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

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

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.

Study Overview

• Status: Unknown
• Conditions: Inclusion Body Myositis (IBM)
• Intervention / Treatment: Drug: Natalizumab

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 Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85018
      • Phoenix Neurological Insitutute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Natalizumab; Natalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks	Drug: Natalizumab; Other Names: Tysabri

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	Safety labs - to determine blood levels	12 months
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)	Pain scores on a visual analog scale	12 months
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)	IM-FRS a functional rating score scale	12 months

Collaborators and Investigators

• Sponsor: Phoenix Neurological Associates, LTD
• Investigators: Principal Investigator: Todd Leveine, MD, Phoenix Neurological

Study record dates

Study Major Dates

• Study Start: May 1, 2013
• Primary Completion (Anticipated): November 1, 2017
• Study Completion (Anticipated): November 1, 2017

Study Registration Dates

• First Submitted: May 31, 2013
• First Submitted That Met QC Criteria: June 26, 2015
• First Posted (Estimate): June 29, 2015

Study Record Updates

• Last Update Posted (Estimate): February 9, 2017
• Last Update Submitted That Met QC Criteria: February 8, 2017
• Last Verified: February 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Myositis; Myositis, Inclusion Body; Physiological Effects of Drugs; Immunologic Factors; Natalizumab
• Other Study ID Numbers: IBM-2013; 117571 (Other Identifier: IND approval)