Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome

What Was Tested

The trial (phase 2) is built around Setmelanotide.

The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related behavior.

The study drug (setmelanotide and placebo) was administered in a blinded fashion.

It had four arms (groups): one (experimental), one (experimental), one (experimental) and Placebo (placebo comparator).

Participants received setmelanotide 1.5 once daily as a subcutaneous injection from Day 15 to Day 41 (4-week, double-blind randomized treatment period), Day 42 to Day 55 (2-week, randomized withdrawal period), and Day 56 to Day 69 (optional 2-week, open-label extension period).

Participants received setmelanotide 2.5 once daily as a subcutaneous injection from Day 15 to Day 41 (4-week, double-blind randomized treatment period), Day 42 to Day 55 (2-week, randomized withdrawal period), and Day 56 to Day 69 (optional 2-week, open-label extension period).

The registry classes the interventions as drug.

Who Took Part

The registry lists 40 actual participants took part.

It was open to people of any sex aged from 16 years up to 65 years.

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

The full record lists 17 inclusion and 25 exclusion criteria; among the exclusions: recent use (within 3 month) of weight loss agents including herbal medication; A Patient Health Questionnaire-9 (PHQ-9) score of ≥ 15; any suicidal ideation of type 4 or 5 on the columbia-suicide severity rating scale (c-ssrs).

How the Study Was Run

The set-up was randomized, parallel-group and quadruple-blind.

Random assignment meant chance, not the researchers, decided who got Setmelanotide and who got the comparison, which limits bias.

The Setmelanotide group and the comparison group ran side by side rather than switching.

Blinding on both sides kept the participants and researchers unaware of who received Setmelanotide.

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

It started in 2015-03-19 with completion listed for 2016-10-26.

It ran at 5 sites in United States (5).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE) - Period 2 (time frame: Days 15 to 41). Number of Participants Who Experienced a TEAE - Period 3 (time frame: Days 42 to 55). Number of Participants Who Experienced a TEAE - Period 4 (time frame: Days 56 to 69).

It also tracked 19 secondary outcomes, for example Percent Change From Baseline in Hyperphagic Drive Score of PWS Hyperphagia Questionnaire - Period 2, Percent Change From Baseline in Hyperphagic Behaviors Score of PWS Hyperphagia Questionnaire - Period 2, Percent Change From Baseline in Hyperphagic Severity Score of PWS Hyperphagia Questionnaire - Period 2, Percent Change From Baseline in Overall Score of PWS Hyperphagia Questionnaire - Period 3, Percent Change From Baseline in Overall Score of PWS Hyperphagia Questionnaire - Period 4 and Mean Setmelanotide Trough Concentrations.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Setmelanotide in 40 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.