Back to results

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

Primary Purpose

Dermatomyositis, Juvenile Dermatomyositis

Status

Completed

Phase

Phase 4

Locations

United States

Study Type

Interventional

Intervention

H.P. Acthar Gel

About this trial

This is an interventional treatment trial for Dermatomyositis focused on measuring dermatomyositis, Acthar gel

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Cleveland Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

H.P Acthar Gel

Arm Description

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

Outcomes

Primary Outcome Measures

Change from baseline in cutaneous manifestations of dermatomyositis at 1, 3, and 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

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

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

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

Statistically significant change between baseline and 1, 3, and 6 months in patient assessed Dermatology Life Quality Index (DLQI) scores at these timepoints.

Full Information

NCT ID

NCT02245841

First Posted

September 11, 2014

Last Updated

September 10, 2021

Sponsor

The Cleveland Clinic

Collaborators

Mallinckrodt

1. Study Identification

Unique Protocol Identification Number

NCT02245841

Brief Title

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

Acronym

Acthar Gel

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2021

Overall Recruitment Status

Completed

Study Start Date

June 15, 2015 (Actual)

Primary Completion Date

July 14, 2021 (Actual)

Study Completion Date

July 14, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

The Cleveland Clinic

Collaborators

Mallinckrodt

4. Oversight

Data Monitoring Committee

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dermatomyositis, Juvenile Dermatomyositis

Keywords

dermatomyositis, Acthar gel

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

H.P Acthar Gel

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

H.P. Acthar Gel

Other Intervention Name(s)

Acthar Gel

Intervention Description

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

Primary Outcome Measure Information:

Title

Change from baseline in cutaneous manifestations of dermatomyositis at 1, 3, and 6 months

Description

Time Frame

6 months

Title

Change from baseline in cutaneous manifestations of dermatomyositis at 1, 3, and 6 months

Description

Change between baseline and 1, 3, and 6 months in cutaneous manifestations of dermatomyositis based on Physician's Global Assessment scores at these timepoints.

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Change from baseline in patient assessment of dermatomyositis at 1, 3, and 6 months

Description

Statistically significant change between baseline and 1, 3, and 6 months in patient assessed "Global Patient Score" at these timepoints.

Time Frame

6 months

Title

Change from baseline in patient assessment of dermatomyositis at 1, 3, and 6 months

Description

Statistically significant change between baseline and 1, 3, and 6 months in patient assessed "Global Itch Score" at these timepoints.

Time Frame

6 months

Title

Change from baseline in patient assessment of dermatomyositis at 1, 3, and 6 months

Description

Statistically significant change between baseline and 1, 3, and 6 months in patient assessed Dermatology Life Quality Index (DLQI) scores at these timepoints.

Time Frame

6 months

Other Pre-specified Outcome Measures:

Title

Number of patients with adverse effects.

Description

Safety and tolerability of H.P. Acthar gel based on frequency and types of adverse effects.

Time Frame

6 months

Title

Number of patients with change in dose of systemic corticosteroids and/or steroid-sparing immunosuppressive agents

Description

Median/mean change in dose of systemic corticosteroids and/or steroid-sparing immunosuppressive agents from initiation to completion of study

Time Frame

6 months

Title

Number of patients with change in HbA1c

Description

Median/mean change in HbA1c

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Anthony P Fernandez, MD, PhD

Organizational Affiliation

The Cleveland Clinic

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cleveland Clinic

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44195

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Citations:

PubMed Identifier

22787386

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.

Results Reference

background

PubMed Identifier

1090839

Citation

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.

Results Reference

background

PubMed Identifier

1089199

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.

Results Reference

background

PubMed Identifier

16546580

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.

Results Reference

background

PubMed Identifier

8423381

Citation

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

Results Reference

background

PubMed Identifier

18616782

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.

Results Reference

background

PubMed Identifier

19863510

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.

Results Reference

background

Learn more about this trial

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

We'll reach out to this number within 24 hrs