ACTHAR GEL for Sarcoidosis-Associated Calcium Dysregulation: An Open-label Pilot Study

What Was Tested

The trial (phase 2, phase 3) is built around ACTH (Corticotropin).

ACTHAR Gel has activity in sarcoidosis associated hypercalciuria and calcium dysregulation.

It had 1 arm (groups): Sarcoidosis related Calcium Dysregulation (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 lung, it may affect any organ.

Although corticosteroids are recognized as the drug of choice for sarcoidosis.

ACTH(adrenocorticotropic hormone) is the only drug that is FDA-approved for this disorder.

Who Took Part

The registry lists 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: Age greater than 18 years old. Able to understand English to the point of comprehending the informed consent form. Biopsy proven sarcoidosis. Historical evidence that the patient's hypercalciuria/hypercalcemia is related to sarcoidosis. This should include a serum parathyroid hormone (PTH) level which is not elevated.

The full record lists 5 inclusion and 7 exclusion criteria; among the exclusions: A change in anti-sarcoidosis medications within 3 months of study entry; A history of hyperparathyroidism or another non-sarcoidosis cause of hypercalcemia/hypercalciuria; A history of Cushing's disease.

How the Study Was Run

The set-up was single-group and open-label.

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

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2015-02 with completion listed for 2015-06.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 9 secondary outcomes, for example Change in serum calcium during 12 week ACTHAR GEL treatment, Change in 1,25 di-hydroxy Vitamin D during 12 week ACTHAR GEL treatment, Change in patient global VAS during 12 week ACTHAR GEL treatment, Change in physician global VAS during 12 week ACTHAR GEL treatment, Change in urinary symptoms during 12 week ACTHAR GEL treatment and Change in Short Form-36 during 12 week ACTHAR GEL treatment.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3 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.