Acthar in Treatment of Refractory Dermatomyositis and Polymyositis

What Was Tested

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

This drug is approved by the Food and Drug Administration (FDA) for dermatomyositis (DM) and polymyositis (PM).

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

The registry classes the interventions as drug.

More on the Plan

There was a recent study published in the peer-review journal Drug Design, Development and Therapy on a retrospective case series evaluating Acthar in the treatment of PM and DM.

Acthar was administered to five patients who had previously failed multiple steroid and immunosuppressant treatment regimens.

The patients received injections of Acthar over the course of 12 weeks or more.

The paper, "Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series," was authored by Dr.

Who Took Part

Enrollment: 12 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.

Examples of who could join: Definite or probable polymyositis (PM) or dermatomyositis (DM) by Bohan and Peter criteria. Age ≥ 18 years. Active myositis as defined by baseline Manual Muscle Testing (MMT-8) no greater than 125/150 and at least 2 additional CSM meeting the criteria stipulated below:. Patient global with a minimum value of 2.0 cm on a 10 cm visual analog scale(VAS). Physician global with a minimum value of 2.0 cm on a 10 cm VAS scale.

The full record lists 24 inclusion and 9 exclusion criteria; among the exclusions: juvenile dm or pm, myositis in overlap with another connective tissue disease, cancer associated myositis, inclusion body myositis, or any other non immune-mediated myopathy; hypersensitivity to acthar; severe cardiac or pulmonary involvement.

How the Study Was Run

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

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.

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

It started in 2013-09 with completion listed for 2015-09.

It ran at 2 sites in United States (2).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 1 secondary outcome, for example Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients.

What This Means in Plain Language

Put simply, this is a phase 2 trial of ACTH (Corticotropin) in 12 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.