hrp0086fc13.6 | Management of Obesity | ESPE2016

Treatment for Early Onset and Extreme Obesity in Two POMC Deficient Patients: Successful Weight Loss with the Melanocortin-4 Receptor Agonist Setmelanotide

Kuhnen Peter , Clement Karine , Gottesdiener Keith , Fiedorek Fred , van der Ploeg Lex , Wiegand Susanna , Blankenstein Oliver , Gruters Annette , Krude Heiko

Background: POMC deficiency is an extremely rare monogenetic obesity syndrome generally characterized by early onset hyperphagia, red hair and adrenal insufficiency. So far treatment of obesity and hyperphagia with MSH substitution failed, either due to ineffectiveness or side effects of available MC4R agonists.Objective and hypotheses: We hypothesized that the new MC-4R agonist setmelanotide might be a treatment option in POMC deficient patients.<p ...

hrp0097p1-65 | Fat, Metabolism and Obesity | ESPE2023

Natural history of corpulence in patients carrying heterozygous pathogenic variants in the five major genes of the leptin-melanocortin pathway.

Nicaise Clarisse , Dubern Béatrice , Poitou Christine , Clement Karine , Tounian Patrick , Lemale Julie , COURBAGE Sophie , Le Bihan Johanne

Introduction: The leptin-melanocortin pathway plays a key-role in weight control. Pathogenic variants in the five major genes (LEP, LEPR, POMC, PCSK1, MC4R) are associated with early severe obesity. However, the specific associated phenotype with presence of mono-allelic variants and especially BMI trajectories are not well known.Objective: In order to identify specific profiles, we compared BMI trajectories during the f...

hrp0094fc2.2 | Fat, Metabolism and Obesity | ESPE2021

Phase 3 Trial of Setmelanotide in Participants With Bardet-Biedl Syndrome: Placebo-Controlled Results

Argente Jesus , Clement Karine , Dollfus Helene , Han Joan , Haqq Andrea , Martos-Moreno Gabriel , Mittleman Robert , Stewart Murray , Webster Matt , Yanovski Jack , Yuan Guojun , Haws Robert ,

Background: In a Phase 3 trial in participants with obesity due to Bardet-Biedl syndrome (BBS) and Alström syndrome, setmelanotide was associated with significant reduction from baseline in body weight and hunger at Week 52 (primary endpoint), with outcomes driven by responses in individuals with BBS.Methods: In the same trial (NCT03746522), individuals with BBS or Alström syndrome were randomized and received ...