ACTHAR Gel for Cutaneous Sarcoidosis: An Open Label Trial

What Was Tested

This registered phase 2, phase 3 trial tested ACTH (Corticotropin).

It had two arms (groups): one (experimental) and one (experimental).

The registry classes the interventions as drug.

More on the Plan

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.

Presently, corticosteroids as considered the drug of choice for the treatment of cutaneous sarcoidosis.

Who Took Part

Enrollment: 10 planned participants were the target.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Patients must have biopsy-proven sarcoidosis. Both lesions must have a SASI induration score of \> 1 and a SASI induration + erythema score of \> 2. If a subject has only lupus pernio facial lesions, one needs to be at least 0.5 cm in diameter.

The full record lists 5 inclusion and 6 exclusion criteria; among the exclusions: previous toxic or allergic reaction to acthar gel; the presence of another skin condition in addition to sarcoidosis that would interfere with the assessment of the sarcoidosis skin lesions; uncontrolled hypertension.

How the Study Was Run

The set-up was randomized, parallel-group and open-label.

Random assignment meant chance, not the researchers, decided who got ACTH (Corticotropin) and who got the comparison, which limits bias.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Being open-label, the ACTH (Corticotropin) study did not mask who received what.

It started in 2015-03 with completion listed for 2015-07.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): The change in SASI induration & erythema score (time frame: Between week 0 and week 12).

It also tracked 4 secondary outcomes, for example Photographic change, SAT skin module, DLQI and Extent of granulomatous inflammation in the biopsy lesion.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of ACTH (Corticotropin) in 10 people.

Its size is small, so on its own it can only give an early look.

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the ACTH (Corticotropin) compound page, all human trials.