NIH Clinical Research Studies

Protocol Number: 94-E-0165

Active Accrual, Protocols Recruiting New Patients

Title:
Studies in the Natural History and Pathogenesis of Childhood Idiopathic Inflammatory Myopathies
Number:
94-E-0165
Summary:
This study will evaluate subjects with 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 under 18 years of age with polymyositis or dermatomyositis or a related condition, or adults with onset of these diseases during childhood may be evaluated under this study. Healthy children will also be enrolled as "controls," for comparison of test results.

All patients will undergo a 1-day evaluation with 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 3- 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. Exercise bicycle testing in children 8 to 18 years of age (exercising for up to 12 minutes while heart rate, blood pressure, electrocardiogram, and oxygen uptake are monitored).

12. Electromyography (record of the electrical activity of muscles)

13. Muscle 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 two or three 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.

Sponsoring Institute:
National Institute of Environmental Health Sciences (NIEHS)
Recruitment Detail
Type: Active Accrual Of New Subjects
Gender: Male & Female
Referral Letter Required: Yes
Population Exclusion(s): None

Eligibility Criteria:
INCLUSION CRITERIA:

All patients admitted to the study must satisfy the following criteria:

1. Meet probable or definite criteria for IIM by Bohan and Peter.

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

3. Selected patients with undefined illness who may have weakness, myalgias, or an elevated CK may be evaluated to establish a diagnosis and, if they are found to have IIM, would be offered the opportunity to enter the study. Such patients would undergo history and physical examination, blood tests when clinically indicated (incl 20 cc for research purposes), and other procedures as clinically indicated, including an MRI examination to establish an optimal site for muscle biopsy.

EXCLUSION CRITERIA:

1. Severe medical diseases requiring intensive care or any other conditions in which drawing 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.

Special Instructions:
This protocol has a particular interest in non-Caucasian patients with forms of myositis other than dermatomyositis.
Keywords:
Juvenile Dermatomyositis
Juvenile Polymyositis
Autoimmune Diseases
Disease Activity Assessment
Myositis-Specific Autoantibodies
Recruitment Keywords:
Idiopathic Inflammatory Myopathy
Conditions:
Dermatomyositis
Polymyositis
Idiopathic Inflammatory Myopathy
Healthy
Investigational Drug(s):
None
Investigational Device(s):
None

Contacts:
Patient Recruitment and Public Liaison Office
Building 61
10 Cloister Court
Bethesda, Maryland 20892-4754
Toll Free: 1-800-411-1222
TTY: 301-594-9774 (local),1-866-411-1010 (toll free)
Fax: 301-480-9793

Electronic Mail:prpl@mail.cc.nih.gov

Citations:
Rider LG. Outcome assessment in the adult and juvenile idiopathic inflammatory myopathies. Rheum Dis Clin North Am. 2002 Nov;28(4):935-77. Review. PMID: 12506779

Rider LG, Schiffenbauer AS, Zito M, Lim KL, Ahmed A, Zemel LS, Rennebohm RM, Passo MH, Summers RM, Hicks JE, Lachenbruch PA, Heyes MP, Miller FW. Neopterin and quinolinic acid are surrogate measures of disease activity in the juvenile idiopathic inflammatory myopathies. Clin Chem. 2002 Oct;48(10):1681-8. PMID: 12324484

Artlett CM, Ramos R, Jiminez SA, Patterson K, Miller FW, Rider LG. Chimeric cells of maternal origin in juvenile idiopathic inflammatory myopathies. Childhood Myositis Heterogeneity Collaborative Group. Lancet. 2000 Dec. 23-30;356(9248):2155-6. PMID: 11191545

Active Accrual, Protocols Recruiting New Patients

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