INSPIRE-IBM: Sporadic Inclusion Body Myositis Natural History Study
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
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Detailed Description
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.
Study Design
Outcome Measures
Primary Outcome Measures
- Inclusion Body Myositis Functional Rating Scale (IBMFRS) [Change from Baseline to Month 24]
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.
- Timed Up and Go (TUG) [Change from Baseline to Month 24]
Stand up from a chair, walk 3 meters, sit back down in chair
- Frequency of immunophenotypic abnormalities in blood and muscle [Change from Baseline to Month 24]
Laboratory assessment
- Forced Vital Capacity (FVC) (sitting) [Change from Baseline to Month 24]
breathing test
Secondary Outcome Measures
- Manual Muscle Testing [Change from Baseline to Month 24]
muscle strength testing
- Hand Held Dynamometry [Change from Baseline to Month 24]
muscle strength testing
- Sydney Swallow Questionnaire [Change from Baseline to Month 24]
swallow ability questionnaire
- EAT-10 Questionnaire [Change from Baseline to Month 24]
swallow ability questionnaire
- NIH PROMIS questionnaires [Change from Baseline to Month 24]
quality of life questionnaires
- Forced Vital Capacity (supine) [Change from Baseline to Month 24]
breathing test done while lying down
- Maximum Inspiratory Pressure/Maximum Expiratory Pressure [Change from Baseline to Month 24]
breathing tests
- sporadic inclusion body myositis physical functioning assessment (sIFA) [Change from Baseline to Month 24]
functional ability questionnaire
Eligibility Criteria
Criteria
Inclusion Criteria:
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Ages 40 years and older
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Fulfills ENMC 2011 criteria of clinically definite or probable sporadic Inclusion body myositis (sIBM)
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Disease onset is within the past 10 years of the time of Baseline visit
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Able to participate and comply with study related procedures
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Able to provide written consent
Exclusion Criteria:
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Current or very recent use (within last 6 months of the Baseline visit) of immunomodulation or immunosuppression therapy.
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Current or very recent use (within last 90 days of the Baseline visit) of an investigational medication or therapy.
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Co-existing significant medical or surgical conditions that, in the opinion of the investigator, will influence study participation or alter natural history.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California, Los Angeles | Los Angeles | California | United States | 90095 |
| 2 | University of California, Irvine | Orange | California | United States | 92868 |
| 3 | University of Colorado | Aurora | Colorado | United States | 80045 |
| 4 | Kansas University Medical Center | Fairway | Kansas | United States | 66205 |
| 5 | Johns Hopkins University | Baltimore | Maryland | United States | 21287 |
| 6 | Brigham and Women's Hospital | Boston | Massachusetts | United States | 02115 |
| 7 | Washington University in St. Louis | Saint Louis | Missouri | United States | 63110 |
| 8 | Ohio State University | Columbus | Ohio | United States | 43221 |
| 9 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
| 10 | University of Pennsylvania | Philadelphia | Pennsylvania | United States | 19104 |
| 11 | University of Texas Southwestern | Dallas | Texas | United States | 75390 |
| 12 | Nerve and Muscle Center of Texas | Houston | Texas | United States | 77030 |
| 13 | University of Washington | Seattle | Washington | United States | 98195 |
Sponsors and Collaborators
- University of California, Irvine
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Investigators
- Principal Investigator: Tahseen Mozaffar, MD FAAN, FANA, University of California, Irvine
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- HS# 2021-6779
- R01AR078340-01