Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
2026年9月11日 更新者：Johns Hopkins University
Advancing Bulbar Assessment in Inclusion Body Myositis Through a Multimodal Imaging Approach
This study will evaluate whether neuromuscular ultrasound and MRI can be used as imaging markers of bulbar involvement and swallowing dysfunction in individuals with inclusion body myositis (IBM). Researchers will compare imaging and clinical findings in participants with IBM with those in participants with other myopathies, ALS, PLS, and healthy volunteers.
The study is a one-time visit. Study procedures may include neurological assessments, swallowing questionnaires, tongue strength testing, neuromuscular ultrasound, and MRI. The goal is to identify more sensitive and objective ways to assess bulbar dysfunction biomarkers.
研究概览
详细说明
Researchers will evaluate ultrasound and Magnetic Resonance Imaging (MRI) as imaging markers to diagnose bulbar involvement and dysphagia in patients with IBM.
Objectives
Primary Objectives:
- Evaluate neuromuscular ultrasound as an imaging marker for assessing the presence and evaluate the severity of dysphagia and bulbar involvement in IBM
- Evaluate MRI of the bulbar region as an imaging marker for assessing the presence and evaluate the severity of dysphagia and bulbar involvement in IBM
Secondary Objectives:
- Evaluate ultrasound elastography (USE) as an imaging tool for assessing muscle stiffness in patients with IBM and compare the findings to motor neuron disease and healthy control cohorts
- Evaluate which imaging modality could be used as an early indicator of dysphagia and bulbar involvement
研究类型
观察性的
注册 (估计的)
30
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Joseph D Niang, B.A.
- 电话号码：410-570-3949
- 邮箱：jniang1@jh.edu
研究联系人备份
- 姓名：Pamela Arrazola, M.S.
- 电话号码：410-955-0749
- 邮箱：parrazo1@jh.edu
学习地点
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Maryland
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Baltimore、Maryland、美国、21287
- 招聘中
- The Johns Hopkins Hospital
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接触：
- Abdullah AlQahtani, MD MPH
- 电话号码：443-819-9480
- 邮箱：aalqaht3@jh.edu
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
是的
取样方法
非概率样本
研究人群
The study population will include approximately 30 participants with inclusion body myositis (IBM), amyotrophic lateral sclerosis (ALS), or primary lateral sclerosis (PLS); non-IBM inflammatory or genetic myopathies; and healthy controls.
The planned enrollment includes 10 participants with IBM and swallowing difficulties, 5 participants with IBM without swallowing difficulties, 5 participants with non-IBM inflammatory or genetic myopathies, 5 participants with bulbar ALS, and 5 healthy controls.
The investigators will recruit patients primarily through the clinical practices of the principal investigator and co-investigators and through referrals.
Healthy controls may be recruited from Johns Hopkins University/Johns Hopkins Hospital staff, Johns Hopkins affiliates, and family members of patients and staff.
The participants will be screened to exclude pre-existing neuromuscular disorders.
Healthy controls will be age-matched to the IBM cohort.
描述
Inclusion Criteria:
- Participants must have a confirmed diagnosis of inclusion body myositis (IBM), amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), or myopathy in accordance with the respective diagnostic criteria.
- Participants with disease must have clinically relevant laboratory testing for disease biomarkers (e.g., Creatine Kinase (CK), Comprehensive Metabolic Panel (CMP), Complete Blood Count (CBC)) within the last 12 months.
- Participants must be able to remain supine for 30 minutes or longer and cannot have significant breathing difficulties.
- Healthy controls must not have any neuromuscular disease.
Exclusion Criteria:
- Unable or unwilling to provide written informed consent.
- Medical history or clinically significant physical examination or laboratory results that, in the opinion of the investigator, would render the participant unsuitable for the study.
- Participants who are unlikely to comply with the study protocol or, in the opinion of the investigator, would not be a suitable candidate for participation in the study.
- Patients with IBM, ALS, or myopathy who have another known neuromuscular condition with known bulbar involvement.
- Any patient who has undergone a procedure for dysphagia, including myotomy, dilatation, or Botox.
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
队列和干预
团体/队列 |
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健康控制
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Inclusion Body Myositis
Participants with diagnosis of IBM
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Inflammatory or Genetic Myopathies
other types of myopathy that is not IBM
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Motor neuron disease(ALS/PLS)
Participants must have spastic dysarthria
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Bulbar Muscle Thickness Assessed by Neuromuscular Ultrasound
大体时间：Day 1 (one-time study visit)
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Bulbar muscle thickness will be measured using B-mode neuromuscular ultrasound in short- and long-axis views.
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Day 1 (one-time study visit)
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Bulbar Muscle Cross-Sectional Area Assessed by Neuromuscular Ultrasound
大体时间：Day 1 (one-time study visit)
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Cross-sectional area of the assessed bulbar muscles will be measured using neuromuscular ultrasound.
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Day 1 (one-time study visit)
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Bulbar Muscle Echogenicity Assessed by Quantitative Grayscale Ultrasound
大体时间：Day 1 (one-time study visit)
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Bulbar muscle echogenicity will be evaluated using quantitative grayscale analysis of neuromuscular ultrasound images.
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Day 1 (one-time study visit)
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Bulbar Muscle Echogenicity Assessed by the Heckmatt Grading Scale
大体时间：Day 1 (one-time study visit)
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Bulbar muscle echogenicity will be assessed using the Heckmatt grading scale on neuromuscular ultrasound images.
Scale 1-4, lower score better.
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Day 1 (one-time study visit)
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Bulbar Muscle Volume Assessed by Magnetic Resonance Imaging
大体时间：Day 1 (one-time study visit)
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Bulbar muscle volume will be measured from magnetic resonance imaging and reported in cubic centimeters (cm³).
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Day 1 (one-time study visit)
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Bulbar Muscle Cross-Sectional Area Assessed by Magnetic Resonance Imaging
大体时间：Day 1 (one-time study visit)
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Bulbar muscle cross-sectional area will be measured from magnetic resonance imaging and reported in square centimeters (cm²).
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Day 1 (one-time study visit)
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Bulbar Muscle Fat Content Assessed by Magnetic Resonance Imaging
大体时间：Day 1 (one-time study visit)
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Bulbar muscle fat content will be measured using magnetic resonance imaging and reported as a percentage (%).
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Day 1 (one-time study visit)
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Tongue Muscle Stiffness Assessed by Ultrasound Elastography
大体时间：Day 1 (one-time study visit)
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Tongue muscle stiffness will be quantitatively assessed using shear-wave ultrasound elastography.
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Day 1 (one-time study visit)
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Center for Neurologic Study Bulbar Function Scale Score
大体时间：Day 1 (one-time study visit)
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Bulbar function will be assessed using the Center for Neurologic Study Bulbar Function Scale (CNS-BFS), a 21-item patient-reported questionnaire evaluating speech, swallowing, and salivation.
Score ranging from 21 to 112, where a score of 21 indicates no symptoms of bulbar dysfunction.
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Day 1 (one-time study visit)
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Neuromuscular Disease Swallowing Status Scale Score
大体时间：Day 1 (one-time study visit)
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Swallowing function will be assessed using the Neuromuscular Disease Swallowing Status Scale (NdSSS), an 8-stage clinician-rated scale ranging from normal oral intake to complete dependence on non-oral feeding.
Stage 8 represents normal function, while Stage 1 represents the most severe impairment.
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Day 1 (one-time study visit)
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Eating Assessment Tool-10 Score
大体时间：Day 1 (one-time study visit)
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Dysphagia symptoms and the symptoms impact on quality of life will be assessed using the 10-item Eating Assessment Tool (EAT-10).
Total score ranges from 0 to 40, with a score of 3 or higher indicating a potential swallowing problem.
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Day 1 (one-time study visit)
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Sydney Swallow Questionnaire Score
大体时间：Day 1 (one-time study visit)
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Severity of oropharyngeal dysphagia symptoms will be assessed using the 17-item Sydney Swallow Questionnaire (SSQ).
