Clinical trial NCT02483845: Natalizumab in Inclusion Body Myositis (IBM)
Official title: Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Inclusion Body Myositis (IBM)
Lead sponsor: Phoenix Neurological Associates, LTD
Sponsor class: OTHER
First posted: 2015-06-29
Enrollment: 6
Sex: ALL
Minimum age (years): 21
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

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

Primary outcomes:
measure: Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies)
timeFrame: 12 months
measure: Determine if natalizumab is effective and safe in the treatment of patients with IBM (manual muscle testing)
timeFrame: 12 months
Secondary outcomes:
measure: 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)
timeFrame: 12 months
measure: 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)
timeFrame: 12 months
measure: 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)
timeFrame: 12 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Neurological Insitutute
state: Arizona
Study officials:
affiliation: Phoenix Neurological
name: Todd Leveine, MD
role: PRINCIPAL_INVESTIGATOR
References: