Oxytocin Trial in Prader-Willi Syndrome

Phase 1CompletedNCT02013258

University of Florida24 enrolled

Overview

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?

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Prader Willi Syndrome

Interventions

Intranasal oxytocinDRUG

This is a double-blind placebo controlled 2x2 study. Subjects will receive OT for 5 consecutive days during their 7 day stay. This will be followed by a wash out period of 4-6 weeks.

PlaceboOTHER

This is a double-blind placebo controlled 2x2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.

Eligibility

Sex: ALLMin age: 5 YearsMax age: 11 Years

Medical Language ↔ Plain English

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

Locations (3)

University of California, Irvine

Orange, California, United States

University of Florida

Gainesville, Florida, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Outcomes

Primary Outcomes

Safety of intranasal oxytocin in children with Prader-Willi syndrome

Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.

Time frame: 3 months

Secondary Outcomes

Evaluation of food intake in Prader-Willi syndrome

Quantitative evaluation of hyperphagia via the Hyperphagia Questionnaire obtained on days 1, 4 and 6 during the 7 day study protocol and conducted in the evening on these days. Score will range from 0 (no hyperphagia behaviors) to 96 (most severe hyperphagia behaviors). Additionally quantity of food consumed will be recorded on Days 1,4, and 6.

Time frame: 3 months

Data from ClinicalTrials.gov

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