This study will assess the safety and efficacy of H.P. Acthar gel for treating the cutaneous manifestations in patients with refractory classic dermatomyositis, juvenile dermatomyositis, and amyopathic dermatomyositis. Our hypothesis is that H.P. Acthar gel will be both safe and effective for such patients.
Adult and juvenile dermatomyositis (DM) are systemic immune-mediated inflammatory diseases most commonly affecting the skin and musculoskeletal system. Amyopathic dermatomyositis is a subtype of dermatomyositis that affects only the skin and lacks the characteristic muscle involvement. Treatment of these conditions, in particular the cutaneous manifestations, is challenging and currently no universally effective single treatment exists. Many patients have cutaneous manifestations that are refractory to numerous medications. H.P. Acthar gel (adrenocorticotropic hormone gel) received FDA approval for treatment of a variety of diseases, including dermatomyositis, in 1952. Despite this there is a paucity of clinical data concerning the efficacy of H.P. Acthar gel for treating dermatomyositis. Recently a small, retrospective case series describing significant improvement in both cutaneous and musculoskeletal symptoms in 5 patients with refractory dermatomyositis treated with H.P. Acthar gel was reported and has resulted in renewed interest in use of this medication in dermatomyositis patient (reference below). The proposed efficacy of H.P. Acthar gel has been attributed to its unique ability to induce production of endogenous cortisol, corticosterone, aldosterone, and to bind melanocortin receptors on lymphocytes and other cells to modulate immunologic responses.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
19
80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Cleveland Clinic
Cleveland, Ohio, United States
Change From Baseline in Cutaneous Dermatomyositis at 6 Months
Change between baseline at 6 months in modified CDASI-A (Cutaneous Dermatomyositis Disease Area and Severity Index) scores at these timepoints. The CDASI-A score ranges from 0 to 100 with higher scores reflecting more severe disease activity.
Time frame: 6 months
Change in Physician's Global Assessment (PGA) Visual Acuity Score From Baseline to 6 Months
Change from baseline to 6 months in Physician's Global Assessment (PGA) visual acuity score. Scores range from 0-10 with Higher scores reflecting severe disease activity.
Time frame: 6 months
Change From Baseline in Patient Assessment of Dermatomyositis at 6 Months.
Change between baseline and 6 months in patient assessed "Global Skin Score" at these timepoints. The Global Skin Score ranges from 0 to 10 with lower 0 representing "worst sign condition imaginable" and 10 representing "perfect health".
Time frame: 6 months
Change From Baseline in Patient Global Itch Score of Dermatomyositis at 6 Months
Change between baseline and 6 months in patient assessed "Global Itch Score" at these timepoints. The Global Itch score ranges from 0 to 10 with higher scores reflect more severe itching.
Time frame: 6 months
Change From Baseline in Patient Assessment of DLQI Dermatomyositis at 6 Months
Change between baseline and 6 months in patient assessed Dermatology Life Quality Index (DLQI) scores at these timepoints. The DLQI ranges from 0 to 30 with higher scores implying more significant impact on quality of life.
Time frame: 6 months
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