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

NCT02245841 | Completed Phase 4 Results

• 1 other identifier: 14-1015
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

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.

Conditions

Dermatomyositis; Juvenile Dermatomyositis

Keywords

dermatomyositis; Acthar gel

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  6 months

Secondary Outcomes (3)

• Change From Baseline in Patient Assessment of Dermatomyositis at 6 Months.

  6 months

• Change From Baseline in Patient Global Itch Score of Dermatomyositis at 6 Months

  6 months

• Change From Baseline in Patient Assessment of DLQI Dermatomyositis at 6 Months

  6 months

Other Outcomes (3)

• Number of Patients With Adverse Effects.

  6 months

• Number of Patients With Change in Dose of Systemic Corticosteroids and/or Steroid-sparing Immunosuppressive Agents

  6 months

• Number of Patients With Change in HbA1c

  6 months

Study Arms (1)

H.P Acthar Gel

EXPERIMENTAL

80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks

Drug: H.P. Acthar Gel

Interventions

H.P. Acthar Gel DRUG

80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks

Also known as: Acthar Gel

H.P Acthar Gel

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 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.

Sponsors & Collaborators

• The Cleveland Clinic: lead
• Mallinckrodt: collaborator

Study Sites (1)

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Related Publications (7)

• Levine T. Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series. Drug Des Devel Ther. 2012;6:133-9. doi: 10.2147/DDDT.S33110. Epub 2012 Jun 11.

  PMID: 22787386 BACKGROUND

• Bohan A, Peter JB. Polymyositis and dermatomyositis (first of two parts). N Engl J Med. 1975 Feb 13;292(7):344-7. doi: 10.1056/NEJM197502132920706. No abstract available.

  PMID: 1090839 BACKGROUND

• Bohan A, Peter JB. Polymyositis and dermatomyositis (second of two parts). N Engl J Med. 1975 Feb 20;292(8):403-7. doi: 10.1056/NEJM197502202920807. No abstract available.

  PMID: 1089199 BACKGROUND

• Gerami P, Schope JM, McDonald L, Walling HW, Sontheimer RD. A systematic review of adult-onset clinically amyopathic dermatomyositis (dermatomyositis sine myositis): a missing link within the spectrum of the idiopathic inflammatory myopathies. J Am Acad Dermatol. 2006 Apr;54(4):597-613. doi: 10.1016/j.jaad.2005.10.041. Epub 2006 Jan 23.

  PMID: 16546580 BACKGROUND

• Euwer RL, Sontheimer RD. Amyopathic dermatomyositis: a review. J Invest Dermatol. 1993 Jan;100(1):124S-127S. doi: 10.1111/1523-1747.ep12356896.

  PMID: 8423381 BACKGROUND

• Klein RQ, Bangert CA, Costner M, Connolly MK, Tanikawa A, Okawa J, Rose M, Fakharzadeh SS, Fiorentino D, Lee LA, Sontheimer RD, Taylor L, Troxel AB, Werth VP. Comparison of the reliability and validity of outcome instruments for cutaneous dermatomyositis. Br J Dermatol. 2008 Sep;159(4):887-94. doi: 10.1111/j.1365-2133.2008.08711.x. Epub 2008 Jul 4.

  PMID: 18616782 BACKGROUND

• Yassaee M, Fiorentino D, Okawa J, Taylor L, Coley C, Troxel AB, Werth VP. Modification of the cutaneous dermatomyositis disease area and severity index, an outcome instrument. Br J Dermatol. 2010 Mar;162(3):669-73. doi: 10.1111/j.1365-2133.2009.09521.x. Epub 2009 Oct 26.

  PMID: 19863510 BACKGROUND

MeSH Terms

Conditions

Dermatomyositis

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Results Point of Contact

• Title: Anthony Fernandez, MD, PhD
• Organization: Cleveland Clinic

fernana6@ccf.org

216-445-8776

Study Officials

• Anthony P Fernandez, MD, PhD

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Md, PhD

Study Record Dates

• First Submitted: September 11, 2014
• First Posted: September 22, 2014
• Study Start: June 15, 2015
• Primary Completion: July 14, 2021
• Study Completion: July 14, 2021
• Last Updated: June 11, 2024
• Results First Posted: June 11, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: CDSAI-A scores and adverse events at each study assessment point will be included and available in the publication indefinitely.
• Access Criteria: Available to all.

Locations

US (1)