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Adult and Juvenile Myositis

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ClinicalTrials.gov Identifier: NCT00017914
Recruitment Status : Recruiting
First Posted : June 20, 2001
Last Update Posted : April 25, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Environmental Health Sciences (NIEHS) )

Tracking Information
First Submitted Date June 19, 2001
First Posted Date June 20, 2001
Last Update Posted Date April 25, 2019
Study Start Date June 8, 1994
Primary Completion Date Not Provided
Current Primary Outcome Measures
 (submitted: February 21, 2015)
Physician global assessment form [ Time Frame: Each study visit in myositis patients ]
Original Primary Outcome Measures Not Provided
Change History Complete list of historical versions of study NCT00017914 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Adult and Juvenile Myositis
Official Title Studies in the Natural History and Pathogenesis of Childhood-Onset and Adult-Onset Idiopathic Inflammatory Myopathies
Brief Summary

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.

Detailed Description

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.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Not Provided
Study Population Not Provided
Condition
  • Dermatomyositis
  • Polymyositis
  • Inclusion Body Myositis
  • Juvenile Dermatomyositis
  • Juvenile Polymyositis
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: May 3, 2014)
1200
Original Enrollment
 (submitted: June 23, 2005)
600
Study Completion Date Not Provided
Primary Completion Date Not Provided
Eligibility Criteria
  • INCLUSION CRITERIA:

All Patients admitted to the study must satisfy at least one of the following criteria among item 1 (A or B or C) OR item 2 (A or B or C) AND item 3:

  1. Patient has documented evidence that he/she meets criteria for an idiopathic inflammatory myopathy

    A. Possible, probable or definite adult or juvenile polymyositis or dermatomyositis by Bohan and Peter criteria

    B. Possible, probable or definite inclusion body myositis by Griggs and/or ENMC criteria

    C. Has an idiopathic inflammatory myopathy that does not meet these criteria, including common or rarer forms of myositis such as cancer- associated, focal, orbital, eosinophilic myositis, macrophagic, proliferative, etc.

  2. Has one of the following conditions:

2A. Have a disorder that is related to an idiopathic inflammatory myopathy, that may include:

i. Inflammatory and non-inflammatory myopathies:

ii. Mimicking and related skin disorders:

iii. Disorders of the complications of myositis (including interstitial lung disease, calcifying disorders, cardiomyopathies, etc.):

iv. Overlapping autoimmune diseases that may be associated with myositis

v. Patients with muscle and/or skin inflammation and documented environmental exposures:

vi. Patients with myositis or complications of myositis and suspected genetic disorders:

vii. Patients with undifferentiated connective tissue disease

viii. 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.

2B. For patients with at least one first-degree relative affected with IIIM, all available first-degree relatives (affected and unaffected) are eligible to participate in the genetics portion of the protocol.

2C. Healthy control subject needed for sub-studies in the protocol. (see Protocol Section 7, Healthy Control Subjects).

3. Ability of patient or parent/guardian to give informed consent to all or part of the study after full information has been provided.

EXCLUSION CRITERIA:

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..

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers Yes
Contacts
Contact: Lisa G Rider, M.D. (301) 451-6272 riderl@mail.nih.gov
Listed Location Countries United States
Removed Location Countries Canada
 
Administrative Information
NCT Number NCT00017914
Other Study ID Numbers 940165
94-E-0165
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Institute of Environmental Health Sciences (NIEHS) )
Study Sponsor National Institute of Environmental Health Sciences (NIEHS)
Collaborators Not Provided
Investigators
Principal Investigator: Lisa G Rider, M.D. National Institute of Environmental Health Sciences (NIEHS)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date November 28, 2018