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CompletedNCT02245841Updated Jun 11, 2024Results posted

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

A Phase 4 interventional study of H.P. Acthar Gel in Dermatomyositis and Juvenile Dermatomyositis, sponsored by The Cleveland Clinic. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-06-11.

Sponsored by The Cleveland Clinic · Phase 4, Interventional, and Treatment

Phase
Phase 4
Study type
Interventional
Enrollment
19
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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.

Read the 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.

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Conditions studied

  • Dermatomyositis
  • Juvenile Dermatomyositis

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Keywords

  • dermatomyositis
  • Acthar gel
03

In context

Dermatomyositis

171 studies on the registry are indexed under Dermatomyositis; 61 are open to participants now.

This study's enrollment of 19 is below the median of 31 across 125 interventional studies indexed under Dermatomyositis.

Browse Dermatomyositis studies →

Lead sponsor

The Cleveland Clinic is the lead sponsor of 818 studies on the registry; 118 are open to participants now.

Of its 89 completed or terminated interventional studies of FDA-regulated products, 72 (81%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
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Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
19 participants (actual)

Study arms

  • 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

Interventions

  • DrugH.P. Acthar Gel

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

    Also known as: Acthar Gel

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What researchers measure

Primary outcomes

  1. 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.

    Time frame: 6 months

  2. 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.

    Time frame: 6 months

Secondary outcomes

  1. 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".

    Time frame: 6 months

  2. 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.

    Time frame: 6 months

  3. 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.

    Time frame: 6 months

Other outcomes

  1. Number of Patients With Adverse Effects.

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

    Time frame: 6 months

  2. 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

    Time frame: 6 months

  3. Number of Patients With Change in HbA1c

    Median/mean change in HbA1c

    Time frame: 6 months

07

Results

Posted Jun 11, 2024

Participant flow

Participant flow — Overall Study
MilestoneH.P Acthar Gel
Started19
Completed15
Not completed4

Outcome measures

PrimaryChange 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.

Time frame:
6 months
Reported as:
Median · units on a scale
Change From Baseline in Cutaneous Dermatomyositis at 6 Months
units on a scaleH.P Acthar Gel
Change From Baseline in Cutaneous Dermatomyositis at 6 Months-9 (-18 to -5)
Statistical analysis
  • H.P Acthar Gel · Wilcoxon signed rank test · p = < .001
PrimaryChange 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.

Time frame:
6 months
Reported as:
Median · units on a scale
Change in Physician's Global Assessment (PGA) Visual Acuity Score From Baseline to 6 Months
units on a scaleH.P Acthar Gel
Change in Physician's Global Assessment (PGA) Visual Acuity Score From Baseline to 6 Months-1.9 (-2.5 to -1.2)
Statistical analysis
  • H.P Acthar Gel · Wilcoxon signed rank test · p = <0.001
SecondaryChange 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".

Time frame:
6 months
Reported as:
Median · units on a scale
Change From Baseline in Patient Assessment of Dermatomyositis at 6 Months.
units on a scaleH.P Acthar Gel
Change From Baseline in Patient Assessment of Dermatomyositis at 6 Months.2 (.5 to 6)
Statistical analysis
  • H.P Acthar Gel · Wilcoxon signed rank test · p = 0.002
SecondaryChange 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.

Time frame:
6 months
Reported as:
Median · units on a scale
Change From Baseline in Patient Global Itch Score of Dermatomyositis at 6 Months
units on a scaleH.P Acthar Gel
Change From Baseline in Patient Global Itch Score of Dermatomyositis at 6 Months-1 (-5 to 1)
Statistical analysis
  • H.P Acthar Gel · Wilcoxon signed rank test · p = 0.050
SecondaryChange 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.

Time frame:
6 months
Reported as:
Median · units on a scale
Change From Baseline in Patient Assessment of DLQI Dermatomyositis at 6 Months
units on a scaleH.P Acthar Gel
Change From Baseline in Patient Assessment of DLQI Dermatomyositis at 6 Months-5 (-11 to -4)
Statistical analysis
  • H.P Acthar Gel · Wilcoxon signed rank test · p = <0.001
Other pre-specifiedNumber of Patients With Adverse Effects.

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

Time frame:
6 months

Results for this outcome have not been posted.

Other pre-specifiedNumber 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

Time frame:
6 months

Results for this outcome have not been posted.

Other pre-specifiedNumber of Patients With Change in HbA1c

Median/mean change in HbA1c

Time frame:
6 months

Results for this outcome have not been posted.

Adverse events

Collected over 6 Months. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
H.P Acthar Gel0/19 (0%)0/19 (0%)11/19 (57.9%)
Most frequent other events
Most frequent other events
EventH.P Acthar Gel
Weight GainGeneral disorders11/19
Difficulty SleepingGeneral disorders6/19

Baseline characteristics

Age, Continuous
Age, Continuous(years)H.P Acthar Gel
Mean56 (35 to 70)
Sex: Female, Male
Sex: Female, Male(Participants)H.P Acthar Gel
Female14
Male5
Race and Ethnicity Not Collected
Race and Ethnicity Not Collected(Participants)H.P Acthar Gel
Region of Enrollment
Region of Enrollment(participants)H.P Acthar Gel
United States19
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI-A)
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI-A)(units on a scale)H.P Acthar Gel
Median19 (14 to 33)
08

Study locations

1 site
  • Cleveland Clinic
    Cleveland, Ohio 44195, United States
09

References and documents

Publications

  • 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. PubMed 22787386 ↗
  • 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. PubMed 1090839 ↗
  • 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. PubMed 1089199 ↗
  • 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. PubMed 16546580 ↗
  • Euwer RL, Sontheimer RD. Amyopathic dermatomyositis: a review. J Invest Dermatol. 1993 Jan;100(1):124S-127S. doi: 10.1111/1523-1747.ep12356896. PubMed 8423381 ↗
  • 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. PubMed 18616782 ↗
  • 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. PubMed 19863510 ↗

Study documents

  • Protocol and statistical analysis plan · Jul 30, 2014

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Limited IPD will be shared within the publication.

Supporting information: Study protocol, Sap

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 11, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT02245841
Lead sponsor
The Cleveland Clinic
Collaborators
Mallinckrodt
Responsible party
Anthony Fernandez, MD, PhD (Md, PhD, The Cleveland Clinic) — Principal investigator
First posted
Sep 22, 2014
Start date
Jun 15, 2015
Primary completion
Jul 14, 2021
Completion
Jul 14, 2021
Results posted
Jun 11, 2024
Last update
Jun 11, 2024

Study contacts

Anthony P Fernandez, MD, PhD
principal investigator · The Cleveland Clinic

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.

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