Oxytocin Trial in Prader-Willi Syndrome
- Registration Number
- NCT02013258
- Lead Sponsor
- University of Florida
- Brief 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?
- Detailed 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 24
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Placebo Placebo Placebo will be administered via nasal spray - 1 spray in each nostril x5 days. Intranasal oxytocin Intranasal oxytocin Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.
- Primary Outcome Measures
Name Time Method Safety of intranasal oxytocin in children with Prader-Willi syndrome 3 months Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
- Secondary Outcome Measures
Name Time Method Evaluation of food intake in Prader-Willi syndrome 3 months 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.
Trial Locations
- Locations (3)
University of Florida
🇺🇸Gainesville, Florida, United States
University of California, Irvine
🇺🇸Orange, California, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States