Clinical trial NCT03414086: Predictor of Clinical Response to Acthar in Myositis
Official title: Predictor of Clinical Response to Acthar in Myositis: Phase II of Acthar Clinical Trial
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myositis
Dermatomyositis
Polymyositis
Keywords: PM
DM
Lead sponsor: University of Pittsburgh
Sponsor class: OTHER
First posted: 2018-01-29
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Arthritis and Autoimmunity Center

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

Detailed description: 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".

Eligibility criteria: 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.

Primary outcomes:
measure: IMACS Core Set Measures Improvement
timeFrame: 6 Months
Secondary outcomes:
measure: Myositis Response Criteria
timeFrame: 6 Months
Other outcomes:
Locations:
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
Study officials:
affiliation: University of Pittsburgh
name: Rohit Aggarwal, MD
role: PRINCIPAL_INVESTIGATOR
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