Intranasal Oxytocin for Infants With Prader-Willi Syndrome

University of Florida

Status and phase: Completed; Phase 2; Phase 1

Conditions: Prader-Willi Syndrome

Treatments: Drug: Oxytocin; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT03245762

IRB201700423 -A

OCR16237 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 15 patients

Sex: All

Ages: 1 week to 6 months old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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15 participants in 2 patient groups, including a placebo group

Intranasal oxytocin

Active Comparator group

Description:

Intervention: 4 IU/day of intranasal oxytocin via a nasal spray device each morning.

Treatment:

Drug: Oxytocin

IN-placebo

Placebo Comparator group

Description:

Intervention: 4 IU/day of placebo via nasal spray device each morning.

Treatment:

Drug: Placebo