Clinical Trial: Natalizumab in Inclusion Body Myositis (IBM)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis

Brief Summary: Muscle biopsies of patients with Inclusion Body Myositis (IBM) have demonstrated a T-cell predominant inflammatory infiltrate, therefore, new agents targeting T -cell mediated cell death may be a novel treatment for IBM. Such an agent capable of preventing T-cell movement out of the vasculature, such as natalizumab, may be beneficial in IBM patients. Six patients will be recruited to participate in this phase I trial.

Detailed Summary:

This is a phase I open label, non-placebo controlled trial evaluating the safety and efficacy of natalizumab in patients with IBM. Pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells will be measured. The investigators will also assess quality of life, the inclusion body myositis functional rating score (IBM-FRS), and patient and physician global impression of change. Manual muscle testing and quantitative dynamometry will also be evaluated to see if patient's strength improves.

Patients who are eligible to participate and have signed a consent form will have a muscle biopsy performed at baseline and at the end of the study. Physical and neurological exams, as well as IBM-FRS, safety labs will be conducted and collected at monthly visits. Patients will start natalizumab therapy at 300mg intravenously every 4 weeks for 24 weeks. All study related procedures will be conducted at Phoenix Neurological Associates, as well as all infusions and muscle biopsies


Sponsor: Phoenix Neurological Associates, LTD

Current Primary Outcome:

  • Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies) [ Time Frame: 12 months ]
    muscle biopsies to determine inflammation
  • Determine if natalizumab is effective and safe in the treatment of patients with IBM (manual muscle testing) [ Time Frame: 12 months ]
    MMT - manual muscle testing to determine strength


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (blood levels) [ Time Frame: 12 months ]
    Safety labs - to determine blood levels
  • Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (pain scores) [ Time Frame: 12 months ]
    Pain scores on a visual analog scale
  • Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (functional rating score scale) [ Time Frame: 12 months ]
    IM-FRS a functional rating score scale


Original Secondary Outcome: Same as current

Information By: Phoenix Neurological Associates, LTD

Dates:
Date Received: May 31, 2013
Date Started: May 2013
Date Completion: November 2017
Last Updated: February 8, 2017
Last Verified: February 2017