Intranasal Oxytocin for Infants With Prader-Willi Syndrome

Study Overview
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Brief Summary
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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
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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.

Official Title
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Intranasal Oxytocin for Treatment of Infants and Children With Prader-Willi Syndrome in Nutritional Phase 1a - Phase 2 Study

Conditions
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Prader-Willi Syndrome

Intervention / Treatment
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* Drug: Oxytocin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Individuals with genetically confirmed PWS who are in nutritional phase 1a, as determined by PI Physical exam and laboratory results that are within the normal range. Presence of a parent/caregiver/guardian that is able to consent for their participation. Exclusion Criteria: Exposure to any investigational agent in the 30 days prior to randomization. Prior chronic treatment with oxytocin. A medical condition that might interfere with the conduct of the study, confound interpretation of study results or endanger the subject's well-being.

Ages Eligible for Study
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Minimum Age: 1 Week
Maximum Age: 6 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Intranasal oxytocin<br>Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning. | Drug: Oxytocin<br>* 4 IU/day of oxytocin administered via nasal spray device each morning.<br>* Other names: Syntocinon;|
| Placebo Comparator: IN-placebo<br>Intervention: 4 IU/day of placebo via nasal spray device each morning. | Drug: Placebo<br>* 4 IU/day of placebo administered via nasal spray device each morning<br>* Other names: Saline;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Suck and Swallow Competency in Infants/Children With PWS Who Are in Nutritional Phase 1a | Swallow study Overall improvement | baseline to day 5 |