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NCT Number: NCT02013258

Oxytocin Trial in Prader-Willi Syndrome

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?

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Key information

Conditions

Prader Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight Prader-Willi Syndrome

Age range

5 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of California, Irvine, Orange, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Intranasal oxytocin

Drug

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.

Other names: Pitocin, Syntocinon

Placebo

Other

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.

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

Other outcomes

  1. Evaluation of social effects of intranasal oxytocin in children with Prader-Willi syndrome

    Time frame: 3 months

    Evaluation of anxiety, food issues, irritability, social communication and behavioral issues will be measured on days 1, 4 and 6 using:

    • Aberrant Behavior Checklist: Behavior rating scale used to measure behavior problems of children and adults with intellectual disabilities ages 6 - 54 years. Scores will range from 0 (no problem behaviors) to 174 (severe aberrant behaviors).
    • Social Responsiveness Scale:designed to measure the breadth of repetitive behaviors. Scores will range from 64 (no repetitive behaviors) to 260 (severe repetitive behaviors).
    • Repetitive Behavior Scales-Revised: Designed to measure the breadth of repetitive behaviors in autism spectrum disorder including: Ritualistic/Sameness Behavior; Stereotypic Behavior; Self-injurious Behavior; Compulsive Behavior; and Restricted Interests. Scores will range from 0 (no autistic behaviors) to 100 (severe autistic behaviors).

  2. Effects of intranasal oxytocin on appetite-regulating hormones

    Time frame: 3 months

    Evaluation of plasma OT, ghrelin and other neuroendocrine hormones involved in appetite regulation (cortisol, orexin A, ghrelin, leptin, oxytocin, insulin).

Sponsors and collaborators

Lead sponsor

University of Florida

Other

Collaborators

  • National Institutes of Health (NIH)

Registry information

Important dates

Study start
2015
Primary completion
2015
Study completion
2015
First posted
Dec 17, 2013
Registry last updated
Apr 5, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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