Oxytocin Trial in Prader-Willi Syndrome

• Status: Completed
• Trial end date: 2015-08-01
• Target enrollment: 0
• Participant gender: All

Summary

Individuals with Prader-Willi syndrome (PWS) have been found to have a deficit of oxytocin-producing neurons and decreased oxytocin receptor gene function, so the purpose of this study is to determine if oxytocin (OT) administration will improve some of the aspects of Prader-Willi syndrome that are particularly troublesome for children and their families (the insatiable appetite and social behaviors).

The research questions are:

1. Does intranasal oxytocin cause any side effects in children with PWS?

2. Does intranasal oxytocin administration alter appetite or behaviors in PWS?

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Florida
• Collaborator: National Institutes of Health (NIH)
• Treatments: Oxytocin

Criteria

Inclusion Criteria:

• Children with genetically confirmed PWS

• Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday)

• Child must be in nutritional phase 2b or 3, as determined by the PI at each site.

• Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date.

• Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.

• Priority will be given to children currently enrolled in the RDCRN Natural History study

Exclusion Criteria:

• Inability to tolerate intranasal administration of medication

• Hepatic insufficiency (AST/ALT greater than 3 times the normal levels for age)

• Renal insufficiency (BUN/Creatinine greater than 3 times the normal levels for age)

• History of an abnormal ECG (as determined by a cardiologist). If there is any question about cardiac function, ECG reports will be reviewed with a cardiologist prior to enrollment in the study.

• Child not receiving growth hormone treatment

• Child with hypertension or hypotension for age and sex (blood pressure >97% for age and sex or blood pressure <3% for age and sex)

• Diabetes mellitus

• Pregnant or lactating.

• Schizophrenia or psychosis

• Taking any psychotropic medications