DAYBREAK: A Study of Setmelanotide in Participants With Specific Gene Variants in the Melanocortin-4 Receptor (MC4R) Pathway

What Was Tested

The trial (phase 2) is built around Setmelanotide.

It had three arms (groups): Stage 1: Setmelanotide (Open-Label) (experimental), Stage 2: Setmelanotide (Double-Blind) (experimental) and Stage 2: Placebo (Double-Blind) (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

164 actual participants took part.

It was open to people of any sex aged from 6 years up to 65 years.

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

Examples of who could join: Participants must have had a pre-identified genetic variant in an established MC4R pathway gene that contributes to obesity. Age 6 to 65 years, inclusive. Study participant and/or parent or guardian were able to understand all study procedures and provide consent/assent. Symptoms or behaviors of hyperphagia.

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 2021-11-30 with completion listed for 2023-08-04.

It ran at 36 sites in United States (21), Spain (4), United Kingdom (4), Germany (3), Canada (1) and Greece (1).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 9 secondary outcomes, for example Mean Change in BMI From Baseline to the End of Stage 1 in All Participants, Per Genotype, Mean Change in BMI From Baseline to the End of Stage 1 in Participants ≥18 Years Old, Per Genotype, Percent Change in BMI From Baseline to the End of Stage 1 in All Participants, Per Genotype, Percent Change in BMI From Baseline to the End of Stage 1 in Participants ≥18 Years Old, Per Genotype, Mean Change in Body Weight From Baseline to the End of Stage 1 in Participants ≥18 Years Old, Per Genotype and Percent Change in Body Weight From Baseline to the End of Stage 1 in Participants ≥18 Years Old, Per Genotype.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Setmelanotide in 164 people.

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.