A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

What Was Tested

This registered phase 3 trial tested Glucagon.

It had two arms (groups): Standard of Care + dasiglucagon (experimental) and Standard of Care Only (other).

The registry classes the interventions as drug and other.

Who Took Part

The registry lists 32 actual participants took part.

It was open to people of any sex aged from 3 months up to 12 years.

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

Examples of who could join: Established and documented diagnosis of CHI based on standard of care. Previously undergone near-total pancreatectomy or being treated with a non-surgical approach, having been evaluated as not eligible for pancreatic surgery. If somatostatin analogues or sirolimus are used, the therapy should be well established as judged by the investigator, especially when considering their biological half-life.

The full record lists 4 inclusion and 13 exclusion criteria; among the exclusions: previous administration of dasiglucagon; known or suspected allergy to the trial drug or related products; previous participation (randomization) in this trial.

How the Study Was Run

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

Because people in this Glucagon study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

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

Being open-label, the Glucagon study did not mask who received what.

It started in 2019-02-07 with completion listed for 2020-08-31.

It ran at 10 sites in United Kingdom (4), United States (3), Germany (2) and Israel (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Hypoglycemia Episode Rate (time frame: Baseline, Week 2-4 (Treatment Period 1)).

It also tracked 14 secondary outcomes, for example Total Amount of Gastric Carbohydrates Administered to Treat Hypoglycemia, Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia, Extent of Hypoglycemia, Amount of Nightly Gastric Carbohydrates Administered, Total Amount of Gastric Carbohydrates Administered and Percent Time in Hypoglycemia.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Glucagon in 32 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.