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| ID | Type | Description | Link |
|---|---|---|---|
| R01AR078340-01 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) | NIH |
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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.
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.
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| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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Paticipants age 40 and older with sporadic IBM, diagnosed according to established criteria.
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| Name | Affiliation | Role |
|---|---|---|
| Tahseen Mozaffar, MD FAAN, FANA | University of California, Irvine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Los Angeles | Los Angeles | California | 90095 | United States | ||
| University of California, Irvine |
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DNA and RNA will be isolated from blood and muscle samples and stored for future research
| 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 |
| Orange |
| California |
| 92868 |
| United States |
| University of Colorado | Aurora | Colorado | 80045 | United States |
| University of Miami | Miami | Florida | 33136 | United States |
| Kansas University Medical Center | Fairway | Kansas | 66205 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21287 | United States |
| Brigham and Women's Hospital | Boston | Massachusetts | 02115 | United States |
| Washington University in St. Louis | St Louis | Missouri | 63110 | United States |
| Ohio State University | Columbus | Ohio | 43221 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| Nerve and Muscle Center of Texas | Houston | Texas | 77030 | United States |
| University of Washington | Seattle | Washington | 98195 | United States |
| ID | Term |
|---|---|
| D018979 | Myositis, Inclusion Body |
| ID | Term |
|---|---|
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
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