Trial | NCT02483845  Report issue

Title

Natalizumab in Inclusion Body Myositis (IBM)
Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Unknown status

  • Study Participants

    6
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.
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
Study Started
May 31
2013
Primary Completion
Nov 30
2017
Anticipated
Study Completion
Nov 30
2017
Anticipated
Last Update
Feb 09
2017
Estimate

Drug Natalizumab

  • Other names: Tysabri

Natalizumab Experimental

Natalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks

Criteria 

Inclusion Criteria:

Definite diagnosis of sporadic IBM through previous muscle biopsies
Age 21-85
FVC> 50%
Muscle function adequate for quantitative muscle testing
JC virus negative at screening

Exclusion Criteria:

Previous therapy with natalizumab.
Treatment with other immunosuppressive agents within the last 12 months
Quadriceps strength less than or equal to 2/5 at baseline
Known malignancy
Pregnancy or breastfeeding
History of abnormal laboratory results indicative of any significant medical disease that would preclude the use of natalizumab
Any clinically significant infectious illness in the 30 days before enrollment
No Results Posted