Intranasal Oxytocin for Infants With Prader-Willi Syndrome

Phase 1

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Oxytocin; Drug: Placebo

• Registration Number: NCT03245762
• Lead Sponsor: University of Florida

Brief Summary

The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional phase 1a. Videofluoroscopic swallow studies will be performed on treatment day 1 and on the day following treatment morning of day 6.

Detailed Description

The overall objective of this Phase 2 trial is to compare the change from baseline to morning of day 6 of Internasal Oxytocin (IN-OT) on suck and swallow competency in infants/children with Prader-Willi Syndrome (PWS) who are in nutritional phase 1a.

Study Hypothesis 1: The Study team hypothesize that replacing Oxytocin (OT) in infants and children who are in nutritional phase 1a will improve their suck and swallow, potentially even eliminating the need for gastrostomy tubes and nasogastric tubes for feeding, and decreasing the risk of aspiration with oral feeding.

Study Hypothesis 2: The Study team hypothesize that replacing OT in infants and children with PWS will result in improved eye contact, daytime alertness, and feelings of bonding between the parents and the infant.

Study Timeline

• Study Start: 2017-08-01
• Study First Submitted: 2017-08-07
• Study First Submitted QC: 2017-08-07
• Study First Posted: 2017-08-10
• Primary Completion: 2018-01-04
• Study Completion: 2018-01-04
• Results First Submitted: 2019-01-02
• Results First Submitted QC: 2019-06-06
• Results First Posted: 2019-06-25
• Status Verified: 2020-03
• Last Update Submitted: 2020-03-24
• Last Update Posted: 2020-03-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI
2. Physical exam and laboratory results that are within the normal range.
3. Presence of a parent/caregiver/guardian that is able to consent for their participation.

Exclusion Criteria

1. Exposure to any investigational agent in the 30 days prior to randomization.
2. Prior chronic treatment with oxytocin.
3. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Intranasal oxytocin	Oxytocin	Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.
IN-placebo	Placebo	Intervention: 4 IU/day of placebo via nasal spray device each morning.

Primary Outcome Measures

Name	Time	Method
Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a	baseline to day 5	Swallow study Overall improvement

Trial Locations

Locations (1)

University of Florida; 🇺🇸 Gainesville, Florida, United States