Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome

What Was Tested

In this phase 3 trial, researchers set out to test ACTH (Corticotropin).

In childhood nephrotic syndrome, the kidneys leak protein, causing body swelling and a variety of possible complications such as infection, blood clots, and kidney failure.

The first-line treatment for nephrotic syndrome is corticosteroids.

It had three arms (groups): Adrenocorticotropic hormone (ACTH) (active comparator), No treatment (no intervention) and Rescue therapy (active comparator).

There is an option for the patient in no-treatment arm to elect to be placed in the active treatment arm of the trial (rescue therapy).

The registry classes the interventions as drug.

More on the Plan

Primary end-points: The primary end-point related to Hypothesis 1 is the proportion of patients in each arm with a relapse during the initial 6 months of treatment.

The primary end-point related to Hypothesis 2 is the proportion of relapse-free patients during the first 6 months and second 6 months of treatment with ACTH.

This will include patients initially randomized to ACTH and patients who receive ACTH as rescue therapy following their initial relapse in patients randomized to no treatment.

The primary end-point related to Hypothesis 3 is the proportion of patients who have relapses in the 6 months following completion of one year of ACTH.

Who Took Part

Enrollment: 31 actual participants took part.

It was open to people of any sex aged from 2 years up to 20 years.

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

Examples of who could join: Age \>1 year at onset of nephrotic syndrome. Age 2-20 years at time of randomization. Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome).

The full record lists 6 inclusion and 14 exclusion criteria; among the exclusions: prior treatment with acth; cyclophosphamide or rituximab within the last 4 months; lactation, pregnancy, or refusal of birth control in females with child-bearing potential.

How the Study Was Run

The set-up was randomized, parallel-group and open-label.

The participants were allocated by chance, so the ACTH (Corticotropin) group and the comparison group should start out alike.

The ACTH (Corticotropin) group and the comparison group ran side by side rather than switching.

Nothing was hidden in this open-label ACTH (Corticotropin) study: the participants and staff both knew the treatment.

It started in 2014-05 with completion listed for 2018-01.

It ran at 16 sites in United States (16).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Number of Participants Experienced a Relapse of Nephrotic Syndrome (time frame: 6 months).

It also tracked 2 secondary outcomes, for example Number of Adverse Events.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of ACTH (Corticotropin) in 31 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.