NCT03197662

Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome

Phase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Eric Hollander · Academic / Other
• Sex: All
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

This study is a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, Glucagon-Like Peptide-1 (GLP-1), insulin, glucagon, testosterone, and estrogen). If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS. Funding Source- FDA OOPD

Detailed description

Prader-Willi Syndrome (PWS) is a rare neurodevelopmental disorder caused by lack of expression of paternally derived imprinted material on chromosome 15q11-q13. PWS is characterized by mild to moderate intellectual disabilities, repetitive/compulsive behaviors and rigidity, social cognition deficits and severe hypotonia at birth, followed by the onset of hyperphagia later in life. Obesity is responsible for the majority of the morbidity and mortality associated with PWS, and compulsive eating behaviors are most responsible for diminishing the quality of life for caregivers and family members. Oxytocin has been implicated in the pathophysiology of PWS and there have been small studies of intranasal oxytocin (IN-OXT) in this population. To date, however, studies have not been adequately powered to detect significance in target symptoms of hyperphagia and associated symptoms of individuals with PWS. The primary goal of this study is to examine the safety and efficacy of IN-OXT on hyperphagia, as measured by the Hyperphagia Questionnaire-Clinical Trails, from baseline to week 8. Currently, there are no effective treatments available to manage hyperphagia in patients with PWS. STUDY DESIGN: This is an 8-week double-blind, randomized study in 50 children with PWS aged 5-17. Participation involves 2 in-person visits to our program and 5 remote visits. Travel expenses will be reimbursed to participating families.

Conditions

• Prader-Willi Syndrome
• Hyperphagia

Interventions

Type	Name	Description
DRUG	Intranasal Oxytocin (IN-OXT)	Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril every day (4 IU each). If needed, treatment will be titrated due to side effects or non-response.
DRUG	Matched Placebo	Each subject will receive a dose of 16 IU QD, and will be instructed to inhale 2 puffs per nostril every day (4 IU each). If needed, treatment will be titrated due to side effects or non-response.

Timeline

• Start date: 2018-04-11
• Primary completion: 2022-08-29
• Completion: 2022-08-29
• First posted: 2017-06-23
• Last updated: 2025-02-07
• Results posted: 2025-02-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study