Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
11. september 2026 oppdatert av: 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.
Studieoversikt
Status
Rekruttering
Detaljert beskrivelse
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
Studietype
Observasjonsmessig
Registrering (Antatt)
30
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Joseph D Niang, B.A.
- Telefonnummer: 410-570-3949
- E-post: jniang1@jh.edu
Studer Kontakt Backup
- Navn: Pamela Arrazola, M.S.
- Telefonnummer: 410-955-0749
- E-post: parrazo1@jh.edu
Studiesteder
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Maryland
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Baltimore, Maryland, Forente stater, 21287
- Rekruttering
- The Johns Hopkins Hospital
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Ta kontakt med:
- Abdullah AlQahtani, MD MPH
- Telefonnummer: 443-819-9480
- E-post: aalqaht3@jh.edu
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Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Voksen
- Eldre voksen
Tar imot friske frivillige
Ja
Prøvetakingsmetode
Ikke-sannsynlighetsprøve
Studiepopulasjon
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.
Beskrivelse
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.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
Kohorter og intervensjoner
Gruppe / Kohort |
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Sunne kontroller
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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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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Bulbar Muscle Thickness Assessed by Neuromuscular Ultrasound
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Center for Neurologic Study Bulbar Function Scale Score
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Etterforskere
- Hovedetterforsker: Abdullah Z AlQahtani, MD MPH, the Johns Hopkins School of Medicine
Publikasjoner og nyttige lenker
Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.
Generelle publikasjoner
- 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.
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Antatt)
1. oktober 2026
Primær fullføring (Antatt)
31. desember 2027
Studiet fullført (Antatt)
31. desember 2027
Datoer for studieregistrering
Først innsendt
11. september 2026
Først innsendt som oppfylte QC-kriteriene
11. september 2026
Først lagt ut (Faktiske)
16. september 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
16. september 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
11. september 2026
Sist bekreftet
1. september 2026
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Muskel- og skjelettsykdommer
- Sykdommer i sentralnervesystemet
- Sykdommer i nervesystemet
- Nevromuskulære sykdommer
- Metabolske sykdommer
- Nevrodegenerative sykdommer
- Ryggmargssykdommer
- TDP-43 Proteinopatier
- Proteostase mangler
- Motor Neuron sykdom
- Patologiske tilstander, tegn og symptomer
- Ernæringsmessige og metabolske sykdommer
- Tegn og symptomer
- Muskelsykdommer
- Amyotrofisk lateral sklerose
- Asteni
- Myositt
- Myositt, Inclusion Body
Andre studie-ID-numre
- IRB00553575
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Nei
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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