Acthar in Treatment of Refractory Dermatomyositis and Polymyositis

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01906372
• Date of registration: 05/07/2013
• Prospective Registration: Yes
• Primary sponsor: Rohit Aggarwal, MD
• Public title: Acthar in Treatment of Refractory Dermatomyositis and Polymyositis
• Scientific title: Open Label Proof of Concept Study to Evaluate Efficacy and Safety of Adrenocorticotropic Hormone Gel in Refractory Dermatomyositis or Polymyositis
• Date of first enrolment: September 2013
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01906372
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Rohit Aggarwal, MD
• Affiliation: University of Pittsburgh, Division of Rheumatology

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Dermatomyositis

Polymyositis

Intervention(s)

Drug: Adrenocorticotropic Hormone Gel

Primary Outcome(s)

Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI). [Time Frame: Primary end point: IMACS preliminary definition of improvement (DOI)]

Secondary Outcome(s)

Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients. [Time Frame: Steroid sparing effect and safety and tolerability at 24 weeks compared to baseline]

Secondary ID(s)

PRO13050507

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Mallinckrodt

Results

• Results available: Yes
• Date Posted: 02/08/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01906372