ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children

What Was Tested

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

The researchers plan to enroll 25 children between the ages of 2 to 21 years.

Children fulfilling strict inclusion criteria, whose parents agree to written informed consent after institutional IRB approval for the study, will be enrolled.

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

The registry classes the interventions as drug.

More on the Plan

Treatment Protocol: Patients who fulfill inclusion criteria and who agree to participate after signing informed consent and assent forms will be treated with the following protocol: Acthar Gel will be dosed by body surface area (BSA) using the Dubois Method.

Study drug will be provided free of cost to patients in the first 6 months only.

If Amlodipine is not covered by the participants insurance, then an alternate calcium channel blocker will be started at the clinical discretion of the PI/co-PI that is covered by insurance.

If the participant is not covered by any insurance the participant will have to pay out of pocket 2.

Who Took Part

It was open to people of any sex aged from 2 years up to 21 years.

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

Examples of who could join: Calculated GFR (eGFR) using modified Schwartz formula of \> 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL). A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study. Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy.

The full record lists 4 inclusion and 5 exclusion criteria; among the exclusions: patients with an inherited or genetic disorder presenting with nephrotic syndrome (eg: nphs 1 \& 2 defects, wt-1 mutations, α actinin 4 mutation, trp-6 mutation); presence of diabetes or severe (stage 2) uncontrolled hypertension; any metabolic condition that specifically precludes the use of acthar gel for treatment.

How the Study Was Run

The set-up was sequential and open-label.

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

It started in 2018-06 with completion listed for 2021-01.

Its stated primary purpose is diagnostic.

What Was Measured

The main thing measured (primary outcome): Remission of proteinuria (time frame: 18 months).

It also tracked 6 secondary outcomes, for example Blood pressure, Serum Creatinine, BMI, Serum Glucose, Serum Lipids and Quality of life measures obtained via survey.

What This Means in Plain Language

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

It was withdrawn before enrolling anyone; no results are posted to the registry yet, so nothing here describes outcomes.

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