Natalizumab in Inclusion Body Myositis (IBM)

NCT ID: NCT02483845

Last Updated: 2017-02-09

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-05-31
• Study Completion Date: 2017-11-30

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.

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

Conditions

Inclusion Body Myositis (IBM)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Natalizumab

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

Group Type: EXPERIMENTAL

Natalizumab

Intervention Type: DRUG

Interventions

Natalizumab

Intervention Type: DRUG

Other Intervention Names

Tysabri

Eligibility 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

• Minimum Eligible Age: 21 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Phoenix Neurological Associates, LTD

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Todd Leveine, MD

Role: PRINCIPAL_INVESTIGATOR

Phoenix Neurological

Locations

Phoenix Neurological Insitutute

Phoenix, Arizona, United States

Countries

United States

Other Identifiers

117571

Identifier Type: OTHER

Identifier Source: secondary_id

IBM-2013

Identifier Type: -

Identifier Source: org_study_id