RM-493 Treatment Trial in Proopiomelanocortin (POMC) Deficient Patients

What Was Tested

In this phase 2 trial, researchers set out to test Setmelanotide.

The purpose of this study is to evaluate the effects of a once daily subcutaneous (SC) injection of RM-493, in subjects with POMC (propiomelanocortin) or other related rare genetic mutations, on body weight, metabolic function and blood pressure.

Patients who respond during the initial 84 days of treatment can enter into long-term (2-year) extensions.

The study drug (RM-493) will be administered in an unblinded fashion.

It had 1 arm (groups): RM-493 Once Daily (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 10 planned participants were the target.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Written informed consent. Homozygous or compound heterozygous (different gene mutation on both alleles) POMC, LEPR or PCSK1 gene mutation. Obesity (BMI \> 30 kg/m2; + 2 BMI SDS). Negative Pregnancy test. No participation in other clinical trials according to AMG (Arzneimittelgesetz) (2 months before and after) at the time of this trial.

The full record lists 16 inclusion and 14 exclusion criteria; among the exclusions: pregnancy or breastfeeding; all contraindications against study medication (including auxiliary substances); interactions with study medication.

How the Study Was Run

The set-up was single-group and open-label.

Being open-label, the Setmelanotide study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2015-01 with completion listed for 2018-01.

It ran at 1 site in Germany.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Effect of RM-493 on Body weight (time frame: Day 1 through Day 84).

It also tracked 5 secondary outcomes, for example Effect of RM-493 on metabolic serum parameters, Effect of RM-493 on blood pressure, Effect of RM-493 on hunger, Effect of RM-493 on body composition/Energy expenditure and Effect of RM-493 on weight loss after treatment continuation of 2 years.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Setmelanotide in 10 people.

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

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

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