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Completed

NCT Number: NCT03414086

Predictor of Clinical Response to Acthar in Myositis

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

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15261, United States

About this study

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".

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Healthy Control

Other

Ten healthy adult patients evaluating serum, PBMC's, and RNA.

Myositis in Remission

Other

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.

Primary outcomes

  1. IMACS Core Set Measures Improvement

    Time frame: 6 Months

    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.

Secondary outcomes

  1. Myositis Response Criteria

    Time frame: 6 Months

    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.

Sponsors and collaborators

Lead sponsor

University of Pittsburgh

Other

Registry information

Official study title

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

Important dates

Study start
2017
Primary completion
2022
Study completion
2022
First posted
Jan 29, 2018
Registry last updated
Aug 9, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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