Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial.

ABSTRACT:

Context

Prader-Willi syndrome (PWS) is a rare genetic disorder characterized by endocrine and neuropsychiatric problems including hyperphagia, anxiousness, and distress. Intranasal carbetocin, an oxytocin analog, was investigated as a selective oxytocin replacement therapy.

Objective

To evaluate safety and efficacy of intranasal carbetocin in PWS.

Design

Randomized, double-blind, placebo-controlled phase 3 trial with long-term follow-up.

Setting

Twenty-four ambulatory clinics at academic medical centers.

Participants

A total of 130 participants with PWS aged 7 to 18 years.

Interventions

Participants were randomized to 9.6 mg/dose carbetocin, 3.2 mg/dose carbetocin, or placebo 3 times daily during an 8-week placebo-controlled period (PCP). During a

SUBMITTER: Roof E 

PROVIDER: S-EPMC10271225 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature