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

June 7, 2024 updated by: Anthony Fernandez, MD, PhD, The Cleveland Clinic
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

19

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • Acthar Gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Cutaneous Dermatomyositis at 6 Months
Time Frame: 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.
6 months
Change in Physician's Global Assessment (PGA) Visual Acuity Score From Baseline to 6 Months
Time Frame: 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.
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Patient Assessment of Dermatomyositis at 6 Months.
Time Frame: 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".
6 months
Change From Baseline in Patient Global Itch Score of Dermatomyositis at 6 Months
Time Frame: 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.
6 months
Change From Baseline in Patient Assessment of DLQI Dermatomyositis at 6 Months
Time Frame: 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.
6 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
6 months
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
6 months
Number of Patients With Change in HbA1c
Time Frame: 6 months
Median/mean change in HbA1c
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Cleveland Clinic

Collaborators

Mallinckrodt

Investigators

  • Principal Investigator: Anthony P Fernandez, MD, PhD, The Cleveland Clinic

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 15, 2015

Primary Completion (Actual)

July 14, 2021

Study Completion (Actual)

July 14, 2021

Study Registration Dates

First Submitted

September 11, 2014

First Submitted That Met QC Criteria

September 19, 2014

First Posted (Estimated)

September 22, 2014

Study Record Updates

Last Update Posted (Actual)

June 11, 2024

Last Update Submitted That Met QC Criteria

June 7, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 14-1015

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Limited IPD will be shared within the publication.

IPD Sharing Time Frame

CDSAI-A scores and adverse events at each study assessment point will be included and available in the publication indefinitely.

IPD Sharing Access Criteria

Available to all.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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