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

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ClinicalTrials.gov Identifier: NCT00917956
Recruitment Status : Completed
First Posted : June 11, 2009
Last Update Posted : February 26, 2010
Sponsor:
Information provided by:

Study Description
Brief Summary:
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 or disease
Inclusion Body Myositis

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.


Study Design

Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Pilot Trial of Lithium in Inclusion Body Myositis (IBM)
Study Start Date : June 2008
Primary Completion Date : November 2009
Study Completion Date : December 2009


Groups and Cohorts


Outcome Measures

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.

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   30 Years to 88 Years   (Adult, Senior)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

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
First Posted: June 11, 2009    Key Record Dates
Last Update Posted: February 26, 2010
Last Verified: February 2010

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