Predictor of Clinical Response to Acthar in Myositis

Study Overview
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Brief Summary
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Comparing the clinical effects of Acthar Gel before and after treatment and compare it to patients with inactive disease.

Detailed Description
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To compare the clinical impact of Acthar Gel at the cellular and molecular level before and after treatment and compare it to patients with inactive disease. The cohort of active myositis subjects are not actually enrolled in this study, but rather the data for those with active myositis will be obtained from a previously completed trial entitled Efficacy and safety of Adenocorticotropic Hormone Gel in Refractory dermatomyositis and polymyositis.

Official Title
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Predictor of Clinical Response to Acthar in Myositis: Phase II of Acthar Clinical Trial

Conditions
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Myositis, Dermatomyositis, Polymyositis

Intervention / Treatment
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* Other: Healthy Control
* Other: Myositis in Remission


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Healthy Controls: An individual will be eligible to be a control subject if his/her age is 18 years or greater. Myositis Remission Control Group Definite or probable PM or DM by Bohan and Peter criteria. PM patients must either possess a myositis-associated autoantibody or undergo adjudication for confirmation of the PM diagnosis by consensus of two experts (Aggarwal or Oddis) to ensure non-PM patients are not enrolled. This step is necessary since there are well known mimics of PM. Age is greater than or equal to 18 years Remission of myositis as defined by a myositis disease global activity score <1 on the MDAAT and no new immunosuppressive or glucocorticoid therapy or dose change within one year. Exclusion Criteria: Healthy Controls: An existing diagnosis of a CTD A potential immune compromised state, for example, treatment with immunosuppressant or anti-rejection medication or a diagnosis of an immune deficiency disease Myositis Remission Control Group: Juvenile DM or PM, myositis in overlap with another connective tissue disease, cancer associated myositis, inclusion body myositis, or any other non immune mediated myopathy. Severe muscle damage defined as a baseline global muscle damage score on the MDI (Myositis Damage Index) of greater than or equal to five centimeters on a ten centimeter VAS. Patients with malignancy within three years of screening (except basal cell cancer or squamous cell cancer of skin. Uncontrolled diabetes, hepatic or renal disease.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Myositis in Remission<br>Subjects who are in remission with their myositis diagnosis. | Other: Myositis in Remission<br>* Ten patients (four who have polymyositis, six dermatomyositis) from our database who are followed in the Myositis Center. We will collect serum, PBMC's, and RNA samples will be obtained at baseline and at six months. Remission is defined as a global myositis disease activity score less than or equal to 1 on the MDAAT assessments with no new immunosuppressive drug or glucocorticoid use and no increase in dose of either in the preceding year.<br>|
| Healthy Controls<br>Subjects who do not have a myositis diagnosis. | Other: Healthy Control<br>* Ten healthy adult patients evaluating serum, PBMC's, and RNA.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| IMACS Core Set Measures Improvement | The International Myositis Assessment & Clinical Studies (IMACS) definition of improvement: Three of any of the six core set measures improved by greater than or equal to twenty percent, with no more than two core set measuring by greater than or equal to twenty five percent. | 6 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Myositis Response Criteria | This criteria yields a continuous improvement score which can be more readily extrapolated to individual response in subjects allowing correlation with baseline and longitudinal immunologic markers in these subjects. | 6 Months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PM, DM