Lithium in Inclusion Body Myositis (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

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Key information

Conditions

Inclusion Body Myositis Muscular Diseases Musculoskeletal Diseases Myositis Myositis, Inclusion Body Nervous System Diseases Neuromuscular Diseases

Age range

30 year–88 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Phoenix Neurological Associates, LTD

Phoenix, Arizona, 85018, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Sponsors and collaborators

Lead sponsor

Phoenix Neurological Associates, LTD

Other

Registry information

Official study title

A Pilot Trial of Lithium in Inclusion Body Myositis (IBM)

Acronym: Li-IBM

Important dates

Study start: 2008
Primary completion: 2009
Study completion: 2009
First posted: Jun 11, 2009
Registry last updated: Feb 26, 2010

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed