Dose-Finding Pilot Study of ACTH in Patients With Idiopathic Membranous Nephropathy

What Was Tested

This registered phase 1 trial tested ACTH (Corticotropin).

Acthar Gel) on the lipid profile and proteinuria in participants with MN.

ACTH or adrenocorticotrophin is a hormone produced by the pituitary gland (a gland at the base of your brain) that is involved in stimulating your adrenal glands to secrete a number of steroid products (e.g. cortisol, aldosterone, corticosterone, and others) that are important in keeping you alive.

It had two arms (groups): one (active comparator) and one (active comparator).

The registry classes the interventions as drug.

Who Took Part

20 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: Age \> 18 years. Patients need to be treated with an ACEI and/or ARB, for at least 3 months prior to ACTH treatment and have adequately controlled blood pressure. Proteinuria of \>4.0 on a 24-hour urine collection.

The full record lists 5 inclusion and 9 exclusion criteria; among the exclusions: age \<18 years; patient must be off glucocorticoid, calcineurin inhibitors (cyclosporin a, tacrolimus) or mycophenolic mofetil for \>1 month, and alkylating agents or rituximab for \>6 months; resistance to the following immunosuppressive routines e.g. steroids alone, calcineurin inhibitors plus or minus steroids, cytotoxic agents plus or minus steroids.

How the Study Was Run

It was run as a randomized, parallel-group and open-label study.

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.

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

It started in 2009-01 with completion listed for 2013-09.

It ran at 2 sites in United States (1) and Canada (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Change in proteinuria (time frame: baseline, 3 months). Change in LDL cholesterol, HDL cholesterol, and triglycerides (time frame: baseline, 3 months). Change in side effects/toxicity (time frame: baseline, 3 months).

It also tracked 2 secondary outcomes, for example Number of subjects with CR or PR and The effect of maximizing angiotensin II blockade on proteinuria.

What This Means in Plain Language

Put simply, this is a phase 1, randomized trial of ACTH (Corticotropin) in 20 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.