Oxytocin Trial in Prader-Willi Syndrome

This study is to investigate if intranasal oxytocin will improve hyperphagia, social skills,
and behaviors in subjects with Prader-Willi syndrome. This will be a randomized placebo
controlled pilot study. The primary outcome measure is to determine if intranasal
administration of oxytocin will cause any adverse events in subjects with Prader-Willi
syndrome. Secondarily, the investigators will also perform evaluations to determine if
intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with
Prader-Willi syndrome.

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