Clinical Trial: Efficacy and Safety of H.P. Acthar Gel for the Treatment of Refractory Cutaneous Manifestations of Dermatomyositis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Efficacy and Safety of H.P. Acthar Gel for the Treatment of Refractory Cutaneous Manifestations of Dermatomyositis

Brief Summary: 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.

Detailed Summary:

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.

Sponsor: The Cleveland Clinic

Current Primary Outcome:

• Change from baseline in cutaneous manifestations of dermatomyositis at 1, 3, and 6 months [ Time Frame: 6 months ]
  Statistically significant change between baseline and 1, 3, and 6 months in cutaneous manifestations of dermatomyositis based on modified CDASI (modified Cutaneous Dermatomyositis Disease Area and Severity Index) scores at these timepoints.
• Change from baseline in cutaneous manifestations of dermatomyositis at 1, 3, and 6 months [ Time Frame: 6 months ]
  Change between baseline and 1, 3, and 6 months in cutaneous manifestations of dermatomyositis based on Physician's Global Assessment scores at these timepoints.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Change from baseline in patient assessment of dermatomyositis at 1, 3, and 6 months [ Time Frame: 6 months ]
  Statistically significant change between baseline and 1, 3, and 6 months in patient assessed "Global Patient Score" at these timepoints.
• Change from baseline in patient assessment of dermatomyositis at 1, 3, and 6 months [ Time Frame: 6 months ]
  Statistically significant change between baseline and 1, 3, and 6 months in patient assessed "Global Itch Score" at these timepoints.
• Change from baseline in patient assessment of dermatomyositis at 1, 3, and 6 months [ Time Frame: 6 months ]
  Statistically significant change between baseline and 1, 3, and 6 months in patient assessed Dermatology Life Quality Index (DLQI) scores at these timepoints.

Original Secondary Outcome: Same as current

Information By: The Cleveland Clinic

Dates:
Date Received: September 11, 2014
Date Started: June 2015
Date Completion: June 2018
Last Updated: December 30, 2016
Last Verified: December 2016