A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older

What Was Tested

The trial (phase 3) is built around Teduglutide (GLP-2 analog).

The main aims of the study are to check for side effects from teduglutide.

Participants will receive a daily injection of teduglutide just under the skin (subcutaneous) for 24 weeks.

Then they are followed up for another 4 weeks.

It had 1 arm (groups): one (experimental).

The registry classes the interventions as drug.

More on the Plan

A study of teduglutide in Japanese children with short bowel syndrome aged 4 months or older.

Who Took Part

3 actual participants took part.

It was open to people of any sex aged from 4 months with no upper age limit.

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

Examples of who could join: Male or female pediatric patient of corrected gestational age 4 months or older.

The full record lists 4 inclusion and 20 exclusion criteria; among the exclusions: A parent/guardian who is not capable of understanding or not willing to adhere to the study visit schedule and other protocol requirements; intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc; cardiac disease that makes the patient vulnerable to changes in fluid status.

How the Study Was Run

Design: single-group and open-label.

Nothing was hidden in this open-label Teduglutide (GLP-2 analog) study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2022-01-04 with completion listed for 2023-09-27.

It ran at 6 sites in Japan (6).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Participants With Treatment-emergent Adverse Events (TEAEs). Number of Participants With Serious Adverse Events (SAEs). Number of Participants With Adverse Events of Special Interest (AESIs).

It also tracked 5 secondary outcomes, for example Change From Baseline in PS Volume, Percent Change From Baseline in PS Volume, Number of Participants Who Achieved Enteral Autonomy and Change From Baseline in Number of Days Per Week of PS Usage at EOT.

What This Means in Plain Language

Put simply, this is a phase 3 trial of Teduglutide (GLP-2 analog) in 3 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.