Treatment of CNS Sarcoidosis With H.P. Acthar Gel

What Was Tested

The trial (phase 4) is built around ACTH (Corticotropin).

The purpose of this study is to see if treatment with H.P.

It had 1 arm (groups): H.P. Acthar Gel (experimental).

Participants will receive active treatment with H.P. Acthar Gel.

The registry classes the interventions as drug.

More on the Plan

The hallmark of the disease is the development of chronic inflammation with formation of non-caseating granulomas that can involve the brain parenchyma and meninges and appear as contrast-enhancing mass lesions on magnetic resonance imaging.

The granulomas are primarily comprised of proinflammatory T cells (Th1 cells and Th17 cells) and macrophages which accumulate during the early stages of granuloma formation.

The inflammation that is generated by these cells is modulated by anti-inflammatory responses mediated by Th2 cells and regulatory T (Treg) cells that later appear and populate the outer regions of the granuloma.

The presence of Treg cells are of particular interest since these cell are also detected in high numbers in peripheral blood and the immune suppression that results may underlie the occurrence of anergy in patients with the disease.

Who Took Part

The registry lists 4 actual participants took part.

It was open to people of any sex.

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

The full record lists 3 inclusion and 0 exclusion criteria.

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 2016-05 with completion listed for 2020-11.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Karnofsky Performance Status (KPS) Score (time frame: 12 months (intention to treat)). Change in Total Number of New Lesions (time frame: 1 year).

It also tracked 5 secondary outcomes, for example Patient-Determined Disease Steps (PDDS), Montreal Cognitive Assessment (MoCA), Symbol-Digit Modalities Test (SDMT), Work Productivity and Activities Impairment -General Health (WPAI-GH) and Beck Depression Inventory.

What This Means in Plain Language

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

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

The trial is marked completed; results are posted on the registry, and this page does not restate them.

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