Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway

What Was Tested

This registered phase 3 trial tested Setmelanotide.

A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.

It had ten arms (groups): one (experimental), one (experimental), one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

Who Took Part

19 actual participants took part.

It was open to people of any sex aged from 6 years with no upper age limit.

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

Examples of who could join: Biallelic or heterozygous POMC/PCSK1 or LEPR (PPL) genetic variants or Bardet-Biedl syndrome (BBS), for which they are being treated with QD setmelanotide. 6 years or older at screening.

How the Study Was Run

The set-up was randomized, crossover and triple-blind.

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.

Each person switched between Setmelanotide and the comparison at some point, usually with a gap in between.

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 2021-12-21 with completion listed for 2023-10-19.

It ran at 7 sites in United States (2), Canada (1), Germany (1), Netherlands (1), Puerto Rico (1) and United Kingdom (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Maximum Drug Concentration (Cmax) of Setmelanotide After QD Administration in the Run-in Period. Cmax of Setmelanotide After QW Administration. Time to Maximum Plasma Concentration (Tmax) of Setmelanotide After QD Administration in the Run-in Period.

It also tracked 3 secondary outcomes, for example Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Number of Participants With Injection Site Reactions (ISRs) From Baseline Through Week 13 and Number of Participants With ISRs From Week 14 Through Week 27.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Setmelanotide in 19 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.