Adult and Juvenile Myositis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2-100
SponsorNational Institute of Environmental Health Sciences (NIEHS)
About this trial
This study will evaluate subjects with adult- and childhood-onset myositis to learn more about their cause and the immune system changes and medical problems associated with them. Myositis is an inflammatory muscle disease that can damage muscles and other organs, resulting in significant disability.
Children or adults with polymyositis or dermatomyositis or a related condition may be evaluated under this study. Healthy children or adults will also be enrolled as "controls," for comparison of test results.
All patients will undergo a complete history (including completing some questionnaires) and physical examination, review of medical records, and blood and urine tests. Patients may then choose to participate in an additional 1- to 5-day evaluation, which will include some or all of the following diagnostic, treatment or research procedures:
1. Standardized muscle strength testing, range of motion of joints and walking (gait) analysis by a physiotherapist; completion of a questionnaire regarding ability to perform daily tasks 2. Skin assessment, possibly including photographs of lesions and a skin biopsy (removal of a small skin sample under local anesthetic) 3. Magnetic resonance imaging (scans that use magnetic fields to visualize tissues) of leg muscles 4. Swallowing studies, including a physical examination and questionnaire on swallowing ability, studies of tongue strength, and ultrasound imaging during swallowing, and possibly, a modified barium swallow 5. Voice and speech assessment, possibly including computerized voice analysis and laryngoscopy-analysis of the larynx (voice box) using a small rigid scope with a camera placed in the mouth to view and record vocal cord function 6. Pulmonary function tests (measurement of air moved into and out of the lungs, using a breathing machine) to evaluate lung function and, possibly, chest X-ray 7. Electrocardiogram (measurement of the electrical activity of the heart) and, possibly, echocardiogram (ultrasound imaging of the heart) 8. Endocrine evaluation 9. Eye examination, in patients with vision loss or other eye symptoms 10. Nutrition assessment to evaluate muscle mass and muscle wasting, including tape measurements or bioelectric impedance testing, a painless procedure in which wires are attached to the extremities with a sticky paste. 11. Muscle ultrasound. 12. Electromyography (record of the electrical activity of muscles) 13. Muscle or skin biopsy (removal of a small piece of muscle tissue for microscopic examination)
All patients may have only a one-time evaluation or may return for one follow-up evaluations (either the 1-day or 3- to 5-day evaluation) over a 1-year period.
Healthy children will undergo a medical history and brief physical examination; blood and urine tests; speech and swallowing studies including questionnaires and physical examination, tongue strength, and ultrasound study; and bioelectric impedance testing. Children 8 to 18 years old may also have exercise testing.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
Patient has documented evidence that he/she meets criteria for an idiopathic inflammatory myopathy
Overlapping autoimmune diseases that may be associated with myositis
Patients with undifferentiated connective tissue disease
Patients with signs or symptoms of myositis (such as weakness, skin rashes, interstitial lung disease) or laboratory abnormalities (such as elevated CK or muscle biopsy with myopathic features) who do not have an established diagnosis of myositis for them to be evaluated to establish a diagnosis
Disqualifiers
Patients <2 years or >100 years will be excluded.
Any conditions in which the drawing of the amount of blood required or undergoing procedures needed for the study is not deemed medically appropriate by the treating physician or the principal investigator.
First or more distant relative of a proband with myositis, for genetics studies.
Ability of patient or parent/guardian to give informed consent to all or part of the study after full information has been provided.
Trial population
1- Any patient who has documented evidence that he/she meets criteria for an idiopathic inflammatory myopathy; 2- Any patient who has been diagnosed with inflammatory/non-inflammatory myopathies, mimicking and related skin disorders, disorders of complications of myositis, overlapping autoimmune diseases that may be associated with myositis, patients with muscle and/or skin inflammation and documented environmental exposures, patients with myositis or complications of myositis and suspected genetic disorders, patients with undifferentiated connective tissue disease, patients with signs or symptoms of myositis or laboratory abnormalities who do not have an established diagnosis of myositis for them to be evaluated to establish a diagnosis; 3- Close relatives of patients with at least one first-degree relative affected with IIIM (affected or unaffected siblings, children, parents, grandparents) can participate in the genetics portion of the protocol; 4- Healthy control subjects.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
1,200 Participants
are grouped into 3 trial groupsGroup A: Healthy Volunteer
Group B: Myositis Patient
Group C: Non-Myositis Patient
Trial outcomes
Primary outcomes
1
Physician Global Assessment Form
1- A secure internet accessible IMACS Outcomes Data Repository of core set disease activity, damage and quality of life measures has been established for adult and juvenile myositis patients as part of IMACS. 2- Compare the clinical and immunopathogenic features of childhood and adult IIM to determine the differences of IIM in these two populations referred to NIH 3- investigate the immunopathogenesis and immunogenetics of IIM.
Time frame
At enrollment and each study visitSponsors and contacts
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Source ClinicalTrials.gov