Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome

What Was Tested

In this phase 3 trial, researchers set out to test Teduglutide (GLP-2 analog).

It had two arms (groups): Teduglutide (experimental) and Standard of Care (SOC) (other).

Participants will receive standard medical therapy for 24 weeks.

The registry classes the interventions as device, drug and other.

Who Took Part

The registry lists 10 actual participants took part.

It was open to people of any sex aged from 4 months up to 12 months.

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

Examples of who could join: Informed consent by the parent or legal guardian. Male or female infant 4 to 12 months corrected gestational age at screening. Weight at least 5 kilogram (kg) and weight-for-length Z-score greater than -2 at screening and baseline. Parent or legal guardian understands and is willing and able to fully adhere to study requirements as defined in this protocol.

The full record lists 6 inclusion and 22 exclusion criteria; among the exclusions: previous treatment with teduglutide; intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc; unstable cardiac disease.

How the Study Was Run

Design: randomized, parallel-group and open-label.

The participants were allocated by chance, so the Teduglutide (GLP-2 analog) group and the comparison group should start out alike.

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

Being open-label, the Teduglutide (GLP-2 analog) study did not mask who received what.

It started in 2018-08-31 with completion listed for 2020-09-24.

It ran at 8 sites in United Kingdom (4), France (2), Finland (1) and Italy (1).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 22 secondary outcomes, for example Number of Participants With Treatment-emergent Adverse Events (TEAEs), Change From Baseline in Body Weight Z-score at Week 24, Change From Baseline in Length Z-Score at Week 24, Change From Baseline in Head Circumference Z-Score at Week 24, Change From Baseline in Weight-for-Length Z-Score at Week 24 and Change From Baseline in Average Total Urine Output at Week 24.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Teduglutide (GLP-2 analog) in 10 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 Teduglutide (GLP-2 analog) compound page, all human trials.