The Efficacy, Safety, and Pharmacology of a Ghrelin O-Acyltransferase Inhibitor for the Treatment of Prader-Willi Syndrome

What Was Tested

The paper reports a study of Ghrelin, published as a randomized controlled trial.

Who Took Part

The study group was 28 patients.

A double-blind, placebo-controlled, phase 2 crossover study was conducted with 2 active treatment periods of 28 days in 19 patients (aged 16-65 years; body mass index (BMI) ≥ 28) with genetically confirmed PWS.

How the Study Was Run

The design is described as randomized, double-blind, placebo-controlled and crossover.

The patients were allocated by chance, so the Ghrelin group and the comparison group should start out alike.

Blinding on both sides kept the patients and researchers unaware of who received Ghrelin.

Some patients received a placebo instead of Ghrelin, giving a reference point.

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

The study took place in 7 hospital-based study centers in the United States and Canada.

What Was Measured

Topics PubMed links to this paper include Acyltransferases, Ghrelin, Hyperphagia and Prader-Willi Syndrome.

The authors tagged it with the keywords GLWL-01, Prader-Willi syndrome (PWS) and ghrelin O-acyltransferase (GOAT).

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Ghrelin in 28 patients.

With fewer than 40 people, it is a small study, so it is better read as an early signal for further research than as a settled answer.

Its findings take up roughly 8 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2022 (The Journal of clinical endocrinology and metabolism), a journal based in United States.

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