Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

What Was Tested

The trial (phase 3) is built around Setmelanotide.

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

Participants received once daily SC injection of placebo (matching setmelanotide) for 14 weeks in a double-blind placebo-controlled treatment period (Period 1).

After the initial 14-week double-blind treatment period, all participants immediately transitioned to open-label setmelanotide once daily SC injection for 38 weeks (Period 2) and then continued to receive setmelanotide in the 14-week open-label treatment period (Period 3).

The registry classes the interventions as drug.

More on the Plan

Eligible participants will enter a 14-week, randomized, double-blind, placebo-controlled treatment period (Period 1) that will be followed by a 38-week open-label treatment period (Period 2) in which all participants will receive setmelanotide.

Following Period 2, participants will continue receiving open-label setmelanotide for 14 weeks (Period 3), after which they could enroll into a separate treatment extension study.

Who Took Part

The registry lists 52 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: BBS clinical diagnosis or AS diagnosis. Greater than or equal to (≥) 6 years of age.

The full record lists 6 inclusion and 16 exclusion criteria; among the exclusions: A Patient Health Questionnaire-9 (PHQ-9) score of ≥15 and/or; participant is, in the opinion of the study investigator, not suitable to participate in the study; inability to comply with once daily (qd) injection regimen.

How the Study Was Run

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

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.

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, investigator and outcomes assessor.

It started in 2018-11-23 with completion listed for 2020-11-16.

It ran at 12 sites in United States (6), France (2), Canada (1), Puerto Rico (1), Spain (1) and United Kingdom (1).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 3 secondary outcomes, for example Mean Percent Change From Baseline in Body Weight (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort and Mean Percent Change From Baseline in the Weekly Average of the Daily Hunger Score (≥12 Years of Age) at Week 52 (Period 2): Pivotal Cohort.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Setmelanotide in 52 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.