ACTHAR Gel for Cutaneous Sarcoidosis: An Open Label Trial

Albany Medical College

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Sarcoidosis; Cutaneous Sarcoidosis

Treatments: Drug: ACTHAR Gel 80 units twice weekly.; Drug: ACTHAR Gel 40 units twice weekly

Study type: Interventional

Funder types: Other

Identifiers

NCT02348905

AMCMAJCUT2014

Details and patient eligibility

About

ACTHAR gel has efficacy in the treatment of cutaneous sarcoidosis

Full 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.

Enrollment: 10 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

10 participants in 2 patient groups

ACTHAR Gel 40 units twice weekly

Experimental group

Description:

ACTHAR Gel at a dose of 40 units twice weekly sub cutaneous injections between Baseline and week 12.

Treatment:

Drug: ACTHAR Gel 40 units twice weekly

ACTHAR Gel 80 units twice weekly

Experimental group

Description:

ACTHAR gel at a dose of 80 units twice weekly sub cutaneous injections between Baseline and week 12.

Treatment:

Drug: ACTHAR Gel 80 units twice weekly.