Natalizumab in Inclusion Body Myositis (IBM) (IBM-NAT)
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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
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Condition or disease | Intervention/treatment | Phase |
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Inclusion Body Myositis (IBM) | Drug: Natalizumab | Phase 1 |
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 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 |

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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 21 Years to 85 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

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
United States, Arizona | |
Phoenix Neurological Insitutute | |
Phoenix, Arizona, United States, 85018 |
Principal Investigator: | Todd Leveine, MD | Phoenix Neurological |
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 |
Myositis Myositis, Inclusion Body Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases |
Nervous System Diseases Natalizumab Immunologic Factors Physiological Effects of Drugs |