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)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 150
- 试验地点
- 13
- 主要终点
- Forced Vital Capacity (FVC) (sitting)
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 40 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
结局指标
主要结局
Forced Vital Capacity (FVC) (sitting)
时间窗: Change from Baseline to Month 24
breathing test
Timed Up and Go (TUG)
时间窗: Change from Baseline to Month 24
Stand up from a chair, walk 3 meters, sit back down in chair
Inclusion Body Myositis Functional Rating Scale (IBMFRS)
时间窗: 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.
Frequency of immunophenotypic abnormalities in blood and muscle
时间窗: Change from Baseline to Month 24
Laboratory assessment
次要结局
- Sydney Swallow Questionnaire(Change from Baseline to Month 24)
- NIH PROMIS questionnaires(Change from Baseline to Month 24)
- Maximum Inspiratory Pressure/Maximum Expiratory Pressure(Change from Baseline to Month 24)
- Manual Muscle Testing(Change from Baseline to Month 24)
- EAT-10 Questionnaire(Change from Baseline to Month 24)
- Forced Vital Capacity (supine)(Change from Baseline to Month 24)
- sporadic inclusion body myositis physical functioning assessment (sIFA)(Change from Baseline to Month 24)
- Hand Held Dynamometry(Change from Baseline to Month 24)
研究者
Tahseen Mozaffar
Professor of Neurology
University of California, Irvine