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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02483845
Recruitment Status : Unknown
Verified February 2017 by Phoenix Neurological Associates, LTD.
Recruitment status was:  Active, not recruiting
First Posted : June 29, 2015
Last Update Posted : February 9, 2017
Sponsor:
Information provided by (Responsible Party):
Phoenix Neurological Associates, LTD

Study Description
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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
Inclusion Body Myositis (IBM) Drug: Natalizumab 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
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Study Type : Interventional  (Clinical Trial)
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 2013
Estimated Primary Completion Date : November 2017
Estimated Study Completion Date : November 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Myositis
Drug Information available for: Natalizumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Natalizumab
Natalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks
 Drug: Natalizumab
Other Name: Tysabri


Outcome Measures
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Primary Outcome Measures :
  1. 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

  2. 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


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) [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 12 months ]
    IM-FRS a functional rating score scale


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   21 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02483845


Locations
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United States, Arizona
Phoenix Neurological Insitutute
Phoenix, Arizona, United States, 85018
Sponsors and Collaborators
Phoenix Neurological Associates, LTD
Investigators
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Principal Investigator: Todd Leveine, MD Phoenix Neurological
More Information
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Responsible Party: Phoenix Neurological Associates, LTD
ClinicalTrials.gov Identifier: NCT02483845    
Other Study ID Numbers: IBM-2013
117571 ( Other Identifier: IND approval )
First Posted: June 29, 2015    Key Record Dates
Last Update Posted: February 9, 2017
Last Verified: February 2017
Additional relevant MeSH terms:
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Myositis
Myositis, Inclusion Body
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
 Nervous System Diseases
Natalizumab
Immunologic Factors
Physiological Effects of Drugs