Natural History of Inflammatory Muscle Diseases
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Enrollment
- 500
- Locations
- 1
- Primary Endpoint
- Define the different types of inflammatory muscle diseases, their etiology, pathogenesis, prognostic factors, and response to different treatments.
Study Overview
Brief Summary
Objective:
To collect information and biospecimens (such as blood, muscle, and skin samples) that will be used to research causes and treatments of inflammatory muscle diseases.
Eligibility:
People aged 2 years and older with suspected or confirmed inflammatory muscle disease. Healthy volunteers aged 18 years and older are also needed.
Design:
Participants will have at least 1 clinic visit. Each visit will last 4 to 8 hours. Some may return for additional visits.
All participants will undergo these procedures (unless they are unable to):
- Physical exam, including blood and urine tests.
- Magnetic resonance imaging (MRI) scan of the thigh. Participants will lie still on a table with padding around 1 thigh. The table will slide into a tube. The scan will last for approximately 40 minutes.
Some procedures are optional:
- Muscle biopsy: An area of skin will be numbed. A quarter-inch cut will be made. Several pieces of muscle tissue, about the size of grains of rice, will be removed.
- Skin biopsy: An area of skin will be numbed. A piece of skin about a quarter inch in diameter will be removed.
- Lymph node biopsy: A small sample of fluid or tissue from a lymph node will taken to check for infection.
- Genetic testing. Some of the samples collected may be used for genetic testing.
Detailed Description
Study Description:
This is an observational study to characterize the different types of inflammatory myopathies, understand their etiology, pathogenesis, prognosis, and response to different treatments.
Objectives:
Primary objective: Define the different types of inflammatory muscle diseases, their etiology, pathogenesis, prognostic factors, and response to different treatments.
Secondary objectives: Study different diagnostic modalities that may help to diagnose and monitor the evolution of the disease in patients with myositis.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 2 Years to 120 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •INCLUSION CRITERIA:
- •In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- •1. Ability of subject, or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent and/or assent document.
- •2a. Patients are adults and minors of age 2 or older, with possible inflammatory myopathy (suspected or confirmed)
- •2b. Healthy volunteers will be adults aged 18 or older.
Exclusion Criteria
- •1. Unwilling to participate in research studies or to provide research samples or data.
Arms & Interventions
Healthy Volunteers
18 years old and up
Patients
greater than 2 years old
Outcomes
Primary Outcomes
Define the different types of inflammatory muscle diseases, their etiology, pathogenesis, prognostic factors, and response to different treatments.
Time Frame: End of each visit
Severity of muscle, skin, pulmonary, and joint involvement. Study of the immune dysregulation, transcriptomic changes, and genetic modifications in patients with inflammatory muscle diseases.
Secondary Outcomes
- Study different diagnostic modalities that may help to diagnose and monitor the evolution of the disease in patients with myositis.(at baseline)