Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
11 de setembro de 2026 atualizado por: 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.
Visão geral do estudo
Status
Recrutamento
Descrição detalhada
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
Tipo de estudo
Observacional
Inscrição (Estimado)
30
Contactos e Locais
Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.
Contato de estudo
- Nome: Joseph D Niang, B.A.
- Número de telefone: 410-570-3949
- E-mail: jniang1@jh.edu
Estude backup de contato
- Nome: Pamela Arrazola, M.S.
- Número de telefone: 410-955-0749
- E-mail: parrazo1@jh.edu
Locais de estudo
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Maryland
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Baltimore, Maryland, Estados Unidos, 21287
- Recrutamento
- The Johns Hopkins Hospital
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Contato:
- Abdullah AlQahtani, MD MPH
- Número de telefone: 443-819-9480
- E-mail: aalqaht3@jh.edu
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Critérios de participação
Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Sim
Método de amostragem
Amostra Não Probabilística
População do estudo
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.
Descrição
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.
Plano de estudo
Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.
Como o estudo é projetado?
Detalhes do projeto
Coortes e Intervenções
Grupo / Coorte |
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Controles saudáveis
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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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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Bulbar Muscle Thickness Assessed by Neuromuscular Ultrasound
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Center for Neurologic Study Bulbar Function Scale Score
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Investigadores
- Investigador principal: Abdullah Z AlQahtani, MD MPH, the Johns Hopkins School of Medicine
Publicações e links úteis
A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.
Publicações Gerais
- 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.
Datas de registro do estudo
Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.
Datas Principais do Estudo
Início do estudo (Estimado)
1 de outubro de 2026
Conclusão Primária (Estimado)
31 de dezembro de 2027
Conclusão do estudo (Estimado)
31 de dezembro de 2027
Datas de inscrição no estudo
Enviado pela primeira vez
11 de setembro de 2026
Enviado pela primeira vez que atendeu aos critérios de CQ
11 de setembro de 2026
Primeira postagem (Real)
16 de setembro de 2026
Atualizações de registro de estudo
Última Atualização Postada (Real)
16 de setembro de 2026
Última atualização enviada que atendeu aos critérios de controle de qualidade
11 de setembro de 2026
Última verificação
1 de setembro de 2026
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Doenças musculoesqueléticas
- Doenças do Sistema Nervoso Central
- Doenças do Sistema Nervoso
- Doenças Neuromusculares
- Doenças Metabólicas
- Doenças Neurodegenerativas
- Doenças da Medula Espinhal
- Proteinopatias TDP-43
- Deficiências de Proteostase
- Doença do neurônio motor
- Condições Patológicas, Sinais e Sintomas
- Doenças Nutricionais e Metabólicas
- Sinais e sintomas
- Doenças Musculares
- Esclerose Lateral Amiotrófica
- Astenia
- Miosite
- Miosite, Corpo de Inclusão
Outros números de identificação do estudo
- IRB00553575
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
NÃO
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Não
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Não
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