Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorJohns Hopkins University
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial population
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.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
30 Participants
are grouped into 4 trial groupsGroup A: Inclusion Body Myositis
Group B: Inflammatory or Genetic Myopathies
Group C: Motor neuron disease(ALS/PLS)
Group D: Healthy controls
Trial outcomes
Primary outcomes
1
Bulbar Muscle Thickness Assessed by Neuromuscular Ultrasound
Bulbar muscle thickness will be measured using B-mode neuromuscular ultrasound in short- and long-axis views.
Time frame
Day 1 (one-time study visit)2
Bulbar Muscle Cross-Sectional Area Assessed by Neuromuscular Ultrasound
Cross-sectional area of the assessed bulbar muscles will be measured using neuromuscular ultrasound.
Time frame
Day 1 (one-time study visit)3
Bulbar Muscle Echogenicity Assessed by Quantitative Grayscale Ultrasound
Bulbar muscle echogenicity will be evaluated using quantitative grayscale analysis of neuromuscular ultrasound images.
Time frame
Day 1 (one-time study visit)4
Bulbar Muscle Echogenicity Assessed by the Heckmatt Grading Scale
Bulbar muscle echogenicity will be assessed using the Heckmatt grading scale on neuromuscular ultrasound images. Scale 1-4, lower score better.
Time frame
Day 1 (one-time study visit)Secondary outcomes
1
Center for Neurologic Study Bulbar Function Scale Score
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.
Time frame
Day 1 (one-time study visit)2
Neuromuscular Disease Swallowing Status Scale Score
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.
Time frame
Day 1 (one-time study visit)3
Eating Assessment Tool-10 Score
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.
Time frame
Day 1 (one-time study visit)4
Sydney Swallow Questionnaire Score
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.
Time frame
Day 1 (one-time study visit)Sponsors and contacts
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Source ClinicalTrials.gov