Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity

What Was Tested

The trial (phase 3) is built around Setmelanotide.

It had two arms (groups): Setmelanotide: PPL Group (experimental) and Setmelanotide: BBS Group (experimental).

The registry classes the interventions as drug.

More on the Plan

All participants will be assigned to receive setmelanotide via daily subcutaneous (SC) injection for 1 year.

Who Took Part

The registry lists 12 actual participants took part.

It was open to people of any sex aged from 2 years up to 5 years.

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

Examples of who could join: Participants must have obesity due to either:. BBS confirmed clinical and genetic diagnosis. Age between 2 to \<6 years at the time of informed consent. Obesity, defined as body mass index (BMI) ≥97th percentile for age and gender and body weight of at least 15 kilograms (kg) at the time of enrollment. Symptoms or behaviors of hyperphagia.

How the Study Was Run

Design: randomized, parallel-group and open-label.

Because people in this Setmelanotide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

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

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

It started in 2022-03-08 with completion listed for 2023-09-18.

It ran at 6 sites in United States (3), Australia (1), Spain (1) and United Kingdom (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Mean Percent Change From Baseline in BMI (time frame: Baseline, Week 52).

It also tracked 7 secondary outcomes, for example Mean Absolute Change From Baseline in BMI Z-score, Mean Change From Baseline in Percent of the 95th Percentile of BMI, Mean Change From Baseline in Bone Age, Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3), Change From Baseline in Body Weight and Number of Participants With Treatment-emergent Adverse Events (TEAEs).

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Setmelanotide in 12 people.

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

The trial is marked completed; results are posted on the registry, and this page does not restate them.

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