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

This study has been completed.
Sponsor:
Information provided by:
Phoenix Neurological Associates, LTD
ClinicalTrials.gov Identifier:
NCT00917956
First received: June 8, 2009
Last updated: February 25, 2010
Last verified: February 2010

June 8, 2009
February 25, 2010
June 2008
November 2009   (final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00917956 on ClinicalTrials.gov Archive Site
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Lithium in Inclusion Body Myositis (IBM)
A Pilot Trial of 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

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples Without DNA
Description:

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

Probability Sample

Patients who fit criteria diagnosed with IBM

Inclusion Body Myositis
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
December 2009
November 2009   (final data collection date for primary outcome measure)

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
Both
30 Years to 88 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00917956
WIRB #: 20071696, Study Number 1093885
Yes
David Saperstein, MD, Phoenix Neurological Associates, LTD
Phoenix Neurological Associates, LTD
Not Provided
Principal Investigator: David D Saperstein, MD Phoenix Neurological Associates, LTD
Phoenix Neurological Associates, LTD
February 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP