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IBM-NAT: Natalizumab in Inclusion Body Myositis (IBM)

Sponsor
Phoenix Neurological Associates, LTD (Other)
Overall Status
Unknown status
CT.gov ID
NCT02483845
Collaborator
(none)
6
Enrollment
1
Location
1
Arm
54
Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 1

Detailed Description

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 Design

Study Type:
Interventional
Actual Enrollment :
6 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis
Study Start Date :
May 1, 2013
Anticipated Primary Completion Date :
Nov 1, 2017
Anticipated Study Completion Date :
Nov 1, 2017

Arms and Interventions

Arm Intervention/Treatment
Experimental: Natalizumab

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

Drug: Natalizumab
Other Names:
  • Tysabri

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies) [12 months]

      muscle biopsies to determine inflammation

    2. Determine if natalizumab is effective and safe in the treatment of patients with IBM (manual muscle testing) [12 months]

      MMT - manual muscle testing to determine strength

    Secondary Outcome Measures

    1. 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) [12 months]

      Safety labs - to determine blood levels

    2. 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) [12 months]

      Pain scores on a visual analog scale

    3. 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) [12 months]

      IM-FRS a functional rating score scale

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    21 Years to 85 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    1. Definite diagnosis of sporadic IBM through previous muscle biopsies

    2. Age 21-85

    3. FVC> 50%

    4. Muscle function adequate for quantitative muscle testing

    5. JC virus negative at screening

    Exclusion Criteria:

    1. Previous therapy with natalizumab.

    2. Treatment with other immunosuppressive agents within the last 12 months

    3. Quadriceps strength less than or equal to 2/5 at baseline

    4. Known malignancy

    5. Pregnancy or breastfeeding

    6. History of abnormal laboratory results indicative of any significant medical disease that would preclude the use of natalizumab

    7. Any clinically significant infectious illness in the 30 days before enrollment

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Phoenix Neurological Insitutute Phoenix Arizona United States 85018

    Sponsors and Collaborators

    • Phoenix Neurological Associates, LTD

    Investigators

    • Principal Investigator: Todd Leveine, MD, Phoenix Neurological

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Phoenix Neurological Associates, LTD
    ClinicalTrials.gov Identifier:
    NCT02483845
    Other Study ID Numbers:
    • IBM-2013
    • 117571
    First Posted:
    Jun 29, 2015
    Last Update Posted:
    Feb 9, 2017
    Last Verified:
    Feb 1, 2017
    Additional relevant MeSH terms:
    Myositis
    Myositis, Inclusion Body
    Natalizumab

    Study Results

    No Results Posted as of Feb 9, 2017