Acthar in Treatment of Refractory Dermatomyositis and Polymyositis
Condition: Dermatomyositis; Polymyositis
MeSH terms: Dermatomyositis; Polymyositis
Intervention: Adrenocorticotropic Hormone Gel
Intervention description: H.P. Acthar Gel 80 units will be self-administered subcutaneously twice weekly by the subject for a period of 6 months.
Intervention MeSH terms: Hormones; Adrenocorticotropic Hormone; Melanocyte-Stimulating Hormones; beta-Endorphin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Masking description: Open Label
Enrollment: 12.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: dermatomyositis; polymyositis

Summary:

      The purpose of this research study is to evaluate the effectiveness of the study drug, ACTH
      Gel in people diagnosed with dermatomyositis a disease that causes muscle weakness and is
      associated with a rash (DM) or polymyositis (PM) a disease that causes muscle weakness
      without a rash. The study doctors want to evaluate whether ACTH Gel will improve the symptoms
      of this disease. This drug is approved by the Food and Drug Administration (FDA) for
      dermatomyositis (DM) and polymyositis (PM). ACTH gel has been an FDA-approved treatment for
      myositis since 1952, and in 2010 the FDA retained PM and DM as diseases approved for ACTH gel
      use.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Definite or probable polymyositis (PM) or dermatomyositis (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 to
             ensure non-PM patients are not enrolled. This step is necessary since there are
             well-known mimics of PM.

          -  Age ≥ 18 years.

          -  Active myositis as defined by baseline Manual Muscle Testing (MMT-8) no greater than
             125/150 and at least 2 additional CSM meeting the criteria stipulated below:

               1. Patient global with a minimum value of 2.0 cm on a 10 cm visual analog scale(VAS)

               2. Physician global with a minimum value of 2.0 cm on a 10 cm VAS scale

               3. Health Assessment Questionnaire (HAQ) disability index with a minimum value of
                  0.25

               4. Elevation of at least one of the muscle enzymes [which includes creatine kinase
                  (CK), aldolase, lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and
                  aspartate aminotransferase (AST)] at a minimum level of 1.3 x the upper limit of
                  normal.

               5. Global extramuscular disease activity score with a minimum value of 1.0 cm on a
                  10 cm VAS scale [this measure is the physician's composite evaluation and is
                  based on assessments of activity scores on the constitutional, cutaneous,
                  skeletal, gastrointestinal, pulmonary and cardiac scales of the Myositis Disease
                  Activity Assessment Tool (MDAAT)].

          -  To ensure that we can enroll active DM patients with a severe rash who may not meet
             the MMT-8 criterion noted above, we propose additional enrollment criteria such that
             the International Myositis Assessment and Clinical Studies (IMACS) definition of
             improvement (DOI) can potentially be met:

               1. Cutaneous VAS score on MDAAT > 3 cm on a 10 cm VAS scale, and

               2. At least 3 of the above 5 (a through e under 4.) criteria.

          -  Refractory myositis is defined by active disease despite an adequate glucocorticoid
             trial (> 2 months of usual glucocorticoid therapy or intolerance to such therapy)
             and/or ≥ 1 conventional immunosuppressive agent (e.g. methotrexate, azathioprine,
             tacrolimus, cyclosporine, mycophenolate mofetil, IVIG, anti-TNF or rituximab) for a
             reasonable dose and duration (> 3 months or intolerance to therapy). It is recommended
             to enroll refractory patients failing (or intolerant to) both glucocorticoids and at
             least 1 conventional immunosuppressive agent.

          -  If the enrolling physician is planning to continue current immunosuppressive agents or
             glucocorticoids as concomitant therapy with Acthar gel during the trial, then patient
             must be on a stable glucocorticoid and/or immunosuppressive dose 2 weeks prior to
             visit 1. The patient should have been on that immunosuppressive medication for at
             least 8 weeks (and at least 4 weeks for glucocorticoids) prior to visit 1.

          -  If the enrolling physician is planning to discontinue current immunosuppressive agent
             or glucocorticoids, then following wash out period is required prior to visit 1.

          -  If previous concomitant medications were discontinued, the following wash out periods
             are required prior to Visit 1

          -  Methotrexate -4 weeks

          -  Other IS agent (e.g. azathioprine, cyclosporine, tacrolimus, leflunomide,
             mycophenolate mofetil) - 4 weeks

          -  IVIg or cyclophosphamide - 2 months

          -  rituximab -6 months

          -  infliximab or adalimumab -8 weeks

          -  glucocorticoids - 2 weeks

          -  etanercept -2 weeks

          -  anakinra -1 week

        Exclusion Criteria:

          -  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.

          -  Hypersensitivity to Acthar

          -  Severe cardiac or pulmonary involvement

          -  Severe muscle damage defined as a baseline global muscle damage score on the MDI
             (Myositis Damage Index) of ≥ 5 cm on a 10 cm VAS.

          -  Patients with malignancy within 3 years of screening (except basal cell cancer or
             squamous cell cancer of skin).

          -  Uncontrolled diabetes, hepatic or renal disease.

          -  Ongoing active or chronic infections.

          -  Pregnant or lactating females.

          -  For any medical or physical or socio-psychological reasons that PI feels would not
             allow the subject to complete the study.