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

Dermatomyositis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02245841
• Other study ID #: 14-1015
• Status: Completed
• Phase: Phase 4
• Start date: June 15, 2015
• Est. completion date: July 14, 2021

Study information

• Verified date: June 2024
• Source: The Cleveland Clinic
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: July 14, 2021
• Est. primary completion date: July 14, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Dermatomyositis
• Juvenile Dermatomyositis

Intervention

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

Locations

Country	Name	City	State
United States	Cleveland Clinic	Cleveland	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
The Cleveland Clinic	Mallinckrodt

Country where clinical trial is conducted

United States, 

References & Publications (7)

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. — View Citation

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. — View Citation

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

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. — View Citation

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. — View Citation

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. Erratum In: Drug Des Devel Ther. 2012;6:163. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Patients With Adverse Effects.	Safety and tolerability of H.P. Acthar gel based on frequency and types of adverse effects.	6 months
Other	Number of Patients With Change in Dose of Systemic Corticosteroids and/or Steroid-sparing Immunosuppressive Agents	Median/mean change in dose of systemic corticosteroids and/or steroid-sparing immunosuppressive agents from initiation to completion of study	6 months
Other	Number of Patients With Change in HbA1c	Median/mean change in HbA1c	6 months
Primary	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
Primary	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	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".	6 months
Secondary	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.	6 months
Secondary	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.	6 months