ACTH as A Re-emerging theRapy for Uveitis (The ACTHAR Study)

What Was Tested

This registered phase 2 trial tested ACTH (Corticotropin).

The study aims to evaluate the potential role of ACTH gel in the management of non-infectious uveitis.

It had two arms (groups): Group 1 (experimental) and Group 2 (experimental).

The registry classes the interventions as drug.

More on the Plan

Uveitis is a term used to describe a group of inflammatory disorders involving the uveal tract (iris, ciliary body and choroid).

Acthar Gel is a highly purified sterile preparation of the adrenocorticotropic hormone (ACTH) gelatin to provide a prolonged release after intramuscular or subcutaneous (SC) injection.

ACTH is part of a group of molecules called melanocortins (MC) (ACTH, α, β, γ-MSH) that are produced, in human bodies, by breakdown of a common larger precursor called proopiomelanocortin (POMC).

A very well-known anti-inflammatory mechanism of action of ACTH is the production glucocorticoids by stimulating the adrenal glands.

Who Took Part

Enrollment: 28 actual participants took part.

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.

The full record lists 3 inclusion and 37 exclusion criteria; among the exclusions: diabetic retinopathy: proliferative diabetic retinopathy (pdr) or non-proliferative diabetic retinopathy (npdr) that compromise the vision; age-related macular degeneration;; myopic degeneration with active subfoveal choroidal neovascularization.

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.

Everyone knew when ACTH (Corticotropin) was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

It started in 2017-11-10 with completion listed for 2020-04-03.

It ran at 7 sites in United States (7).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Incidence of ocular adverse events (AEs) (time frame: Month 6). Incidence of systemic adverse events (AEs) (time frame: Month 6).

It also tracked 16 secondary outcomes, for example Mean change from Baseline (BL) in vitreous haze (VH), Mean change from BL in graded anterior chamber cells, Mean change from BL in Best Corrected Visual Acuity (BCVA), Mean change in degree of inflammation, Mean change in leakage of disc and Change from Baseline in macular thickness.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of ACTH (Corticotropin) in 28 people.

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

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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