Clinical Trial: Arimoclomol in Sporadic Inclusion Body Myositis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis

Brief Summary: Inclusion body myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over 50 years of age. This study will assess the safety and tolerability of Arimoclomol in IBM as compared to placebo over 4 months of treatment.

Detailed Summary: IBM is a chronic disorder in which muscles become inflamed (swollen) and cause muscle weakening. The cause is unknown. There is new evidence to suggest that the pathology in IBM results from cellular changes induced by a variety of stressful events and diseases. In response to these stressful events the body's normal response is to increase the levels of Heat Shock Proteins (HSP) to help counteract and stop these cellular changes. In people with IBM this increase does not appear sufficient enough to reverse these toxic cellular changes. Arimoclomol causes the body to make more of this HSP protein. By increasing HSP levels in IBM patients we hope to reverse the toxic cellular changes that might be responsible for the pathology of IBM.
Sponsor: Richard Barohn, MD

Current Primary Outcome: Count of Adverse Events Reported [ Time Frame: Month 12 ]

Measure reflects the total number of adverse events reported during course of the study.


Original Primary Outcome: Adverse event reporting [ Time Frame: Every 2 weeks for 4 months ]

Current Secondary Outcome:

  • Heat Shock Protein 70 (HSP70) Levels in the Tissue [ Time Frame: Change from Baseline to Month 4 ]
    Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.
  • Muscle Strength Testing [ Time Frame: Change from Baseline to Month 4 ]
    Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.
  • Muscle Strength Testing [ Time Frame: Change from Baseline to Month 8 ]
    Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.
  • Muscle Strength Testing [ Time Frame: Change from Baseline to Month 12 ]
    Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.
  • Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score [ Time Frame: Change from Baseline to Month 4 ]
    Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
  • Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score [ Time Frame: Change from Baseline to Month 8 ]
    Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
  • Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score [ Time Frame: Change from Baseline to Month 12 ]
    Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
  • Maximum Isometric Voluntary Contraction Testing (MVICT) Score [ Time Frame: Change from Baseline to Month 4 ]
    Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.
  • Maximum Isometric Voluntary Contraction Testing (MVICT) Score [ Time Frame: Change from Baseline to Month 8 ]
    Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.
  • Maximum Isometric Voluntary Contraction Testing (MVICT) Score [ Time Frame: Change from Baseline to Month 12 ]
    Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.


Original Secondary Outcome:

  • Muscle Strength Testing [ Time Frame: Monthly for 4 months ]
  • IBM functional rating scale [ Time Frame: Monthly for 4 months ]
  • Muscle biopsy [ Time Frame: pre and post treatment ]


Information By: University of Kansas Medical Center

Dates:
Date Received: October 8, 2008
Date Started: September 2008
Date Completion:
Last Updated: November 22, 2016
Last Verified: November 2016