Liraglutide for Weight Management in Children and Adolescents With Prader-Willi Syndrome and Obesity

Gwenaëlle Diene, Moris Angulo, Paula M. Hale, Cecilie H. Jepsen, Paul L. Hofman, Anita Hokken-Koelega, Chethana Ramesh, Serap Turan, Maïthé Tauber*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

16 Citations (Scopus)
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CONTEXT: Prader-Willi syndrome (PWS) is characterized by lack of appetite control and hyperphagia, leading to obesity. Pharmacological options for weight management are needed. OBJECTIVE: To determine whether liraglutide treatment for weight management is superior to placebo/no treatment in pediatric individuals with PWS. METHODS: This was a multicenter, 52-week, placebo-controlled trial with a 16-week double-blinded period. Adolescents (n = 31, aged 12-17 years; Tanner stage 2-5) and children (n = 24, aged 6-11 years; Tanner stage <2) with PWS and obesity were included. Patients were randomized 2:1 to liraglutide 3.0 mg (or maximum-tolerated dose) or placebo for 16 weeks, after which placebo was stopped. Liraglutide was continued for 52 weeks. All patients followed a structured diet and exercise program throughout the trial. The coprimary endpoints were change in body mass index (BMI) standard deviation score (SDS) from baseline to 16 and 52 weeks. Secondary endpoints included other weight-related parameters, hyperphagia, and safety. RESULTS: Change in BMI SDS from baseline to weeks 16 and 52 was not significantly different between treatments in adolescents (estimated treatment difference: -0.07 at week 16 and -0.14 at week 52) and children (-0.06 and -0.07, respectively). Changes in other weight-related parameters between treatments were not significant. At week 52, hyperphagia total and drive scores were lower in adolescents treated with liraglutide vs no treatment. The most common adverse events with liraglutide were gastrointestinal disorders. CONCLUSION: Although the coprimary endpoints were not met, changes in hyperphagia total and drive scores in adolescents warrant further studies on liraglutide in this population.

Original languageEnglish
Pages (from-to)4-12
Number of pages9
JournalThe Journal of clinical endocrinology and metabolism
Issue number1
Publication statusPublished - 1 Jan 2023

Bibliographical note

Funding Information:
Conflict of Interest G.D. declares no conflicts of interest relevant to this publication. M.A. reports participation as a speaker for Novo Nordisk. P.L.H. has no conflicts of interest. P.M.H. and C.H.J. are employees and stockholders of Novo Nordisk. C.R. is an employee of Novo Nordisk. S.T. and A.H-K. declare no conflicts of interest relevant to this publication. M.T. has received fees for participating in scientific board meetings at Merck Serono, Millendo, Novo Nordisk, and Pfizer, has received a research grant from Pfizer, and holds 3 patents for oxytocin-related products in Prader–Willi syndrome.

Publisher Copyright:
© 2022 The Author(s). Published by Oxford University Press on behalf of the Endocrine Society.


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