk to the patient about the study and the screening process to see if he or she is a potential study candidate. You could also talk to Dr. Pasnoor about the study: call 913.588.6094 or email at rbarohn@kumc.edu or mpasnoor@kumc.edu.