Clinical Trial: Study of Arimoclomol in Inclusion Body Myositis (IBM)

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)

Brief Summary: Funding Source - FDA OOPD. The purpose of this study is to evaluate the safety and efficacy of the study drug, Arimoclomol in IBM patients.

Detailed Summary:
Sponsor: University of Kansas Medical Center

Current Primary Outcome: Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 12 ]

Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.


Original Primary Outcome: Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 20 ]

Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.


Current Secondary Outcome:

  • Manual Muscle Testing (MMT) [ Time Frame: Change from Baseline to Month 20 ]
  • Maximum voluntary isometric contraction (MVICT) of quadriceps [ Time Frame: Change from Baseline to Month 20 ]
  • Grip and pinch test [ Time Frame: Change from Baseline to Month 20 ]
  • modified timed up and go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
  • 6 minute walk test with 2 minute distance captured [ Time Frame: Change from Baseline to Month 20 ]
  • Health Assessment Questionnaire (HAQ-DI) [ Time Frame: Change from Baseline to Month 20 ]
  • SF-36 [ Time Frame: Change from Baseline to Month 20 ]
    Measured using the Short form health survey with 36 items (SF-36).
  • Count of adverse events [ Time Frame: Change from Baseline to Month 20 ]
  • Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 20 ]
    Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.


Original Secondary Outcome:

  • Manual Muscle Testing (MMT) [ Time Frame: Change from Baseline to Month 20 ]
  • Maximum voluntary isometric contraction (MVICT) of quadriceps [ Time Frame: Change from Baseline to Month 20 ]
  • Grip and pinch test [ Time Frame: Change from Baseline to Month 20 ]
  • modified timed up and go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
  • 6 minute walk test with 2 minute distance captured [ Time Frame: Change from Baseline to Month 20 ]
  • General physical function measure by measure of the SF36 [ Time Frame: Change from Baseline to Month 20 ]
  • Health Assessment Questionnaire (HAQ-DI) [ Time Frame: Change from Baseline to Month 20 ]
  • Health-Related Quality of Life (HRQoL) [ Time Frame: Change from Baseline to Month 20 ]
    Measured using the Short form health survey with 36 items (SF-36).
  • Count of adverse events [ Time Frame: Change from Baseline to Month 20 ]
  • Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 12 ]
    Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.


Information By: University of Kansas Medical Center

Dates:
Date Received: April 25, 2016
Date Started: May 2017
Date Completion: December 2021
Last Updated: April 24, 2017
Last Verified: April 2017