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Efficacy and Safety of H.P. Acthar Gel for the Treatment of Refractory Cutaneous Manifestations of Dermatomyositis (Acthar Gel)

This study is currently recruiting participants.
Verified December 2016 by Anthony Fernandez, MD, PhD, The Cleveland Clinic
Sponsor:
ClinicalTrials.gov Identifier:
NCT02245841
First Posted: September 22, 2014
Last Update Posted: January 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Mallinckrodt
Information provided by (Responsible Party):
Anthony Fernandez, MD, PhD, The Cleveland Clinic
  Purpose
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.

Condition Intervention Phase
Dermatomyositis Juvenile Dermatomyositis Drug: H.P. Acthar Gel Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy and Safety of H.P. Acthar Gel for the Treatment of Refractory Cutaneous Manifestations of Dermatomyositis

Resource links provided by NLM:


Further study details as provided by Anthony Fernandez, MD, PhD, The Cleveland Clinic:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Other Outcome Measures:
  • Number of patients with adverse effects. [ Time Frame: 6 months ]
    Safety and tolerability of H.P. Acthar gel based on frequency and types of adverse effects.

  • Number of patients with change in dose of systemic corticosteroids and/or steroid-sparing immunosuppressive agents [ Time Frame: 6 months ]
    Median/mean change in dose of systemic corticosteroids and/or steroid-sparing immunosuppressive agents from initiation to completion of study

  • Number of patients with change in HbA1c [ Time Frame: 6 months ]
    Median/mean change in HbA1c


Estimated Enrollment: 15
Study Start Date: June 2015
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: H.P Acthar Gel
80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Drug: H.P. Acthar Gel
80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Other Name: Acthar Gel

Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must be 18 years of age or older with refractory cutaneous symptoms related to either classic dermatomyositis (CD), juvenile dermatomyositis (JD), or amyopathic dermatomyositis(AD). Diagnosis will be based on either Bohan and Peter criteria (CD and JD) or Sontheimer's criteria (AD)
  • Must have had a skin biopsy with histologic features consistent with dermatomyositis and current cutaneous manifestations consistent with dermatomyositis.
  • Although not mandatory, patients with evidence of current or previous active myositis will be eligible for enrollment. Patients will be considered to have refractory disease if cutaneous manifestations exist despite treatment with steroids and at least one steroid-sparing systemic treatment commonly found to be useful in patients with dermatomyositis. These may include azathioprine, cyclosporine, mycophenolate mofetil, IVIG, methotrexate, cyclophosphamide, chlorambucil, sirolimus, adalimumab, infliximab and rituximab.
  • Use of topical medications and sunscreen currently and in past will be noted but not weighed for assessment of refractory cutaneous disease.

Exclusion Criteria:

  • Patients with dermatomyositis who have minimal-to-no active cutaneous features (focal involvement with less than 1% total body surface area involved or minimal modified CDASI activity score).
  • Patients whose cutaneous findings are not consistent with dermatomyositis and/or have previous biopsy results suggestive of an alternative diagnosis
  • Patients with inflammatory myositis other than dermatomyositis, such as polymyositis or inclusion body myositis.
  • Patients with malignancy-associated dermatomyositis
  • Patients with clear features of an overlap myositis
  • Patients younger than 18 years old
  • Patients with acutely active or chronic infections.
  • Patients with uncontrolled diabetes, hypertension, cardiovascular, hepatic, or renal disease
  • Pregnant or lactating females.
  • Patients with any medical condition that is felt by the primary investigator to place the patient at unreasonable risk for adverse effects during treatment with H.P. Acthar.
  • Hypersensitivity to H.P. Acthar, any of its components (allergy to pig-derived proteins)
  • Patients with osteoporosis
  • Patients who have had surgery within 8 weeks of screening
  • Patients with a history of or current gastric ulcers
  • Patients taking daily doses of systemic corticosteroids greater than the equivalent of 40mg prednisone.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02245841


Contacts
Contact: Anthony Fernandez, MD, PhD 216 445 8776 fernana6@ccf.org
Contact: Lisa Rittwage, BSN, RN 216 444-4659 rittwal@ccf.org

Locations
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Principal Investigator: Anthony Fernandez, MD, PhD         
Sponsors and Collaborators
The Cleveland Clinic
Mallinckrodt
Investigators
Principal Investigator: Anthony P Fernandez, MD, PhD The Cleveland Clinic
  More Information

Publications:

Responsible Party: Anthony Fernandez, MD, PhD, Md, PhD, The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT02245841     History of Changes
Other Study ID Numbers: 14-1015
First Submitted: September 11, 2014
First Posted: September 22, 2014
Last Update Posted: January 2, 2017
Last Verified: December 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Anthony Fernandez, MD, PhD, The Cleveland Clinic:
dermatomyositis
Acthar gel

Additional relevant MeSH terms:
Dermatomyositis
Polymyositis
Myositis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs