Setmelanotide in patients aged 2-5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial

What Was Tested

This clinical trial, phase iii paper looks at Alpha-MSH and Setmelanotide.

No approved therapies for patients younger than 6 years old currently exist.

Who Took Part

The age groups tagged for this paper are young children, both men and women.

This phase 3, open-label, multicentre trial, conducted across six sites in the USA, the UK, Spain, and Australia, enrolled eligible patients aged 2-5 years who had hyperphagia and obesity due to biallelic POMC (including PCSK1) or LEPR variants or genetically confirmed BBS.

How the Study Was Run

The design is described as open-label and multicenter.

Everyone knew when Alpha-MSH was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Running at several centres spread the Alpha-MSH study over different clinics and patient groups.

Additional endpoints measured safety, hunger, weight-related outcomes, and caregiver burden.

The study is registered at ClinicalTrials.gov (NCT04966741) and is complete.

What Was Measured

Topics PubMed links to this paper include Receptor, Melanocortin, Type 4, alpha-MSH, Obesity, Pro-Opiomelanocortin, Pediatric Obesity and Hyperphagia.

What This Means in Plain Language

The paper is an open-label and multicenter study of Alpha-MSH and Setmelanotide.

The abstract goes on to report its results in about 10 sentences; this page does not restate them, so read the paper for the findings and their limits.

The publication is from 2025 (The lancet. Diabetes & endocrinology), a journal based in England.

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