ACTHAR Therapy for Central Nervous System Sarcoidosis

What Was Tested

This registered phase 4 trial tested ACTH (Corticotropin).

There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in severe Central Nervous System (CNS) sarcoidosis.

The registry classes the interventions as drug.

More on the Plan

Central nervous system (CNS) involvement is one of the most severe manifestations of sarcoidosis.

Treatment of moderate and severe CNS sarcoidosis typically involves a combination of corticosteroids and cytotoxic agents such as methotrexate (2).

ACTHAR is a 39-amino acid peptide natural form of adrenocorticotropin hormone (ACTH) that was initially approved in 1952 by the FDA.

It has since been approved for 19 indications including respiratory sarcoidosis, multiple sclerosis, and infantile spasms.

Who Took Part

It was open to people of any sex.

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

Examples of who could join: Patient with sarcoidosis as defined by ATS/ERS/WASOG (American Thoracic Society/European Thoracic Society/World Association for Sarcoidosis and Other Granulomatous Disorders). Stable baseline immunosuppressive medications. Moderate to severe disease as defined by at least one of the following criteria:. Cranial nerve palsy. Neurologic deficits related to intraparenchymal brain, spinal cord and/or cauda equina involvement.

The full record lists 8 inclusion and 7 exclusion criteria; among the exclusions: diagnosis of any underlying neurologic disorder that would potentially confound interpretation of the study results; evidence of current serious infection, or a history of chronic or recurring infections; allergies to pig-derived proteins.

How the Study Was Run

Design: 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 2019-02-01 with completion listed for 2020-11-02.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What This Means in Plain Language

Put simply, this is a phase 4 trial of ACTH (Corticotropin) in people.

It was withdrawn before enrolling anyone; no results are posted to the registry yet, so nothing here describes outcomes.

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