Score range 0 to 1,700, with higher scores indicating more severe swallowing problem.
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Day 1 (one-time study visit)
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Maximum Tongue Strength Assessed by the Iowa Oral Performance Instrument
大体时间：Day 1 (one-time study visit)
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Maximum tongue strength will be measured using the Iowa Oral Performance Instrument (IOPI).
Three trials will be performed, and the highest pressure will be recorded in kilopascals (kPa).
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Day 1 (one-time study visit)
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Tongue Endurance Assessed by the Iowa Oral Performance Instrument
大体时间：Day 1 (one-time study visit)
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Tongue endurance will be measured using the Iowa Oral Performance Instrument (IOPI) as the amount of time, in seconds, the participant can maintain 50% of their maximum tongue pressure.
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Day 1 (one-time study visit)
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Inclusion Body Myositis Functional Rating Scale Score
大体时间：Day 1 (one-time study visit)
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Functional status in participants with inclusion body myositis will be assessed using the Inclusion Body Myositis Functional Rating Scale (IBMFRS), including swallowing-related function.
Score ranges from 0 to 40, higher score better.
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Day 1 (one-time study visit)
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Amyotrophic Lateral Sclerosis Functional Rating Scale Score
大体时间：Day 1 (one-time study visit)
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Functional disability and disease severity in participants with amyotrophic lateral sclerosis will be assessed using the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS).
Score ranges from 0 (maximum disability/worst performance) to 48 (normal function).
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Day 1 (one-time study visit)
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 首席研究员：Abdullah Z AlQahtani, MD MPH、the Johns Hopkins School of Medicine
出版物和有用的链接
负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。
一般刊物
- Jackson CE, Barohn RJ, Gronseth G, Pandya S, Herbelin L; Muscle Study Group. Inclusion body myositis functional rating scale: a reliable and valid measure of disease severity. Muscle Nerve. 2008 Apr;37(4):473-6. doi: 10.1002/mus.20958.
- Wada A, Kawakami M, Liu M, Otaka E, Nishimura A, Liu F, Otsuka T. Development of a new scale for dysphagia in patients with progressive neuromuscular diseases: the Neuromuscular Disease Swallowing Status Scale (NdSSS). J Neurol. 2015 Oct;262(10):2225-31. doi: 10.1007/s00415-015-7836-y. Epub 2015 Jul 4.
- Reyngoudt H, Baudin PY, Caldas de Almeida Araujo E, Bachasson D, Boisserie JM, Mariampillai K, Annoussamy M, Allenbach Y, Hogrel JY, Carlier PG, Marty B, Benveniste O. Effect of sirolimus on muscle in inclusion body myositis observed with magnetic resonance imaging and spectroscopy. J Cachexia Sarcopenia Muscle. 2024 Jun;15(3):1108-1120. doi: 10.1002/jcsm.13451. Epub 2024 Apr 13.
- McIlduff CE, Martucci MG, Shin C, Qi K, Pacheck AK, Gutierrez H, Mortreux M, Rutkove SB. Quantitative ultrasound of the tongue: Echo intensity is a potential biomarker of bulbar dysfunction in amyotrophic lateral sclerosis. Clin Neurophysiol. 2020 Oct;131(10):2423-2428. doi: 10.1016/j.clinph.2020.06.027. Epub 2020 Jul 17.
- Anderson NC, Lloyd TE. Inclusion body myositis: an update. Curr Opin Rheumatol. 2025 Jan 1;37(1):80-85. doi: 10.1097/BOR.0000000000001060. Epub 2024 Oct 21.
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年10月1日
初级完成 (估计的)
2027年12月31日
研究完成 (估计的)
2027年12月31日
研究注册日期
首次提交
2026年9月11日
首先提交符合 QC 标准的
2026年9月11日
首次发布 (实际的)
2026年9月16日
研究记录更新
最后更新发布 (实际的)
2026年9月16日
上次提交的符合 QC 标准的更新
2026年9月11日
最后验证
2026年9月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.