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?
- Study Type: Interventional
- Study Design
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Primary Purpose: Treatment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Study Primary Completion Date: August 2015
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.
- Drug: Intranasal oxytocin
- This is a double-blind placebo controlled 2×2 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.
- Other: Placebo
- This is a double-blind placebo controlled 2×2 study. Placebo will be given via intranasal spray, one spray in each nostril daily x 5 days. One month interval between arms of treatment.
Arms, Groups and Cohorts
- Active Comparator: Intranasal oxytocin
- Intranasal oxytocin. 16 IU intranasal oxytocin x 5 days. One month interval between arms of treatment.
- Placebo Comparator: Placebo
- Placebo will be administered via nasal spray – 1 spray in each nostril x5 days.
Clinical Trial Outcome Measures
- Safety of intranasal oxytocin in children with Prader-Willi syndrome
- Time Frame: 3 months
- Occurrence of adverse event, description and quantification of clinical and behavior severity, pre- and post- intranasal oxytocin and placebo administration.
- Evaluation of food intake in Prader-Willi syndrome
- Time Frame: 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.
Participating in This Clinical Trial
- 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
Gender Eligibility: All
Minimum Age: 5 Years
Maximum Age: 11 Years
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- University of Florida
- National Institutes of Health (NIH)
- Provider of Information About this Clinical Study
- Overall Official(s)
- Jennifer L Miller, MD, Principal Investigator, University of Florida
Tauber M, Mantoulan C, Copet P, Jauregui J, Demeer G, Diene G, Rogé B, Laurier V, Ehlinger V, Arnaud C, Molinas C, Thuilleaux D. Oxytocin may be useful to increase trust in others and decrease disruptive behaviours in patients with Prader-Willi syndrome: a randomised placebo-controlled trial in 24 patients. Orphanet J Rare Dis. 2011 Jun 24;6:47. doi: 10.1186/1750-1172-6-47.
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