CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism

What Was Tested

In this phase 2 trial, researchers set out to test Glucagon.

It had three arms (groups): CSI-Glucagon (Double-Blind Phase - 2 days) (experimental), Placebo (Double-Blind Phase - 2 days) (placebo comparator) and CSI-Glucagon (Open-label Phase - Up to 28 days) (experimental).

Vehicle solution delivered as a 24-hour continuous subcutaneous infusion via a patch pump.

The registry classes the interventions as drug and other.

More on the Plan

CSI-Glucagon™ is expected to provide a better inpatient treatment option compared to the current standard of care.

The study will consist of three phases: 1.

After 48 hours of blinded treatment, all subjects will transition to open-label active treatment.

Open-label Treatment Phase: The third study period will involve use of CSI-Glucagon™ to manage blood glucose with minimal GIR for up to 28 days of cumulative exposure.

Who Took Part

Enrollment: 5 actual participants took part.

It was open to people of any sex aged up to 12 months.

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

Examples of who could join: Diagnosed with hyperinsulinism:. May be on diazoxide and/or octreotide, but these drugs will be weaned off prior to randomization. May be on dextrose feeds. Patient may be a participant in other study protocols such as observational studies, as long as no investigational intervention has taken place within 24 hrs. prior to screening. Less than 12 months of age at screening.

The full record lists 7 inclusion and 9 exclusion criteria; among the exclusions: history of allergy to glucagon or excipients in the csi-glucagon formulation; diazoxide naïve or within five days of starting diazoxide; patients with sepsis.

How the Study Was Run

The set-up was randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the Glucagon group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Blinding on both sides kept the participants and researchers unaware of who received Glucagon.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2016-10 with completion listed for 2018-09.

It ran at 5 sites in United States (5).

Its stated primary purpose is prevention.

What Was Measured

It also tracked 3 secondary outcomes, for example Percent Change in GIR (Double-Blind) and Percent Change in Glucose Infusion Rate (Open-Label).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Glucagon in 5 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 Glucagon compound page, all human trials.