A Trial of Setmelanotide in Acquired Hypothalamic Obesity

What Was Tested

This registered phase 3 trial tested Setmelanotide.

A separate sub-study in patients with congenital HO is detailed under NCT06760546.

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

The registry lists 143 actual participants took part.

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: Documented evidence of acquired hypothalamic obesity (HO). Age 4 years and older.

The full record lists 4 inclusion and 13 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.

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

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

Neither the participants nor the staff measuring them knew who was on Setmelanotide, so expectations could not easily colour the readings.

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

It started in 2023-04-26 with completion listed for 2025-03-18.

It ran at 28 sites in United States (16), Germany (4), Japan (3), United Kingdom (3), Canada (1) and Netherlands (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Pivotal Cohort: Mean Percent Change From Baseline in Body Mass Index (BMI) After 52 Weeks on a Therapeutic Regimen (time frame: Baseline, after approximately 52 Weeks on a Therapeutic Regimen (up to approximately 60 weeks)).

It also tracked 16 secondary outcomes, for example Pivotal Cohort: Change From Baseline in Weekly Average Daily Most Hunger Score in Participants ≥12 Years of Age, Pivotal Cohort: Mean Percent Change From Baseline in Body Weight in Participants ≥18 Years, Pivotal Cohort: Mean Change From Baseline in BMI Z-Score in Participants <18 Years of Age, Pivotal Cohort: Mean Change From Baseline in Percent of BMI 95th Percentile in Participants <18 Years of Age, Pivotal Cohort: Percentage of Participants With BMI <30 kg/m^2 (Aged ≥18 Years) or <95th Percentile (Aged <18 Years) From Baseline and Pivotal Cohort: Mean Change From Baseline in Total Score on the Impact of Weight on Quality of Life-Kids (IWQOL-Kids).

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Setmelanotide in 143 people.

Its status is active not recruiting; results are posted on the registry, and this page does not restate them.

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