A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

What Was Tested

This registered phase 3 trial tested Setmelanotide.

This is a sub-study of Study RM-493-040 (NCT05774756).

It had two arms (groups): Setemelanotide (experimental) and Placebo (placebo comparator).

Randomized 2:1 (Setmelanotide: Placebo).

Randomized 2:1 (Setmelanotide: Placebo).

The registry classes the interventions as drug.

Who Took Part

39 planned participants were the target.

It was open to people of any sex aged from 4 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Age 4 years and older.

The full record lists 4 inclusion and 12 exclusion criteria; among the exclusions: diagnosis of prader-willi syndrome (pws) or rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (rohhadnet); bariatric surgery or procedure within last 2 years; diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior.

How the Study Was Run

It was run as a randomized, parallel-group and triple-blind study.

Random assignment meant chance, not the researchers, decided who got Setmelanotide and who got the comparison, which limits bias.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Blinding on both sides kept the participants and researchers unaware of who received Setmelanotide.

Those kept unaware of assignments: participant, care provider and investigator.

It started in 2025-09-23 with completion listed for 2027-03-13.

It ran at 11 sites in United States (8) and United Kingdom (3).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Mean % change in BMI (time frame: From Baseline after 26 weeks on a therapeutic regimen).

It also tracked 11 secondary outcomes, for example Mean change in the weekly average of the daily most hunger score in patients ≥12 years old, Mean change in Symptoms of Hyperphagia total score, Mean percent change in weight in patients ≥18 years, Proportion of patients with BMI <30 kg/m2 (patients aged ≥18 years) or <95th percentile (patients aged <18 years) and Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL).

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Setmelanotide in 39 people.

Its size is small, so on its own it can only give an early look.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Setmelanotide compound page, all human trials.