Sporadic Inclusion Body Myositis Natural History Study

Study Overview
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Brief Summary
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This is a prospective natural history study on patients with clinically defined sIBM. Participants will be assessed every 6 months over two years (five visits total). We will include 150 participants, enrolled across 13 sites, with sporadic IBM, diagnosed according to established criteria.

Detailed Description
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This is a prospective natural history study on patients with clinically defined sIBM. Participants will be assessed every 6 months over two years (five visits total). We will include 150 participants with sporadic IBM, diagnosed according to established criteria. Participants will require an routine serum sample via blood draw to evaluate for NT5c1A antibody status. This testing will be performed at Washington University School of Medicine in the Neuromuscular Laboratory. Investigators will be blinded to antibody status. A subset of participants (40) will undergo a muscle biopsy at the Baseline visit. Aim 1. To determine for the first time whether NT5c1A antibodies mediate disease progression over a two-year interval in patients with sIBM. We will perform a prospective, non-interventional, observational study on patients with sIBM with follow up and evaluations every 6 months over a two-year time frame. Primary analyses are: 1) the rates of disease progression and severity as measured by rates of decline in IBM Functional Rating Scale (IBMFRS) score and Timed Get Up and Go (TUG); 2) the presence or absence of serum antibodies to NT5c1A; and 3) the presence and frequency of variant T-cells in the serum and skeletal muscle. Aim 2. To perform a detailed morphological, histochemical and immunohistochemical analysis of fresh muscle biopsy specimens obtained from a subset of patients with sIBM. Aim 3. To characterize the distribution of immunosenescent lymphocytes in circulating blood from patients with sIBM. Aim 4. To quantify the decline in the respiratory function of sIBM patients.

Official Title
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Influence of NT5c1A Antibodies on Disease Progression, Clinical Phenotype and Blood and Muscle Biomarkers in Sporadic Inclusion Body Myositis - A Prospective Evaluation (INSPIRE-IBM Study)

Conditions
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Sporadic Inclusion Body Myositis, Inclusion Body Myositis

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Ages 40 years and older Fulfills ENMC 2011 criteria of clinically definite or probable sporadic Inclusion body myositis (sIBM) Disease onset is within the past 10 years of the time of Baseline visit Able to participate and comply with study related procedures Able to provide written consent Exclusion Criteria: Current or very recent use (within last 6 months of the Baseline visit) of immunomodulation or immunosuppression therapy. Current or very recent use (within last 90 days of the Baseline visit) of an investigational medication or therapy. Co-existing significant medical or surgical conditions that, in the opinion of the investigator, will influence study participation or alter natural history.

Ages Eligible for Study
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Minimum Age: 40 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Inclusion Body Myositis Functional Rating Scale (IBMFRS) | The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person. | Change from Baseline to Month 24 | 
| Timed Up and Go (TUG) | Stand up from a chair, walk 3 meters, sit back down in chair | Change from Baseline to Month 24 | 
| Frequency of immunophenotypic abnormalities in blood and muscle | Laboratory assessment | Change from Baseline to Month 24 | 
| Forced Vital Capacity (FVC) (sitting) | breathing test | Change from Baseline to Month 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Manual Muscle Testing | muscle strength testing | Change from Baseline to Month 24 | 
| Hand Held Dynamometry | muscle strength testing | Change from Baseline to Month 24 | 
| Sydney Swallow Questionnaire | swallow ability questionnaire | Change from Baseline to Month 24 | 
| EAT-10 Questionnaire | swallow ability questionnaire | Change from Baseline to Month 24 | 
| NIH PROMIS questionnaires | quality of life questionnaires | Change from Baseline to Month 24 | 
| Forced Vital Capacity (supine) | breathing test done while lying down | Change from Baseline to Month 24 | 
| Maximum Inspiratory Pressure/Maximum Expiratory Pressure | breathing tests | Change from Baseline to Month 24 | 
| sporadic inclusion body myositis physical functioning assessment (sIFA) | functional ability questionnaire | Change from Baseline to Month 24 |