ACTHAR Gel for Cutaneous Sarcoidosis: An Open Label Trial

ACTHAR gel has efficacy in the treatment of cutaneous sarcoidosis

Study Overview

• Status: Unknown
• Conditions: Sarcoidosis; Cutaneous Sarcoidosis
• Intervention / Treatment: Drug: ACTHAR Gel 40 units twice weekly; Drug: ACTHAR Gel 80 units twice weekly.

Detailed Description

Sarcoidosis is a multisystem granulomatous disease of unknown cause. Although sarcoidosis most commonly affects the lung3, it may affect any organ. Although corticosteroids are recognized as the drug of choice for sarcoidosis, ACTH is the only drug that is FDA-approved for this disorder. However, there is limited data on the efficacy of ACTH for this condition.

Presently, corticosteroids as considered the drug of choice for the treatment of cutaneous sarcoidosis. However, ACTHER GEL not only has obvious anti-inflammatory effects by resulting in corticosteroid production, but it may also activate melanocortin receptors. The melanocortin system has powerful anti-inflammatory properties that may be beneficial in the treatment of cutaneous sarcoidosis.

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Haroon Chaudhry, MBBS; Phone Number: 518-262-1542; Email: chaudhh@mail.amc.edu
• Study Contact Backup: Name: Marc A. Judson, MD; Phone Number: 518-262-5196

Study Locations

• United States
  • New York
    • Albany, New York, United States, 12208
      • Albany Medical College
      • Contact: Haroon Chaudhry, MBBS; Phone Number: 518-262-1542; Email: chaudhh@mail.amc.edu
      • Principal Investigator: Marc A. Judson, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Patients must have biopsy-proven sarcoidosis.
2. a) Patients with active cutaneous sarcoidosis and two active lesions on stable therapy or no therapy for at least two months AND/OR b) Patients with active cutaneous sarcoidosis and lupus pernio lesions on stable therapy or no therapy for at least two months. A maximum of 5 patients may be enrolled fulfilling criterion b) above.
3. Both lesions must have a SASI induration score of > 1 and a SASI induration + erythema score of > 2.
4. If two lesions are present, one must be > 1cm in diameter and the subject must be willing to have it biopsied. The second lesion must be at least 0.5 cm in diameter.
5. If a subject has only lupus pernio facial lesions, one needs to be at least 0.5 cm in diameter.

Exclusion Criteria:

1. Previous toxic or allergic reaction to ACTHAR gel
2. The presence of another skin condition in addition to sarcoidosis that would interfere with the assessment of the sarcoidosis skin lesions.
3. Uncontrolled hypertension.
4. Uncontrolled diabetes.
5. Active infection.
6. A medical condition that, in the opinion of the investigator would place the subject at significant risk by administering ACTHAR gel.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ACTHAR Gel 40 units twice weekly; ACTHAR Gel at a dose of 40 units twice weekly sub cutaneous injections between Baseline and week 12.	Drug: ACTHAR Gel 40 units twice weekly; ACTHAR Gel (adrenocorticotropic hormone) 40 units twice weekly between Baseline and week 12.
Experimental: ACTHAR Gel 80 units twice weekly; ACTHAR gel at a dose of 80 units twice weekly sub cutaneous injections between Baseline and week 12.	Drug: ACTHAR Gel 80 units twice weekly.; ACTHAR Gel (adrenocorticotropic hormone) 80 units twice weekly between Baseline and week 12.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The change in SASI induration & erythema score.	Between week 0 and week 12.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Photographic change	Photographic change (improved, worse, no change as determined by investigators blinded to the time of the photographs)	Between week 0 and week 12.
SAT skin module		Between week 0 and week 12.
DLQI		Between week 0 and week 12.
Extent of granulomatous inflammation in the biopsy lesion	Extent of granulomatous inflammation in the biopsy lesion (as judged by pathologists blinded to the time of the biopsy).	Between week 0 and week 12.

Collaborators and Investigators

• Sponsor: Albany Medical College
• Investigators: Principal Investigator: Marc A Judson, MD, Albany Medical College

Study record dates

Study Major Dates

• Study Start: March 1, 2015
• Primary Completion (Anticipated): July 1, 2015
• Study Completion (Anticipated): January 1, 2016

Study Registration Dates

• First Submitted: June 20, 2014
• First Submitted That Met QC Criteria: January 23, 2015
• First Posted (Estimate): January 28, 2015

Study Record Updates

• Last Update Posted (Estimate): January 28, 2015
• Last Update Submitted That Met QC Criteria: January 23, 2015
• Last Verified: January 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Lymphoproliferative Disorders; Lymphatic Diseases; Sarcoidosis; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Adrenocorticotropic Hormone
• Other Study ID Numbers: AMCMAJCUT2014