Oxytocin Trial in Prader-Willi Syndrome

University of Florida

Status and phase: Completed; Phase 1

Conditions: Prader Willi Syndrome

Treatments: Other: Placebo; Drug: Intranasal oxytocin

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT02013258

5208-N

Details and patient eligibility

About

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?

Full description

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.

Enrollment: 24 patients

Sex: All

Ages: 5 to 11 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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24 participants in 2 patient groups, including a placebo group

Intranasal oxytocin

Active Comparator group

Description:

Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.

Treatment:

Drug: Intranasal oxytocin

Placebo

Placebo Comparator group

Description:

Placebo will be administered via nasal spray - 1 spray in each nostril x5 days.

Treatment:

Other: Placebo