Lithium in Inclusion Body Myositis (IBM) - Full Text View

Purpose

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

Condition
Inclusion Body Myositis


Study Type:	Observational
Study Design:	Observational Model: Cohort Time Perspective: Prospective
Official Title:	A Pilot Trial of Lithium in Inclusion Body Myositis (IBM)

Resource links provided by NLM:

Genetics Home Reference related topics: idiopathic inflammatory myopathy

MedlinePlus related topics: Myositis

Drug Information available for: Lithium carbonate Lithium citrate

Genetic and Rare Diseases Information Center resources: Inclusion Body Myositis Idiopathic Inflammatory Myopathy

U.S. FDA Resources

Further study details as provided by Phoenix Neurological Associates, LTD:

Biospecimen Retention: Samples Without DNA

Muscle Biopsies will be taken at baseline and at the end of 6 months. Tissue will be frozen then sent out for anaylsis.


Estimated Enrollment:	20
Study Start Date:	June 2008
Study Completion Date:	December 2009
Primary Completion Date:	November 2009 (Final data collection date for primary outcome measure)

Detailed Description:

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.

Eligibility


Ages Eligible for Study:	30 Years to 88 Years (Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No
Sampling Method:	Probability Sample

Study Population

Patients who fit criteria diagnosed with IBM

Criteria

Inclusion Criteria:

Age >30
Meet diagnostic criteria for definite IBM
Muscle function adequate for quantitative muscle testing
Able to give informed consent
Women of child bearing potential must have a negative pregnancy test

Exclusion Criteria:

Presence of uncontrolled diabetes, hypothyroidism, chronic infection, chronic renal insufficiency, congestive heart failure, cancer, or other chronic serious medical conditions
Significant arrhythmias or conduction defect abnormalities on ECG
Pregnant or lactating
Coexistence of other neuromuscular or neurological diseases that would interfere with assessment
Known bleeding disorder
On Warfarin
Contraindications to muscle biopsy: allergy to local anesthetic, skin infection, known bleeding disorder

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00917956

Locations


United States, Arizona
Phoenix Neurological Associates, LTD
Phoenix, Arizona, United States, 85018

Sponsors and Collaborators

Phoenix Neurological Associates, LTD

Investigators


Principal Investigator:	David D Saperstein, MD	Phoenix Neurological Associates, LTD

More Information

Additional Information:

Related Info This link exits the ClinicalTrials.gov site


Responsible Party:	David Saperstein, MD, Phoenix Neurological Associates, LTD
ClinicalTrials.gov Identifier:	NCT00917956 History of Changes
Other Study ID Numbers:	WIRB #: 20071696 Study Number 1093885
Study First Received:	June 8, 2009
Last Updated:	February 25, 2010
Health Authority:	United States: Institutional Review Board

Keywords provided by Phoenix Neurological Associates, LTD:


Lithium IBM InclusionIBM Myositis

Additional relevant MeSH terms:


Myositis Myositis, Inclusion Body Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Lithium Carbonate Antidepressive Agents	Psychotropic Drugs Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Antimanic Agents Tranquilizing Agents Central Nervous System Depressants Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 27, 2016

Lithium in Inclusion Body Myositis (IBM) (Li-IBM)