Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome

What Was Tested

In this phase 3 trial, researchers set out to test Carbetocin for hyperparathyroidism.

12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome (PWS).

It had two arms (groups): Carbetocin (experimental) and Placebo (placebo comparator).

Matching placebo nasal spray administered TID.

The registry classes the interventions as drug.

Who Took Part

The registry lists 175 actual participants took part.

It was open to people of any sex aged from 5 years up to 30 years.

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

Examples of who could join: Male or female participants aged 5 to 30 years. Diagnosis of Prader-Willi syndrome with a documented disease-causing genetic mutation. Meeting minimum severity thresholds for hyperphagia based on validated clinical assessments at Screening and Baseline. Residing with a caregiver who is able and willing to comply with study procedures and participate in study visits.

The full record lists 5 inclusion and 10 exclusion criteria; among the exclusions: diagnosis of schaaf-yang syndrome or another genetic, hormonal, or chromosomal disorder other than prader-willi syndrome; active upper respiratory infection at screening or baseline; recent or planned nasal surgery that could affect intranasal drug administration.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

The hyperparathyroidism participants were allocated by chance, so the Carbetocin group and the comparison group should start out alike.

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

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

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

It started in 2023-11-27 with completion listed for 2025-08-19.

It ran at 30 sites in United States (19), Spain (3), United Kingdom (3), Canada (2), Germany (2) and France (1).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change From Baseline at Week 12 in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score (time frame: Baseline to Week 12).

It also tracked 4 secondary outcomes, for example Change From Baseline at Week 12 in Clinical Global Impression of Severity (CGI-S) for PWS Score, Clinical Global Impression of Change (CGI-C) for PWS Score at Week 12 and Change From Baseline at Week 12 in CGI-S for Hyperphagia in PWS Score.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Carbetocin in 175 people with hyperparathyroidism.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Carbetocin compound page, Hyperparathyroidism research.