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Prader–Willi syndrome trial · GLP Finds editorial

Why the type of obesity matters: liraglutide in Prader–Willi syndrome

A small pediatric trial studied liraglutide in Prader–Willi syndrome.

The short answer

This small pediatric trial did not demonstrate an added weight benefit. An appetite-related finding was interesting, but less conclusive.

Who was studied?

Prader–Willi syndrome is a genetic condition associated with intense hunger and difficulty controlling food intake.

Researchers randomized 56 children and adolescents aged 6–17; 55 entered the main analysis. Participants received daily liraglutide or placebo alongside structured diet and exercise support.

After 16 weeks, placebo injections stopped. Liraglutide continued through week 52.

Weight and appetite findings in a pediatric trial; 56 randomized, 55 analyzed
The main outcome and exploratory appetite findings are distinct

Original visual based on the study.

Weight and appetite findings in a pediatric trial; 56 randomized, 55 analyzed
Group or measureResult
Main BMI outcomeNo clear advantage
Appetite scores at week 52Exploratory findings in adolescents
Study designPlacebo injections stopped after week 16

What did the trial find?

Liraglutide did not clearly outperform the comparison group on the main outcome: BMI adjusted for age and sex. Other weight measurements also showed no clear advantage.

Some appetite scores improved in adolescents at week 52. However, this was an exploratory finding after participants knew their treatment, with many comparisons tested.

What should readers take away?

Results from common obesity cannot automatically be applied to a specific genetic condition. A small negative trial also cannot rule out every possible benefit.

Digestive side effects and low-blood-sugar episodes occurred. Different treatment durations complicate safety comparisons.

Novo Nordisk funded, designed and conducted the trial.

Sources

Diene and colleagues, Journal of Clinical Endocrinology and Metabolism, 2022