Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials
Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials
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DOI：
10.1016/s2213-8587(20)30364-8
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发表时间：
2020-12-01
影响因子：
44.5
通讯作者：
Kuehnen, Peter
中科院分区：
文献类型：
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作者：
Clement, Karine;van den Akker, Erica;Kuehnen, Peter
黑皮质素4受体（melanocortin 4 receptor，MC 4 R）是瘦素-黑皮质素途径的一个组成部分，参与体重调节。严重的早发性肥胖可能是由影响MC 4 R通路的基因中的双等位基因变体引起的。我们报告了MC 4受体激动剂setmelanotide在因阿黑皮素原（POMC）缺乏性肥胖或瘦素受体（LEPR）缺乏性肥胖而严重肥胖的个体中的试验结果。这些单臂、开放标签、多中心、3期试验在加拿大、美国、比利时、法国、德国、荷兰和英国的10家医院进行。6岁或以上POMC或LEPR缺乏性肥胖的参与者接受开放标签的setmelanotide治疗12周。体重减轻至少5 kg的受试者（或>= 5%，如果体重≥ 5%，儿科受试者，如果基线体重<
Background The melanocortin 4 receptor (MC4R), a component of the leptin-melanocortin pathway, plays a part in bodyweight regulation. Severe early-onset obesity can be caused by biallelic variants in genes that affect the MC4R pathway. We report the results from trials of the MC4R agonist setmelanotide in individuals with severe obesity due to either pro-opiomelanocortin (POMC) deficiency obesity or leptin receptor (LEPR) deficiency obesityMethods These single-arm, open-label, multicentre, phase 3 trials were done in ten hospitals across Canada, the USA, Belgium, France, Germany, the Netherlands, and the UK. Participants aged 6 years or older with POMC or LEPR deficiency obesity received open-label setmelanotide for 12 weeks. Participants with at least 5 kg weight loss (or >= 5% if weighing = 5% in paediatric participants if baseline bodyweight was