Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome

What Was Tested

This registered phase 3 trial tested Liraglutide.

This trial is conducted globally.

The aim of this trial is to investigate the effect of liraglutide for weight management in paediatric subjects with Prader-Willi Syndrome.

It had two arms (groups): Liraglutide (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 56 actual participants took part.

It was open to people of any sex aged from 6 years up to 18 years.

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

Examples of who could join: Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial. Confirmed diagnosis of PWS (Prader-Willi Syndrome) (by genetic testing). Male or female, age at the time of signing informed consent: - Part A: above or equal to 12 years and less than 18 years. Tanner stage 2-5 pubertal development for part A, and Tanner stage 1 for part B. Stable body weight during the previous 90 days before screening ( below 10 kg self-reported weight change).

The full record lists 7 inclusion and 9 exclusion criteria; among the exclusions: type 1 diabetes mellitus (t1dm); type 2 diabetes mellitus (t2dm); calcitonin equal or above 50 ng/l.

How the Study Was Run

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

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

The Liraglutide group and the comparison group ran side by side rather than switching.

As a double-blind Liraglutide study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant and investigator.

It started in 2015-11-09 with completion listed for 2020-11-04.

It ran at 32 sites in France (13), United States (5), Turkey (Türkiye) (5), Italy (3), Australia (2) and Canada (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks (time frame: Week 0, Week 16). Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 52 Weeks (time frame: Week 0, Week 52).

It also tracked 104 secondary outcomes, for example Change in BMI From Baseline at Week 16, Change in BMI From Baseline at Week 52, Change in Body Weight (Kilogram (kg)) From Baseline at Week 16, Change in Body Weight (kg) From Baseline at Week 52, Change in Body Weight (Pounds (lb)) From Baseline at Week 16 and Change in Body Weight (lb) From Baseline at Week 52.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Liraglutide in 56 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Liraglutide compound page, all human trials.