Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period

What Was Tested

This randomized controlled trial paper looks at Alpha-MSH and Setmelanotide.

The researchers aimed to evaluate the effect of setmelanotide on bodyweight in these patients.

Who Took Part

Patients aged 6 years or older were included if they had a clinical diagnosis of Bardet-Biedl syndrome or Alström syndrome and obesity (defined as BMI >97th percentile for age and sex for those aged 6-15 years and ≥30 kg/m2 for those aged ≥16 years).

How the Study Was Run

In design terms this was a randomized, double-blind, open-label, placebo-controlled and multicenter study.

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

Blinding on both sides kept the participants and researchers unaware of who received Alpha-MSH.

Being open-label, the Alpha-MSH study did not mask who received what.

A placebo (an inactive look-alike) gave the Alpha-MSH group a baseline to be compared against.

This multicentre, randomised, 14-week double-blind, placebo-controlled, phase 3 trial followed by a 52-week open-label period, was performed at 12 sites (hospitals, clinics, and universities) in the USA, Canada, the UK, France, and Spain.

This study is registered with ClinicalTrials.gov, NCT03746522.

What Was Measured

Topics PubMed links to this paper include Receptor, Melanocortin, Type 4, Alstrom Syndrome, Bardet-Biedl Syndrome, Obesity and alpha-MSH.

What This Means in Plain Language

The paper is a randomized, double-blind and open-label study of Alpha-MSH and Setmelanotide.

Its findings take up roughly 6 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2022 (The lancet. Diabetes & endocrinology), a journal based in England.

More context: the Alpha-MSH compound page and the Setmelanotide compound page, all human trials.