Lithium in Inclusion Body Myositis (IBM) (Li-IBM)
IBM is the most common acquired muscle disease occurring over the age of 50. The underlying cause remains unknown and there is currently no effective treatment. Pathological studies have revealed abnormal collections of proteins in the muscle cells from patients with IBM. These include proteins called phosphorylated tau (p-tau). A similar process appears to occur in Alzheimer disease, with accumulations of p-tau developing in brain cells. Lithium decreases the activity of the GSK, an enzyme that has a key role in the development of p-tau. Lithium and other GSK inhibitors have been shown to decrease the accumulation of p-tau in nerve cells in animal models of Alzheimer disease. The proposed research is a pilot study to see if lithium might be an effective treatment for IBM
Inclusion Body Myositis
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||A Pilot Trial of Lithium in Inclusion Body Myositis (IBM)|
Muscle Biopsies will be taken at baseline and at the end of 6 months. Tissue will be frozen then sent out for anaylsis.
|Study Start Date:||June 2008|
|Study Completion Date:||December 2009|
|Primary Completion Date:||November 2009 (Final data collection date for primary outcome measure)|
Trial Status Open for Enrollment
What is the Purpose of this Study? There is currently no effective treatment for IBM and its pathogenesis remains uncertain. This study is designed to test the hypothesis that treatment of patients with IBM with Lithium can improve muscle strength and reduce the markers of disease activity believed to be important in the disease pathogenesis.
Who is Eligible to Participate? Patients diagnosed with Inclusion Body Myositis above the age of 30 who have adequate muscle function for quantitative muscle testing and who have a FVC >50%. Patients who have uncontrolled diabetes, renal insufficiency, congestive heart failure, cancer, hypothyroidism, current use of immunosuppressive medication, currently on warfarin or any known bleeding disorder are excluded.
What is involved in this Study? This is an open label trial looking at the effects of Li on muscle strength. Procedures consist of a baseline muscle biopsy, blood work, and other screening procedures. After the muscle biopsy patients are started on Li at 300mg/d. Lithium doses are dependent on tolerability and target lithium levels. Monthly visits consist of EKGs, Li level labs, questionnaires, and muscle strength testing. At month 6, patients will receive another muscle biopsy. The muscle biopsies are performed to analyze p-tau levels in the muscle.
How long is the Study? It is approximately 6 months long; however patients have the option to stay on the study for an additional 6 months.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00917956
|United States, Arizona|
|Phoenix Neurological Associates, LTD|
|Phoenix, Arizona, United States, 85018|
|Principal Investigator:||David D Saperstein, MD||Phoenix Neurological Associates, LTD|